Scholars@Duke
Harry Paul Erba

Harry Paul Erba CV

Professor of Medicine
Medicine, Hematologic Malignancies and Cellular Therapy
harry.erba@duke.edu
DUMC Box 3961, Durham, NC 27710
2400 Pratt St Suite 5000, DUMC Box 3961, Durham, NC 27710
Office hours
CV

Selected Publications

V-FAST: a phase 1b master trial to investigate CPX-351 combined with targeted agents in adults with newly diagnosed AML.

Journal Article Blood Neoplasia · November 2025 Preclinical data suggest CPX-351, approved for patients with newly diagnosed therapy-related acute myeloid leukemia (AML) or AML with myelodysplasia-related changes, may exhibit synergy with targeted agents, suggesting a rationale for combining targeted ag ... Full text Link to item Cite

Ziftomenib in Relapsed or Refractory NPM1-Mutated AML.

Journal Article J Clin Oncol · November 2025 PURPOSE: Ziftomenib-a potent, highly selective, oral menin inhibitor-was well tolerated and demonstrated encouraging clinical activity as monotherapy for relapsed/refractory NPM1-mutated (NPM1-m) and KMT2A-rearranged AML in the KOMET-001 phase I trial. MET ... Full text Link to item Cite

FLT3-ITD measurable residual disease from the QuANTUM-First trial.

Journal Article Blood advances · October 2025 QuANTUM-First was a randomized trial that demonstrated that the addition of quizartinib, a potent and selective FLT3 inhibitor, to induction and consolidation chemotherapy, followed by monotherapy maintenance, improved the survival for patients with newly ... Full text Cite

Impact of hematopoietic cell transplantation and quizartinib in newly diagnosed patients with acute myeloid leukemia and FMS-like tyrosine kinase 3-internal tandem duplications in the QuANTUM-First trial.

Journal Article Haematologica · September 1, 2025 QuANTUM-First (ClinicalTrials.gov identifier: NCT02668653) was a randomized phase III trial in patients with newly diagnosed FLT3-internal tandem duplication (ITD)-positive acute myeloid leukemia (AML) treated with quizartinib or placebo plus standard indu ... Full text Link to item Cite

Synergistic Activity of Combined FLT3-ITD and MDM2 Inhibition with Quizartinib and Milademetan in FLT3-ITD Mutant/TP53 Wild-type Acute Myeloid Leukemias.

Journal Article Clin Cancer Res · July 15, 2025 PURPOSE: Acute myeloid leukemia (AML) is characterized by frequent mutations in FMS-like tyrosine kinase 3 (FLT3), overexpression of murine double minute 2 (MDM2), and TP53 wild-type (WT). Monotherapies targeting FLT3 frequently result in the development o ... Full text Link to item Cite

Data from Synergistic Activity of Combined FLT3-ITD and MDM2 Inhibition with Quizartinib and Milademetan in <i>FLT3</i>-ITD Mutant/<i>TP53</i> Wild-type Acute Myeloid Leukemias

Other · July 15, 2025 <div>AbstractPurpose:<p>Acute myeloid leukemia (AML) is characterized by frequent mutations in FMS-like tyrosine kinase 3 (<i>FLT3</i>), overexpression of murine double minute 2 (MDM2), and <i>TP53</i> wild-type ... Full text Cite

Evaluating the impact of stratification on the power and cross-arm balance of randomized phase 2 clinical trials.

Journal Article Clin Trials · June 2025 Background/aimsRandomized clinical trials often use stratification to ensure balance between arms. Analysis of primary endpoints of these trials typically uses a "stratified analysis," in which analyses are performed separately in each subgroup defined by ... Full text Link to item Cite

Frontline Therapy of AML in the Fit and Younger Population-Incorporating Molecularly Targeted Agents.

Journal Article Am J Hematol · March 2025 Backbone therapy for acute myeloid leukemia for younger adults has for 50 years been based on a combination of cytarabine and anthracycline. Over the past 10 years the addition of several targeted agents has been found to improve the outcomes of subsets of ... Full text Link to item Cite

Proteogenomic characterization of highly enriched viable leukemic blasts in acute myeloid leukemia: A SWOG report.

Journal Article EJHaem · December 2024 INTRODUCTION: Acute myeloid leukemia (AML) remains one of the deadliest hematopoietic malignancies. A better understanding of the molecular biology governing AML may lead to improved risk stratification and facilitate the development of novel therapies. Pr ... Full text Link to item Cite

Correlation of Baseline Gene Mutations With Quizartinib Efficacy in Patients With FLT3-ITD-Positive Newly Diagnosed Acute Myeloid Leukemia in the Phase 3 QuANTUM-First Trial

Conference Blood · November 5, 2024 Background: Acute myeloid leukemia (AML) is characterized by genetic heterogeneity, poor overall survival (OS), and variable treatment responses. FLT3 gene mutations are among the most fr ... Full text Cite

Efficacy and Safety of Uproleselan Combined with Chemotherapy Vs. Chemotherapy Alone in Relapsed/Refractory Acute Myeloid Leukemia: Findings from an International Phase 3 Trial

Conference Blood · November 5, 2024 Introduction: Uproleselan (GMI-1271) is an E-selectin antagonist that disrupts AML cell survival pathways, overcomes chemotherapy resistance, and potentially deepens chemotherapy response ... Full text Cite

Real-World Treatment Patterns and Effectiveness of Midostaurin Versus Quizartinib in FLT3-ITD Mutated Acute Myeloid Leukemia Undergoing Intensive Induction

Conference Blood · November 5, 2024 Introduction:Mutations in FMS-like tyrosine kinase 3 (FLT3) are one of the most common genetic alterations in acute myeloid leukemia (AML), with intern ... Full text Cite

Evaluation of ELN2022 Risk Stratification in NPM1 Mutated AML: A Study from the Fred Hutch and SWOG

Conference Blood · November 5, 2024 NPM1 mutations (NPM1+) are found in about 30% of adult AML. While NPM1+ are generally associated with favorable risk, prior studies have shown that NPM1+ older patients, defined as ≥ 65 y ... Full text Cite

Consent to Future Use of Residual and Banked Specimens Among Adults with Hematologic Malignancies: Analyzing SWOG Clinical Trial Data from 2000-2024

Conference Blood · November 5, 2024 Introduction: Racial and socioeconomic disparities have been shown to influence long-term outcomes among adults with hematologic malignancies. Equitable research participation in clinical ... Full text Cite

Phase 1 Results: First-in-Human Phase 1/2 Study of the Menin-MLL Inhibitor Enzomenib (DSP-5336) in Patients with Relapsed or Refractory Acute Leukemia

Conference Blood · November 5, 2024 Introduction: Menin inhibitors are an important new class of agents in development for acute leukemia. Over expression of HOXA9 and MEIS1 driven by the menin and KMT2A/MLL interaction occ ... Full text Cite

Ziftomenib Combined with Intensive Induction (7+3) in Newly Diagnosed NPM1-m or KMT2A-r Acute Myeloid Leukemia: Interim Phase 1a Results from KOMET-007

Conference Blood · November 5, 2024 Background: Nucleophosmin 1 (NPM1) mutations and lysine methyltransferase 2A (KMT2A) rearrangements drive leukemogenesis in approximately 35-40% of acute myeloid leukemias (AML). Ziftomen ... Full text Cite

Phase 1 Safety and Efficacy of Tuspetinib Plus Venetoclax Combination Therapy in Study Participants with Relapsed or Refractory Acute Myeloid Leukemia (AML) Support Exploration of Triplet Combination Therapy of Tuspetinib Plus Venetoclax and Azacitidine for Newly Diagnosed AML

Conference Blood · November 5, 2024 INTRODUCTION: Combination therapy with a hypomethylating agent (HMA) and venetoclax (VEN) has improved response rates and overall survival of newly diagnosed AML patients. However, long-t ... Full text Cite

Results from a Phase 1 Open-Label Dose Escalation and Expansion Trial of Oral Azacitidine + Cedazuridine (ASTX030) in Patients with Myelodysplastic Syndromes (MDS) and MDS/Myeloproliferative Neoplasms (MPN)

Conference Blood · November 5, 2024 Background:Azacitidine (AZA) and decitabine (DEC) are parenteral DNA methyltransferase inhibitors (DNMTis) approved for the treatment of patients with ... Full text Cite

Addition of Inotuzumab to a Pediatric Inspired Chemotherapy Regimen in Young Adult Patients with B-Cell Acute Lymphoblastic Leukemia: Findings from the Alliance A041501 Phase 3 Randomized Trial

Conference Blood · November 5, 2024 Introduction: Inotuzumab ozogamicin (INO) is an antibody targeting CD22 conjugated with a cytotoxic antitumor antibiotic (calicheamicin) approved for treatment of relapsed/refractory CD22 ... Full text Cite

4q Copy Neutral Loss of Heterozygosity and KMT2A Partial Tandem Duplication Identified By Chromosomal Genomic Array Testing (CGAT) Correlated with Decreased Overall Survival in Acute Myeloid Leukemia (AML)

Conference Blood · November 5, 2024 Background:CGAT can find copy neutral loss of heterozygosity (cnLOH) and genomic copy number alterations (CNAs) that are undetectable by conventional f ... Full text Cite

Anchored Matching Adjusted Indirect Treatment Comparison of Quizartinib Vs Midostaurin in Newly Diagnosed Patients with FLT3-ITD-Positive Acute Myeloid Leukaemia

Conference Blood · November 5, 2024 IntroductionQuizartinib and midostaurin are FLT3 inhibitors approved for newly diagnosed (ND) fit FLT3-ITD+ AML based on QuANTUM-First (Q-F) and RATIFY ... Full text Cite

Quantum-First: Effects of Quizartinib (Q) on RFS, OS, CIR, and MRD in Newly Diagnosed (nd) Patients (pts) with FMS-like Tyrosine Kinase 3-Internal Tandem Duplication-Positive (FLT3-ITD+) Acute Myeloid Leukemia (AML) Who Received Continuation (CONT) Therapy (tx)

Conference Blood · November 5, 2024 Introduction: The phase 3 QuANTUM-First study (NCT02668653) demonstrated that in nd FLT3-ITD+ AML pts, adding the oral, highly potent, selective, type 2 FLT3 inhibitor Q to standard chemo ... Full text Cite

Trial in Progress: The Phase 3, Randomized, Double-Blind, Placebo-Controlled QuANTUM-Wild Study of Quizartinib in Combination With Chemotherapy and as Single-Agent Maintenance in Newly Diagnosed, FLT3-ITD-Negative Acute Myeloid Leukemia

Conference Blood · November 5, 2024 BACKGROUND: Quizartinib (Quiz) is an oral, selective, type-II inhibitor of FMS-like tyrosine kinase 3 (FLT3), with potent binding affinity against wild-type (wt) FLT3, FLT3 internal tande ... Full text Cite

Ziftomenib Combined with Venetoclax/Azacitidine in Relapsed/Refractory NPM1-m or KMT2A-r Acute Myeloid Leukemia: Interim Phase 1a Results from KOMET-007

Conference Blood · November 5, 2024 Background: In approximately 35-40% of acute myeloid leukemia (AML) cases, leukemogenesis is driven by nucleophosmin 1 (NPM1) mutations or rearrangements in lysine methyltransferase 2A (K ... Full text Cite

Addition of Blinatumomab to Consolidation Therapy Among Older Newly Diagnosed Patients (pts) with BCR::ABL1 Negative B-Lineage Acute Lymphoblastic Leukemia (ALL) in the ECOG-ACRIN E1910 Randomized Phase III Trial

Conference Blood · November 5, 2024 IntroductionE1910 randomized pts with ALL aged 30-70 years who were measurable residual disease negative (MRD-) by multi-color flow cytometry (FC) afte ... Full text Cite

Assessment of Outcomes of Allogeneic Stem Cell Transplantation By Treatment Arm in Newly Diagnosed Measurable Residual Disease Negative Patients with B-Lineage Acute Lymphoblastic Leukemia Randomized to Conventional Chemotherapy +/- Blinatumomab in the ECOG-ACRIN E1910 Phase III National Clinical Trials Network Trial

Conference Blood · November 5, 2024 Introduction: Based on a donor versus no-donor analysis, UKALLXII/E2993 showed a survival benefit for adults with acute lymphoblastic leukemia (ALL) who underwent allogeneic hematopoietic ... Full text Cite

Ziftomenib in relapsed or refractory acute myeloid leukaemia (KOMET-001): a multicentre, open-label, multi-cohort, phase 1 trial.

Journal Article Lancet Oncol · October 2024 BACKGROUND: Ziftomenib (KO-539) is an oral selective menin inhibitor with known preclinical activity in menin-dependent acute myeloid leukaemia models. The primary objective of this study was to determine the recommended phase 2 dose in patients with relap ... Full text Link to item Cite

Blinatumomab for MRD-Negative Acute Lymphoblastic Leukemia in Adults.

Journal Article N Engl J Med · July 25, 2024 BACKGROUND: Many older adults with B-cell precursor acute lymphoblastic leukemia (BCP-ALL) have a relapse despite having a measurable residual disease (MRD)-negative complete remission with combination chemotherapy. The addition of blinatumomab, a bispecif ... Full text Link to item Cite

Association between class III obesity and overall survival in previously untreated younger patients with acute myeloid leukemia enrolled on SWOG S1203.

Journal Article Leukemia · July 2024 There has been ongoing debate on the association between obesity and outcomes in acute myeloid leukemia (AML). Currently few studies have stratified outcomes by class I obesity, class II obesity, and class III obesity, and a more nuanced understanding is b ... Full text Link to item Cite

Follow-up from the A041202 study shows continued efficacy of ibrutinib regimens for older adults with CLL.

Journal Article Blood · April 18, 2024 A041202 (NCT01886872) is a phase 3 study comparing bendamustine plus rituximab (BR) with ibrutinib and the combination of ibrutinib plus rituximab (IR) in previously untreated older patients with chronic lymphocytic leukemia (CLL). The initial results show ... Full text Link to item Cite

Abstract CT060: Phase 1 study of azacitidine in combination with evorpacept for higher-risk myelodysplastic syndrome (MDS)

Conference Cancer Research · April 5, 2024 AbstractBackground:Evorpacept (EVO) is a high affinity CD47-blocking fusion protein with an inactive human immunoglobulin Fc region designed to enhance the activity of other anti-neoplastic therapies with mi ... Full text Cite

Association between class III obesity and overall survival in previously untreated younger patients with acute myeloid leukemia enrolled on SWOG S1203.

Journal Article Res Sq · March 11, 2024 There has been ongoing debate on the association between obesity and outcomes in acute myeloid leukemia (AML). Currently there are few studies that have stratified outcomes by class I obesity, class II obesity, and class III obesity; and a more nuanced und ... Full text Link to item Cite

Quantification of measurable residual disease using duplex sequencing in adults with acute myeloid leukemia.

Journal Article Haematologica · February 1, 2024 The presence of measurable residual disease (MRD) is strongly associated with treatment outcomes in acute myeloid leukemia (AML). Despite the correlation with clinical outcomes, MRD assessment has yet to be standardized or routinely incorporated into clini ... Full text Link to item Cite

A randomized phase III study of standard versus high-dose cytarabine with or without vorinostat for AML.

Journal Article Leukemia · January 2024 Prior experience indicated that use of higher doses of cytarabine during induction for acute myeloid leukemia (AML) with a histone deacetylase inhibitor resulted in high response rates. S1203 was a randomized multicenter trial for previously untreated pati ... Full text Link to item Cite

The New NCI Precision Medicine Trials.

Journal Article Clin Cancer Res · December 1, 2023 Basket, umbrella, and platform trial designs (master protocols) have emerged over the last decade to study precision medicine approaches in oncology. First-generation trials like NCI-MATCH (Molecular Analysis for Therapy Choice) have proven the principle t ... Full text Link to item Cite

Treatment Patterns and Outcomes of Patients with Acute Myeloid Leukemia (AML) from 2013 to 2022: A Connect ® Myeloid Registry Study

Conference Blood · November 2, 2023 IntroductionThere has been a rapid evolution in diagnostic and management evaluation for AML, associated with the introduction of new therapies, some of which target specific mutations, ... Full text Cite

Kinetics of Complete Remission (CR) and CR Duration and Its Impact on Overall Survival (OS) and Event-Free Survival (EFS) in Quantum-First

Conference Blood · November 2, 2023 BackgroundResults from the phase 3 QuANTUM-First study (NCT02668653) showed that in patients (pts) with newly diagnosed (nd) acute myeloid leukemia (AML) and positive for FMS-like tyrosi ... Full text Cite

Quantum-First Trial: FMS-like Tyrosine Kinase 3-Internal Tandem Duplication ( FLT3-ITD)-Specific Measurable Residual Disease (MRD) Clearance Assessed through Induction (IND) and Consolidation (CONS) Is Associated with Improved Overall Survival (OS) in Newly Diagnosed (nd) FLT3-ITD+ AML Patients (pts)

Conference Blood · November 2, 2023 BackgroundDetection of MRD by flow cytometry or gene fusion transcript quantitation increasingly is used to guide treatment decisions in AML. Molecular monitoring of recurrent gene mutat ... Full text Cite

Quantum-First: Safety By Treatment Phase and By Age in Newly Diagnosed (nd) Patients (pts) with FMS-like Tyrosine Kinase 3-Internal Tandem Duplication ( FLT3-ITD) Positive Acute Myeloid Leukemia (AML)

Conference Blood · November 2, 2023 BackgroundResults from the phase 3 QuANTUM-First study (NCT02668653) showed that in nd FLT3-ITD+ AML pts, the addition of the oral, highly potent, selective, type 2 FLT3 inhibitor quizar ... Full text Cite

Assessment of Outcomes of Consolidation Therapy By Number of Cycles of Blinatumomab Received in Newly Diagnosed Measurable Residual Disease Negative Patients with B-Lineage Acute Lymphoblastic Leukemia: In the ECOG-ACRIN E1910 Randomized Phase III National Clinical Trials Network Trial

Conference Blood · November 2, 2023 Introduction: ECOG ACRIN E1910 is a randomized phase III trial that showed that adults with newly diagnosed BCR::ABL1 negative acute lymphoblastic leukemia (ALL) who become MRD negative (&lt;0.01%) after induction c ... Full text Cite

Phase 1/2 First-in-Human Study of the Menin-MLL Inhibitor DSP-5336 in Patients with Relapsed or Refractory Acute Leukemia

Conference Blood · November 2, 2023 Introduction: Menin inhibitors are an exciting new class of agents in development for patients (pts) with acute leukemia who overexpress HOXA9 and MEIS1 genes. Although there is a growing list of fusion genes in acute l ... Full text Cite

Class III Obesity Is Associated with Worse Overall Survival in Previously Untreated Younger Patients with Acute Myeloid Leukemia Enrolled on SWOG 1203

Conference Blood · November 2, 2023 Mohamed Sorror and Mary-Elizabeth Percival contributed equally to this project.Introduction:There has been inconsistent evidence supporting the associati ... Full text Cite

Long-Term Follow up for SWOG 1318: Combination of Dasatinib, Prednisone, and Blinatumomab for Older Patients with Philadelphia-Chromosome (Ph) Positive Acute Lymphoblastic Leukemia (ALL)

Conference Blood · November 2, 2023 Tyrosine kinase inhibitors (TKIs) have improved the outcomes of patients with Philadelphia chromosome positive (Ph+) acute lymphoblastic leukemia (ALL). Many older patients (pts) are not good candidates for intensive ch ... Full text Cite

Characteristics and prognostic impact of IDH mutations in AML: a COG, SWOG, and ECOG analysis.

Journal Article Blood Adv · October 10, 2023 Somatic mutations in isocitrate dehydrogenase (IDH) genes occur frequently in adult acute myeloid leukemia (AML) and less commonly in pediatric AML. The objective of this study was to describe the prevalence, mutational profile, and prognostic significance ... Full text Link to item Cite

A phase 1b study of venetoclax and alvocidib in patients with relapsed/refractory acute myeloid leukemia.

Journal Article Hematol Oncol · October 2023 Relapsed/refractory (R/R) Acute Myeloid Leukemia (AML) is a genetically complex and heterogeneous disease with a poor prognosis and limited treatment options. Thus, there is an urgent need to develop therapeutic combinations to overcome drug resistance in ... Full text Link to item Cite

Genomic Data Heterogeneity across Molecular Diagnostic Laboratories: A Real-World Connect Myeloid Disease Registry Perspective on Variabilities in Genomic Assay Methodology and Reporting.

Journal Article J Mol Diagn · August 2023 Genomic data variability from laboratory reports can impact clinical decisions and population-level analyses; however, the extent of this variability and the impact on the data's value are not well characterized. This pilot study used anonymized genetic an ... Full text Link to item Cite

Transplantation Referral Patterns for Patients with Newly Diagnosed Higher-Risk Myelodysplastic Syndromes and Acute Myeloid Leukemia at Academic and Community Sites in the Connect® Myeloid Disease Registry: Potential Barriers to Care.

Journal Article Transplant Cell Ther · July 2023 Hematopoietic stem cell transplantation (HCT) is indicated for patients with higher-risk (HR) myelodysplastic syndromes (MDS) and acute myeloid leukemia (AML). Age, performance status, patient frailty, comorbidities, and nonclinical factors (eg, cost, dist ... Full text Link to item Cite

Acute myeloid leukaemia.

Journal Article Lancet · June 17, 2023 Progress in acute myeloid leukaemia treatment is occurring at an unprecedented pace. The past decade has witnessed an increasingly improved scientific understanding of the underlying biology of acute myeloid leukaemia, leading to enhanced prognostication t ... Full text Link to item Cite

Quizartinib plus chemotherapy in newly diagnosed patients with FLT3-internal-tandem-duplication-positive acute myeloid leukaemia (QuANTUM-First): a randomised, double-blind, placebo-controlled, phase 3 trial.

Journal Article Lancet · May 13, 2023 BACKGROUND: Patients with acute myeloid leukaemia (AML) positive for internal tandem duplication (ITD) mutations of FLT3 have poor outcomes. Quizartinib, an oral, highly potent, selective, type 2 FLT3 inhibitor, plus chemotherapy showed antitumour activity ... Full text Link to item Cite

Abstract 5560: Prognostic effect of co-mutations on patients with NPM1+ acute myeloid leukemia (AML) enrolled in the Connect® Myeloid registry

Conference Cancer Research · April 4, 2023 AbstractIntroduction: The 2017 World Health Organization classification of myeloid neoplasms and acute myeloid leukemia (AML) recognizes AML with mutated NPM1 as a distinct entity with favorable prognosis. H ... Full text Cite

Dasatinib/prednisone induction followed by blinatumomab/dasatinib in Ph+ acute lymphoblastic leukemia.

Journal Article Blood advances · April 2023 Novel treatment strategies are needed for the treatment of Philadelphia chromosome-positive (Ph+) acute lymphoblastic leukemia (ALL) in older patients. This trial evaluated the feasibility and outcomes with the anti-CD19 bispecific T-cell-engaging antibody ... Full text Cite

Quantification of measurable residual disease using duplex sequencing in adults with acute myeloid leukemia.

Journal Article medRxiv · March 27, 2023 The presence of measurable residual disease (MRD) is strongly associated with treatment outcomes in acute myeloid leukemia (AML). Despite the correlation with clinical outcomes, MRD assessment has yet to be standardized or routinely incorporated into clini ... Full text Link to item Cite

Verification of prognostic expression biomarkers is improved by examining enriched leukemic blasts rather than mononuclear cells from acute myeloid leukemia patients.

Journal Article Biomark Res · March 16, 2023 BACKGROUND: Studies have not systematically compared the ability to verify performance of prognostic transcripts in paired bulk mononuclear cells versus viable CD34-expressing leukemic blasts from patients with acute myeloid leukemia. We hypothesized that ... Full text Link to item Cite

A phase Ib trial of mivavotinib (TAK-659), a dual SYK/FLT3 inhibitor, in patients with relapsed/refractory acute myeloid leukemia.

Journal Article Haematologica · March 1, 2023 Mivavotinib (TAK-659) is an investigational type 1 tyrosine kinase inhibitor with dual activity against spleen tyrosine kinase (SYK) and FMS-like tyrosine kinase 3 (FLT3). We conducted a phase Ib study to investigate the safety, tolerability, and efficacy ... Full text Link to item Cite

The impact of early PEG-asparaginase discontinuation in young adults with ALL: a post hoc analysis of the CALGB 10403 study.

Journal Article Blood Adv · January 24, 2023 Asparaginase is a key component of pediatric-inspired regimens in young adults with acute lymphoblastic leukemia (ALL). Truncation of asparaginase therapy is linked to inferior outcomes in children with ALL. However, a similar correlation in adults is lack ... Full text Link to item Cite

Improved outcomes with "7+3" induction chemotherapy for acute myeloid leukemia over the past four decades: analysis of SWOG trial data.

Journal Article Haematologica · January 1, 2023 We have previously shown that complete response (CR) rates and overall survival of patients with acute myeloid leukemia have improved since the 1980s. However, we have not previously evaluated how the length of first CR (CR1) has changed over this time per ... Full text Link to item Cite

AML-262 Pivekimab Sunirine (PVEK, IMGN632) Triplet With Azacitidine and Venetoclax Shows Broad Activity in Adverse Genetic Subsets of Relapsed/Refractory Acute Myeloid Leukemia and Reduced Infusion-Related Reactions.

Journal Article Clin Lymphoma Myeloma Leuk · October 2022 CONTEXT: Pivekimab sunirine (PVEK, IMGN632) is a first-in-class ADC comprising a CD123 high-affinity antibody, a cleavable linker, and an IGN (indolinobenzodiazepine pseudodimer) payload. PVEK with azacitidine (AZA) and venetoclax (VEN) is a novel triplet ... Full text Link to item Cite

AML-029 Quizartinib Prolonged Overall Survival (OS) vs Placebo Plus Intensive Induction and Consolidation Therapy Followed by Single-Agent Continuation in Patients Aged 18-75 Years With Newly Diagnosed FLT3-Internal Tandem Duplication Positive (FLT3-ITD+) Acute Myeloid Leukemia (AML).

Journal Article Clin Lymphoma Myeloma Leuk · October 2022 OBJECTIVES: Quizartinib is an oral, potent, selective, type II FLT3 inhibitor with prolonged OS as a single-agent in relapsed/refractory FLT3-ITD+ AML. We report results from the global, randomized, double-blind, phase 3 QuANTUM-First trial (NCT02668653), ... Full text Link to item Cite

AML-251 Overall Survival (OS) With Intensive Chemotherapy (IC) vs Non-IC in Patients With Newly Diagnosed (ND) AML from the Connect® Myeloid Disease Registry Ineligible for Randomized Clinical Trials (RCT).

Journal Article Clin Lymphoma Myeloma Leuk · October 2022 CONTEXT: Patients with acute myeloid leukemia (AML) in RCTs may not reflect the population seen in clinical practice due to strict eligibility criteria. OBJECTIVE: To evaluate outcomes in clinical practice with IC vs venetoclax-containing regimens based on ... Full text Link to item Cite

Disparities in trial enrollment and outcomes of Hispanic adolescent and young adult acute lymphoblastic leukemia.

Journal Article Blood Adv · July 26, 2022 In this secondary analysis of Hispanic adolescents and young adults (AYA) with acute lymphoblastic leukemia (ALL) treated on Cancer and Leukemia Group B (CALGB) 10403, we evaluated outcomes and geographic enrollment patterns relative to US population data. ... Full text Link to item Cite

Long-term outcomes for ibrutinib-rituximab and chemoimmunotherapy in CLL: updated results of the E1912 trial.

Journal Article Blood · July 14, 2022 Herein, we present the long-term follow-up of the randomized E1912 trial comparing the long-term efficacy of ibrutinib-rituximab (IR) therapy to fludarabine, cyclophosphamide, and rituximab (FCR) and describe the tolerability of continuous ibrutinib. The E ... Full text Link to item Cite

Gilteritinib or Chemotherapy for Relapsed or Refractory FLT3 -Mutated AML

Journal Article New England Journal of Medicine · May 12, 2022 Full text Cite

SWOG 1318: A Phase II Trial of Blinatumomab Followed by POMP Maintenance in Older Patients With Newly Diagnosed Philadelphia Chromosome-Negative B-Cell Acute Lymphoblastic Leukemia.

Journal Article J Clin Oncol · May 10, 2022 PURPOSE: Chemotherapy outcomes in older patients with Philadelphia (Ph) chromosome-negative B-acute lymphoblastic leukemia (ALL) are very poor. Here, we evaluated blinatumomab as induction and consolidation therapy followed by prednisone, vincristine, 6-me ... Full text Link to item Cite

Recent Advances in the Treatment and Management of Acute Myeloid Leukemia: Expert Perspectives for Improved Clinical and Economic Outcomes

Journal Article Journal of Managed Care Medicine · January 1, 2022 The past decade has witnessed considerable advances in unraveling molecular and genetic underpinnings of acute myeloid leukemia (AML). With a better understanding of the disease, several targeted therapies have been developed which are enhancing survival i ... Cite

Clinical Manifestations and Treatment of Acute Myeloid Leukemia

Chapter · January 1, 2022 Acute lymphoblastic leukemia (ALL) is a heterogeneous group of diseases characterized by clonal proliferation of lymphoid progenitors (lymphoblasts). Improved diagnostic tools permit accurate and prompt diagnosis, and aid in evaluation of minimal residual ... Full text Cite

Measurable residual disease does not preclude prolonged progression-free survival in CLL treated with ibrutinib.

Journal Article Blood · December 30, 2021 E1912 was a randomized phase 3 trial comparing indefinite ibrutinib plus 6 cycles of rituximab (IR) to 6 cycles of fludarabine, cyclophosphamide, and rituximab (FCR) in untreated younger patients with CLL. We describe measurable residual disease (MRD) leve ... Full text Link to item Cite

Personalized Prediction Model to Risk Stratify Patients With Myelodysplastic Syndromes.

Journal Article J Clin Oncol · November 20, 2021 PURPOSE: Patients with myelodysplastic syndromes (MDS) have a survival that can range from months to decades. Prognostic systems that incorporate advanced analytics of clinical, pathologic, and molecular data have the potential to more accurately and dynam ... Full text Link to item Cite

A Phase 1b/2 Study of the CD123-Targeting Antibody-Drug Conjugate IMGN632 As Monotherapy or in Combination with Venetoclax and Azacitidine for Patients with CD123-Positive Acute Myeloid Leukemia

Conference Blood · November 5, 2021 AbstractBackground: Overexpression of CD123 is characteristic of a number of hematological malignancies, including acute myeloid leukemia (AML) and blastic plasmacytoid dendritic cell neoplasm (BPDCN). IMGN6 ... Full text Cite

Phase 1/2 Study Investigating CC-90011, a Potent, Selective, and Reversible Oral Inhibitor of Lysine-Specific Demethylase 1 (LSD1), Plus Concurrent Venetoclax (VEN) and Azacitidine (AZA) in Patients with Acute Myeloid Leukemia (AML)

Conference Blood · November 5, 2021 AbstractBackgroundAML may be initiated by cytogenetic alterations or mutations in genes encoding epigenetic regulators. Epigenetic dysregulation influences the transformation ... Full text Cite

Quality of Life in Patients &lt;=70 Years of Age with Chronic Lymphocytic Leukemia Treated Frontline with Ibrutinib-Rituximab Versus Fludarabine Cyclophosphamide Rituximab: Analysis from ECOG-ACRIN E1912

Conference Blood · November 5, 2021 AbstractBackground: The ECOG-ACRIN randomized phase 3 clinical trial E1912 established ibrutinib-rituximab (IR) as the standard of care for CLL patients &lt;= 70 years in the frontline setting by improvi ... Full text Cite

Safety and Efficacy from a Phase 1b/2 Study of IMGN632 in Combination with Azacitidine and Venetoclax for Patients with CD123-Positive Acute Myeloid Leukemia

Conference Blood · November 5, 2021 AbstractBACKGROUND: Despite improvements in the treatment of "unfit or older" AML patients with the combination of azacitidine (AZA) and venetoclax (VEN), long-term survival for these patients remains poor. ... Full text Cite

Evorpacept (ALX148), a CD47-Blocking Myeloid Checkpoint Inhibitor, in Combination with Azacitidine: A Phase 1 / 2 Study in Patients with Myelodysplastic Syndrome (ASPEN-02)

Conference Blood · November 5, 2021 AbstractBackground: Evorpacept is a high affinity CD47-blocking fusion protein with an inactive human immunoglobulin Fc region designed to enhance the activity of other anti-neoplas ... Full text Cite

Preliminary Results By Age Group of Treatment with CPX-351 Plus Venetoclax in Adults with Newly Diagnosed AML: Subgroup Analysis of the V-FAST Phase 1b Master Trial

Conference Blood · November 5, 2021 AbstractIntroduction: CPX-351 (United States: Vyxeos ®; Europe: Vyxeos ® Liposomal), a dual-drug liposomal encapsulation of daunorubicin and cytarabine in a synergistic 1:5 molar ratio, is approved for the t ... Full text Cite

Treatment Patterns and Outcomes of Patients with Lower-Risk Myelodysplastic Syndromes in the Connect ® Myeloid Disease Registry

Conference Blood · November 5, 2021 AbstractIntroduction: Many patients (pts) with lower-risk myelodysplastic syndromes (LR-MDS) die without progressing to higher-risk (HR)-MDS or acute myeloid leukemia (AML), and the ... Full text Cite

Treatment Patterns and Survival Outcomes of Patients with Acute Myeloid Leukemia Who Achieved Remission in the Connect ® Myeloid Disease Registry

Conference Blood · November 5, 2021 AbstractIntroduction: Acute myeloid leukemia (AML) is an aggressive myeloid malignancy that predominantly affects older patients (pts; median age at diagnosis 68 years). Despite treatment advances, remission ... Full text Cite

Global Proteomic Profiling Identifies Novel Prognostic Factors in Undifferentiated Leukemia Blasts from Patients with NPM1 Mutations: A Previously Unreported Approach to Biomarker Discovery from the Fred Hutch and SWOG

Conference Blood · November 5, 2021 AbstractAML blasts harbor multiple genomic, epigenomic, and transcriptomic abnormalities. Proteins are responsible for much of the functional biology derived from these transforming events, yet few studies h ... Full text Cite

Proteogenomic Characterization of Highly Enriched Viable Leukemic Blasts in Acute Myeloid Leukemia: A SWOG Report

Conference Blood · November 5, 2021 AbstractAcknowledgements: This work was funded by the following NIH/NCI/NCTN grant awards: RO1CA190661, R01CA160872, U10CA180888, U10CA180819, U24CA196175. The authors wish to gratefully acknowledge the impo ... Full text Cite

Prediction of Early Mortality with Non-Intensive Acute Myeloid Leukemia (AML) Therapies: Analysis of 1336 Patients from MRC/NCRI and SWOG

Conference Blood · November 5, 2021 AbstractBackground: Therapeutic resistance and treatment tolerance vary greatly in patients with AML, likely due to the advanced age and genetic diversity in pharmacokinetics of those affected. Undoubtedly, ... Full text Cite

Randomized, Phase III Study of Early Intervention with Venetoclax and Obinutuzumab Versus Delayed Therapy with Venetoclax and Obinutuzumab in Newly Diagnosed Asymptomatic High-Risk Patients with Chronic Lymphocytic Leukemia/Small Lymphocytic Lymphoma (CLL/SLL): Evolve CLL/SLL Study (SWOG S1925; NCT#04269902)

Conference Blood · November 5, 2021 AbstractBackground: Currently, asymptomatic patients with CLL/SLL are observed without treatment until development of symptoms or cytopenias. Historically, early intervention studies in patients with CLL/SLL ... Full text Cite

Experience with IMGN632, a Novel CD123-Targeting Antibody-Drug Conjugate (ADC), in Frontline Patients with Blastic Plasmacytoid Dendritic Cell Neoplasm (BPDCN)

Conference Blood · November 5, 2021 AbstractBACKGROUND: BPDCN is a rare, aggressive hematologic malignancy characterized by historically poor overall survival and limited therapeutic options. Despite the recent approval of tagraxofusp-erzs for ... Full text Cite

A Study of IMGN632, a Novel CD123-Targeting Antibody-Drug Conjugate, for Patients with Frontline and Relapsed/Refractory Blastic Plasmacytoid Dendritic Cell Neoplasm (BPDCN)

Conference Blood · November 5, 2021 AbstractBackground: Overexpression of CD123 (IL-3Rα receptor) is a hallmark of blastic plasmacytoid dendritic cell neoplasm (BPDCN), thus making this antigen an attractive target for the development of new t ... Full text Cite

Preliminary Results from a Phase Ib Study Exploring MDM2 Inhibitor Siremadlin (HDM201) in Combination with B-Cell Lymphoma-2 (BCL-2) Inhibitor Venetoclax in Patients with Acute Myeloid Leukemia (AML) or High-Risk Myelodysplastic Syndrome (HR-MDS)

Conference Blood · November 5, 2021 AbstractBackground: Treatment options for patients (pts) with relapsed/refractory (r/r) AML and HR-MDS who cannot tolerate standard therapy are limited, and innovative combination approaches are needed. Dysr ... Full text Cite

Long-Term Results of Alliance A041202 Show Continued Advantage of Ibrutinib-Based Regimens Compared with Bendamustine Plus Rituximab (BR) Chemoimmunotherapy

Conference Blood · November 5, 2021 AbstractIntroduction:Alliance for Clinical Trials in Oncology A041202 is a NCI National Clinical Trials Network phase 3 study (NCT01886872) comparing BR (Arm 1) with ibrutini ... Full text Cite

A Phase 2 Study of Dasatinib, Prednisone, and Blinatumomab for Older Patients with Philadelphia-Chromosome (Ph) Positive or Ph-like Acute Lymphoblastic Leukemia (ALL) (with Dasatinib Sensitive Fusions/ Mutations)

Conference Blood · November 5, 2021 AbstractTyrosine kinase inhibitors (TKIs) have improved the outcomes of patients with Philadelphia chromosome positive (Ph+) acute lymphoblastic leukemia (ALL). Many older patients (pts) are not good candida ... Full text Cite

Adverse event burden in older patients with CLL receiving bendamustine plus rituximab or ibrutinib regimens: Alliance A041202.

Journal Article Leukemia · October 2021 Ibrutinib has superior progression-free survival compared with bendamustine plus rituximab (BR) in older CLL patients, however, differences in treatment duration, six monthly BR cycles versus continuous ibrutinib, complicate adverse event (AE) comparisons. ... Full text Link to item Cite

Associations between complete remission and 2- to 3-year survival following 7 + 3 induction for acute myeloid leukemia.

Journal Article Leuk Lymphoma · August 2021 Among acute myeloid leukemia (AML) patients treated with 7 + 3 induction, we evaluate the association between complete morphologic remission (CR) and long-term overall survival (OS) over four decades. We analyzed 1247 patients age ≤65 randomized to 7 + 3 a ... Full text Link to item Cite

Real-world diagnostic testing patterns for assessment of ring sideroblasts and SF3B1 mutations in patients with newly diagnosed lower-risk myelodysplastic syndromes.

Journal Article Int J Lab Hematol · June 2021 INTRODUCTION: The presence of ring sideroblasts (RS) and mutation of the SF3B1 gene are diagnostic of lower-risk (LR) myelodysplastic syndromes (MDS) and are correlated with favorable outcomes. However, information on testing and reporting in community-bas ... Full text Link to item Cite

Randomized, phase III study of early intervention with venetoclax and obinutuzumab versus delayed therapy with venetoclax and obinutuzumab in newly diagnosed asymptomatic high-risk patients with chronic lymphocytic leukemia/small lymphocytic lymphoma (CLL/SLL): EVOLVE CLL/SLL study (SWOG S1925, NCT#04269902).

Conference Journal of Clinical Oncology · May 20, 2021 TPS7567 Background: Currently, asymptomatic patients with CLL/SLL are observed without treatment until development of symptoms or cytopenias. Historically, early intervention studies with chemoimmunotherapy have not resulted in a ... Full text Cite

Preliminary results of V-FAST, a phase 1b master trial to investigate CPX-351 combined with targeted agents in newly diagnosed AML.

Conference Journal of Clinical Oncology · May 20, 2021 7026 Background: CPX-351 (US: Vyxeos; EU: Vyxeos Liposomal), a dual-drug liposomal encapsulation of daunorubicin and cytarabine in a synergistic 1:5 molar drug ratio, is approved by the US FDA and EMA for adults with newly diagno ... Full text Cite

Flotetuzumab as salvage immunotherapy for refractory acute myeloid leukemia.

Journal Article Blood · February 11, 2021 Approximately 50% of acute myeloid leukemia (AML) patients do not respond to induction therapy (primary induction failure [PIF]) or relapse after <6 months (early relapse [ER]). We have recently shown an association between an immune-infiltrated tumor micr ... Full text Link to item Cite

Comparison of CALGB 10403 (Alliance) and COG AALL0232 toxicity results in young adults with acute lymphoblastic leukemia.

Journal Article Blood Adv · January 26, 2021 Adolescents and young adults (AYAs) with acute lymphoblastic leukemia have improved outcomes when treated with pediatric-inspired regimens. CALGB 10403 was the largest prospective study to evaluate the feasibility of using a pediatric regimen in AYAs with ... Full text Link to item Cite

Morphologic Leukemia-Free State in Acute Myeloid Leukemia Is Sufficient for Successful Allogeneic Hematopoietic Stem Cell Transplant

Conference Blood · November 5, 2020 Allogeneic hematopoietic cell transplant (HCT) improves survival in patients with relapsed or high risk acute myeloid leukemia (AML). Complete remission (CR) is typically a pre-requisite for transplantation, though many do not achieve a formal CR. ... Full text Cite

Flotetuzumab As Salvage Therapy for Primary Induction Failure and Early Relapse Acute Myeloid Leukemia

Conference Blood · November 5, 2020 Introduction. Approximately 40% of patients (pts) with newly diagnosed AML either fail to achieve complete remission with intensive induction therapy or experience disease recurrence after a short remission (CR1 &lt;6 months). While these prima ... Full text Cite

A Phase 1b/2 Study of IMGN632, a CD123-Targeting Antibody-Drug Conjugate (ADC), As Monotherapy or in Combination with Venetoclax and/or Azacitidine for Patients with CD123-Positive Acute Myeloid Leukemia

Conference Blood · November 5, 2020 Background: IMGN632 is a CD123-targeting ADC, comprised of a high affinity anti-CD123 antibody coupled to a DNA-alkylating payload of the novel IGN (indolinobenzodiazepine pseudodimer) class. An ongoing Phase I trial in patients with CD123-positive ... Full text Cite

Prophylactic Ruxolitinib for Cytokine Release Syndrome (CRS) in Relapse/Refractory (R/R) AML Patients Treated with Flotetuzumab

Conference Blood · November 5, 2020 Introduction: CRS is a potentially life-threatening toxicity observed following T cell-redirecting therapies. CRS is associated with elevated cytokines, including IL6, IFNγ, TNFα, IL2 and GM-CSF. Glucocorticosteroids (GC) and the IL6 receptor block ... Full text Cite

Characteristics and Prognostic Effects of IDH Mutations across the Age Spectrum in AML: A Collaborative Analysis from COG, SWOG, and ECOG

Conference Blood · November 5, 2020 Background: Somatic mutations in the IDH genes are common in acute myeloid leukemia (AML). Mutations occur at active site arginine residues in IDH1 (R132) and IDH2 (R140, R172). IDH inhibitors, ivosidenib (IDH1) and enasid ... Full text Cite

Incidence and Prognostic Impact of Crh in Acute Myeloid Leukemia

Conference Blood · November 5, 2020 Background: For many years, achievement of complete response (CR, currently blasts &lt; 5% blasts by morphology, ANC &gt; 1,000, platelets &gt; 100,000) was the initial goal in acute myeloid leukemia (AML). However, disease recurred in ... Full text Cite

V-FAST: A Phase 1b Master Trial to Investigate CPX-351 Combined with Various Targeted Agents in Patients with Previously Untreated Acute Myeloid Leukemia

Conference Blood · November 5, 2020 Introduction: CPX-351 (Vyxeos®; daunorubicin and cytarabine liposome for injection), a dual-drug liposomal encapsulation of daunorubicin and cytarabine in a synergistic 1:5 molar drug ratio, has been approved by the US FDA and EMA for the treatment ... Full text Cite

Clinical Profile of IMGN632, a Novel CD123-Targeting Antibody-Drug Conjugate (ADC), in Patients with Relapsed/Refractory (R/R) Blastic Plasmacytoid Dendritic Cell Neoplasm (BPDCN)

Conference Blood · November 5, 2020 BACKGROUND: BPDCN is a rare, aggressive hematologic malignancy characterized by poor overall survival and limited therapeutic options. Overexpression of CD123 (IL-3Rα) is present in all BPDCN cases, therefore establishing this marker as a rational ... Full text Cite

Immune Senescence and Exhaustion Correlate with Response to Flotetuzumab, an Investigational CD123×CD3 Bispecific Dart® Molecule, in Acute Myeloid Leukemia

Conference Blood · November 5, 2020 We have recently shown that bone marrow (BM) RNA profiles stratify patients with acute myeloid leukemia (AML) into immune-infiltrated and immune-depleted subtypes and that type I/II interferon (IFN)-related gene signatures associate with complete r ... Full text Cite

Post-Remission Treatment (Tx) Patterns for Patients (Pts) with Newly Diagnosed (ND) Acute Myeloid Leukemia (AML) in the Connect® MDS/AML Disease Registry

Conference Blood · November 5, 2020 Introduction: Outcomes among pts with AML have improved over the last 20 years with the introduction of novel, targeted, and reformulated chemotherapy agents, as well as the optimization of allogeneic hematopoietic stem cell transplantation (HSCT) ... Full text Cite

Diagnosis and Treatment of Patients With Acute Myeloid Leukemia With Myelodysplasia-Related Changes (AML-MRC).

Journal Article Am J Clin Pathol · November 4, 2020 OBJECTIVES: Acute myeloid leukemia (AML) with myelodysplasia-related changes (AML-MRC) represents a high-risk and somewhat diverse subtype of AML, and substantial confusion exists about the pathologic evaluation needed for diagnosis, which can include the ... Full text Link to item Cite

Outcomes of high-risk acute promyelocytic leukemia patients treated with arsenic trioxide (ATO)/all trans retinoic acid (ATRA) based induction and consolidation without maintenance phase: A case Series.

Journal Article Hematol Oncol Stem Cell Ther · September 2020 Patients with high-risk acute promyelocytic leukemia (APL) have inferior outcomes compared with patients with low-risk APL, predominantly due to higher risk of early mortality related to hemorrhage. The majority of regimens contain prolonged maintenance, b ... Full text Link to item Cite

American Society of Hematology 2020 guidelines for treating newly diagnosed acute myeloid leukemia in older adults.

Journal Article Blood Adv · August 11, 2020 BACKGROUND: Older adults with acute myeloid leukemia (AML) represent a vulnerable population in whom disease-based and clinical risk factors, patient goals, prognosis, and practitioner- and patient-perceived treatment risks and benefits influence treatment ... Full text Link to item Cite

Special considerations in the management of adult patients with acute leukaemias and myeloid neoplasms in the COVID-19 era: recommendations from a panel of international experts.

Journal Article Lancet Haematol · August 2020 The ongoing COVID-19 pandemic caused by severe acute respiratory syndrome coronavirus 2 is a global public health crisis. Multiple observations indicate poorer post-infection outcomes for patients with cancer than for the general population. Herein, we hig ... Full text Link to item Cite

Bias in Mean Survival From Fitting Cure Models With Limited Follow-Up.

Journal Article Value Health · August 2020 OBJECTIVES: When populations contain mixtures of cured and uncured patients, the use of traditional parametric approaches to estimate overall survival (OS) can be biased. Mixture cure models may reduce bias compared with traditional parametric models, but ... Full text Link to item Cite

Diagnostic and molecular testing patterns in patients with newly diagnosed acute myeloid leukemia in the Connect® MDS/AML Disease Registry.

Journal Article EJHaem · July 2020 Diagnostic and molecular genetic testing are key in advancing the treatment of acute myeloid leukemia (AML), yet little is known about testing patterns outside of clinical trials, especially in older patients. We analyzed diagnostic and molecular testing p ... Full text Link to item Cite

Rapid Donor Identification Improves Survival in High-Risk First-Remission Patients With Acute Myeloid Leukemia.

Journal Article JCO Oncol Pract · June 2020 PURPOSE: Patients with acute myeloid leukemia with high-risk cytogenetics in first complete remission (CR1) achieve better outcomes if they undergo allogeneic hematopoietic cell transplantation (HCT) compared with consolidation chemotherapy alone. However, ... Full text Link to item Cite

Microtransplantation in older patients with AML: A pilot study of safety, efficacy and immunologic effects.

Journal Article Am J Hematol · June 2020 Older AML patients have low remission rates and poor survival outcomes with standard chemotherapy. Microtransplantation (MST) refers to infusion of allogeneic hematopoietic stem cells without substantial engraftment. MST has been shown to improve clinical ... Full text Link to item Cite

A phase 2 study of ATRA, arsenic trioxide, and gemtuzumab ozogamicin in patients with high-risk APL (SWOG 0535).

Journal Article Blood Adv · April 28, 2020 High-risk acute promyelocytic leukemia (APL) remains a therapeutic challenge, with higher associated rates of early mortality and relapse than standard-risk APL. All-trans retinoic acid (ATRA) plus arsenic trioxide (ATO) is a well-established treatment for ... Full text Link to item Cite

Recently approved molecularly targeted therapies in AML

Journal Article ADVANCES IN CELL AND GENE THERAPY · April 2020 Full text Cite

Ivosidenib induces deep durable remissions in patients with newly diagnosed IDH1-mutant acute myeloid leukemia.

Journal Article Blood · February 13, 2020 Ivosidenib (AG-120) is an oral, targeted agent that suppresses production of the oncometabolite 2-hydroxyglutarate via inhibition of the mutant isocitrate dehydrogenase 1 (IDH1; mIDH1) enzyme. From a phase 1 study of 258 patients with IDH1-mutant hematolog ... Full text Link to item Cite

Lenalidomide Plus Hypomethylating Agent as a Treatment Option in Acute Myeloid Leukemia With Recurrent Genetic Abnormalities-AML With inv(3)(q21.3q26.2) or t(3;3)(q21.3;q26.2); GATA2, MECOM.

Journal Article Clin Lymphoma Myeloma Leuk · January 2020 INTRODUCTION: Acute myeloid leukemia (AML) is a heterogeneous clonal hematopoietic neoplasm. The cytogenetic changes associated with AML affect the response rate and survival and are one of the most important independent prognostic factors. AML with inv(3) ... Full text Link to item Cite

AML risk stratification models utilizing ELN-2017 guidelines and additional prognostic factors: a SWOG report.

Journal Article Biomark Res · 2020 BACKGROUND: The recently updated European LeukemiaNet risk stratification guidelines combine cytogenetic abnormalities and genetic mutations to provide the means to triage patients with acute myeloid leukemia for optimal therapies. Despite the identificati ... Full text Link to item Cite

Ibrutinib and Rituximab Provides Superior Clinical Outcome Compared to FCR in Younger Patients with Chronic Lymphocytic Leukemia (CLL): Extended Follow-up from the E1912 Trial.

Journal Article Blood · November 13, 2019 DISCLOSURES: Shanafelt: Pharmacyclics: Research Funding; Patent: Patents & Royalties: US14/292,075 on green tea extract epigallocatechin gallate in combination with chemotherapy for chronic lymphocytic leukemia; Polyphenon E International: Research Funding ... Full text Link to item Cite

Increasing Lengths of First Complete Remission with 7+3 Induction Chemotherapy for Acute Myeloid Leukemia over the Past Four Decades: Analysis of SWOG Trial Data

Conference Blood · November 13, 2019 Background: We have previously shown that the survival of patients with AML who fail to achieve complete remission (CR) with 7+3 has improved since the 1980s. However, although CR rates with 7+3 have improved over the last four decades, we have not ... Full text Cite

Clinical Profile of IMGN632, a Novel CD123-Targeting Antibody-Drug Conjugate (ADC), in Patients with Relapsed/Refractory (R/R) Acute Myeloid Leukemia (AML) or Blastic Plasmacytoid Dendritic Cell Neoplasm (BPDCN)

Conference Blood · November 13, 2019 INTRODUCTION: Overexpression of CD123, the alpha subunit of the IL-3 receptor, is seen in multiple hematological malignancies, including AML, BPDCN, and ALL. IMGN632 is a CD123-targeting ADC, comprising a high affinity anti-CD123 antibody coupled t ... Full text Cite

SWOG 1312 Final Results: A Phase 1 Trial of Inotuzumab in Combination with CVP (Cyclophosphamide, Vincristine, Prednisone) for Relapsed/ Refractory CD22+ Acute Leukemia

Conference Blood · November 13, 2019 The prognosis of patients (pts) with relapsed/ refractory acute lymphoblastic leukemia (ALL) remains poor and novel therapies are needed. The anti-CD22 immunoconjugate inotuzumab ozogamicin (IO) has demonstrated promising ... Full text Cite

Incidence of Invasive Fungal Infections in Acute Leukemia Patients Utilizing Micafungin Prophylaxis Compared to Second-Generation Azole Prophylaxis

Conference Blood · November 13, 2019 Introduction/Background:Standard therapy for both acute myeloid leukemia (AML) and acute lymphoblastic leukemia (ALL) is myelosuppressive, and patients are expected to be neutropenic for a prolonged period. Due to t ... Full text Cite

Ramp-up Treatment Strategy in Philadelphia Positive Acute Lymphoblastic Leukemia Is Associated with Deep Molecular Remissions and Favorable Outcomes

Conference Blood · November 13, 2019 Introduction: The advent of Tyrosine Kinase Inhibitors (TKI) changed the treatment paradigm of Philadelphia chromosome Positive (Ph+) Acute Lymphoblastic Leukemia (ALL). Different treatment strategies exist including combi ... Full text Cite

Accrual Barriers and Detection of Early Toxicity Signal in Older Less-Fit Patients Treated with Azacitidine and Nivolumab for Newly Diagnosed Acute Myeloid Leukemia (AML) or High-Risk Myelodysplastic Syndrome (MDS) in the SWOG 1612 Platform Randomized Phase II/III Clinical Trial

Conference Blood · November 13, 2019 Background: Less-fit patients with acute myeloid leukemia (AML) or high-risk myelodysplasia (MDS) age 60 years and older constitute the majority of patients with AML/MDS but are not well represented in clinical trials. DNA ... Full text Cite

Real World Use of IDH2- Targeted Inhibitors in a Single Academic Medical Center Experience Since Enasidenib FDA-Approval

Conference Blood · November 13, 2019 BackgroundEnasidenib, a novel inhibitor of isocitrate dehydrogenase 2 (IDH2), offers targeted therapy to the ~12% of acute myeloid leukemia (AML) patients in which a recurrent IDH2 mutation exists. While currently o ... Full text Cite

Outcomes in Patients with Therapy-Related Acute Myeloid Leukemia (t-AML) Who Achieved Remission with CPX-351 Versus 7+3: Phase 3 Exploratory Analysis

Conference Blood · November 13, 2019 Introduction: t-AML is generally defined as AML arising from prior cytotoxic therapy, ionizing radiotherapy, or immunotherapy for an unrelated disease and is associated with poorer outcomes relative to de novo AML, with a ... Full text Cite

A Double-Blind, Placebo-Controlled, Phase 3 Registration Trial to Evaluate the Efficacy of Uproleselan (GMI-1271) with Standard Salvage Chemotherapy in Patients with Relapsed/Refractory (R/R) Acute Myeloid Leukemia

Conference Blood · November 13, 2019 BackgroundBinding of E-selectin (E-sel) to sialyl Lex, the E-sel ligand, on the leukemic cell surface activates cell survival pathways and promotes chemotherapy resistance in AML. Higher ex ... Full text Cite

Development and Performance of Risk Stratification Models for AML Patients Utilizing ELN-2017 Guidelines and Additional Prognostic Factors: A SWOG Report

Conference Blood · November 13, 2019 ABSTRACT:INTRODUCTION: ELN-2017 guideline is the gold standard for risk stratifying AML patients. However, clinical prognostic factors such as age are not incorporated into the guideline. Therefore, we examined if n ... Full text Cite

A Phase 1b/2 Study of the CD123-Targeting Antibody-Drug Conjugate IMGN632 As Monotherapy or in Combination with Venetoclax and/or Azacitidine for Patients with CD123-Positive Acute Myeloid Leukemia

Conference Blood · November 13, 2019 Background: Overexpression of CD123, the alpha subunit of the IL-3 receptor, is characteristic of a number of hematological malignancies, including acute myeloid leukemia (AML), thus providing an attractive candidate for targeted therapeutic approa ... Full text Cite

Allogeneic hematopoietic cell transplantation compared to chemotherapy consolidation in older acute myeloid leukemia (AML) patients 60-75 years in first complete remission (CR1): an alliance (A151509), SWOG, ECOG-ACRIN, and CIBMTR study.

Journal Article Leukemia · November 2019 The preferred post-remission therapy for older patients with acute myeloid leukemia (AML) in first complete remission (CR1) remains uncertain. In this retrospective, multicenter study, we compared the outcomes for older AML patients (age 60-77 years) recei ... Full text Link to item Cite

Gilteritinib or Chemotherapy for Relapsed or Refractory FLT3-Mutated AML.

Journal Article N Engl J Med · October 31, 2019 BACKGROUND: Patients with relapsed or refractory acute myeloid leukemia (AML) with mutations in the FMS-like tyrosine kinase 3 gene (FLT3) infrequently have a response to salvage chemotherapy. Gilteritinib is an oral, potent, selective FLT3 inhibitor with ... Full text Link to item Cite

Ibrutinib-Rituximab or Chemoimmunotherapy for Chronic Lymphocytic Leukemia.

Journal Article N Engl J Med · August 1, 2019 BACKGROUND: Data regarding the efficacy of treatment with ibrutinib-rituximab, as compared with standard chemoimmunotherapy with fludarabine, cyclophosphamide, and rituximab, in patients with previously untreated chronic lymphocytic leukemia (CLL) have bee ... Full text Link to item Cite

Phase 1b study of the MDM2 inhibitor AMG 232 with or without trametinib in relapsed/refractory acute myeloid leukemia.

Journal Article Blood Adv · July 9, 2019 This open-label, phase 1 study evaluated the safety, pharmacokinetics, and maximum tolerated dose of AMG 232, an investigational oral, selective mouse double minute 2 homolog inhibitor in relapsed/refractory acute myeloid leukemia (AML). AMG 232 was admini ... Full text Link to item Cite

A phase III trial to evaluate the efficacy of uproleselan (GMI-1271) with chemotherapy in patients with relapsed/refractory acute myeloid leukemia.

Conference Journal of Clinical Oncology · May 20, 2019 TPS7066 Background: Binding of E-selectin (E-sel) to sialyl Lex, the E-sel ligand (E-sel-L), on the leukemic cell surface activates cell survival pathways and promotes chemotherapy resistance in AML. Expressi ... Full text Cite

A pediatric regimen for older adolescents and young adults with acute lymphoblastic leukemia: results of CALGB 10403.

Journal Article Blood · April 4, 2019 Retrospective studies have suggested that older adolescents and young adults (AYAs) with acute lymphoblastic leukemia (ALL) have better survival rates when treated using a pediatric ALL regimen administered by pediatric treatment teams. To address the feas ... Full text Link to item Cite

Shifting paradigms in the treatment of older adults with AML.

Journal Article Semin Hematol · April 2019 The treatment of acute myeloid leukemia has been associated with dismal outcomes despite available therapies. The morbidity and mortality associated with acute myeloid leukemia (AML) are most profound in older patients who account for the majority of cases ... Full text Link to item Cite

The relationship between clinical trial accrual volume and outcomes in acute myeloid leukemia: A SWOG/ECOG-ACRIN study (S0106 and E1900).

Journal Article Leuk Res · March 2019 PURPOSE: To study whether institutional clinical trial accrual volume affects clinical outcomes of younger (age less than 61 years) patients with acute myeloid leukemia. PATIENTS AND METHODS: We investigated the impact of clinical trial accrual on response ... Full text Link to item Cite

Relative survival following response to 7 + 3 versus azacytidine is similar in acute myeloid leukemia and high-risk myelodysplastic syndromes: an analysis of four SWOG studies.

Journal Article Leukemia · February 2019 Here we quantify and compare the absolute and relative overall survival (OS) benefits conveyed by complete remission (CR) in AML and high-risk MDS, and by CR with incomplete count recovery (CRi) in AML and by hematologic improvement (HI) in MDS, following ... Full text Open Access Link to item Cite

Genomic Biomarkers to Predict Resistance to Hypomethylating Agents in Patients With Myelodysplastic Syndromes Using Artificial Intelligence.

Journal Article JCO Precis Oncol · 2019 PURPOSE: We developed an unbiased framework to study the association of several mutations in predicting resistance to hypomethylating agents (HMAs) in patients with myelodysplastic syndromes (MDS), analogous to consumer and commercial recommender systems i ... Full text Open Access Link to item Cite

Ibrutinib Regimens versus Chemoimmunotherapy in Older Patients with Untreated CLL.

Journal Article N Engl J Med · December 27, 2018 BACKGROUND: Ibrutinib has been approved by the Food and Drug Administration for the treatment of patients with untreated chronic lymphocytic leukemia (CLL) since 2016 but has not been compared with chemoimmunotherapy. We conducted a phase 3 trial to evalua ... Full text Link to item Cite

2nd cycle Remission Achievement with 7+3 Is Associated with Shorter Survival in Adults with Newly Diagnosed Acute Myeloid Leukemia: Analysis of Recent SWOG Trials

Conference Blood · November 29, 2018 AbstractBackground: Intensive chemotherapy will induce a complete morphologic remission (CR) in many adults with acute myeloid leukemia (AML). Whether it matters that a remission is obtained early, i.e. with ... Full text Cite

Lenalidomide Plus Hypomethylating Agent for Acute Myeloid Leukemia (AML) with Recurrent Genetic Abnormalities -AML with Inv(3)(q21.3q26.2) or t(3;3)(q21.3;q26.2); GATA2, Mecom

Conference Blood · November 29, 2018 AbstractIntro: Acute myeloid leukemia (AML) is a heterogeneous clonal hematopoietic neoplasm. The cytogenetic changes associated with AML impact response rate and survival, and is one of the most important i ... Full text Cite

Omission of Maintenance in Patients with High-Risk Acute Promyelocytic Leukemia (APL) in the Era of ATRA/Arsenic Consolidation

Conference Blood · November 29, 2018 AbstractIntroduction: The role of maintenance treatment in patients with high-risk Acute Promyelocytic Leukemia (APL) in the era of ATO/ATRA is unclear.Methods: We retrospect ... Full text Cite

Healthcare Resource Utilization (HCRU) in Patients (pts) with Newly Diagnosed (ND) Acute Myeloid Leukemia (AML) Treated in the Connect® MDS/AML Disease Registry

Conference Blood · November 29, 2018 AbstractIntroduction: A number of treatment (Tx) options are available for ND AML pts including high-intensity (HI) chemotherapy ("7+3" regimen) followed by consolidation chemotherapy or allogeneic hematopoi ... Full text Cite

Ibrutinib Alone or in Combination with Rituximab Produces Superior Progression Free Survival (PFS) Compared with Bendamustine Plus Rituximab in Untreated Older Patients with Chronic Lymphocytic Leukemia (CLL): Results of Alliance North American Intergroup Study A041202

Conference Blood · November 29, 2018 AbstractIntroduction:The most effective initial therapy for older adults with CLL has not yet been established due to the lack of comparison of chem ... Full text Cite

Allogeneic Hematopoietic Cell Transplantation (HCT) Vs. Non-HCT Consolidation Therapies in Acute Myeloid Leukemia (AML) Patients 60-75 Years of Age in First Complete Remission (CR1): An Alliance (A151509), SWOG, ECOG-ACRIN and CIBMTR Study

Conference Blood · November 29, 2018 AbstractIntroduction: The preferred post-remission therapy for older patients (pts) with AML remains uncertain. We compared outcomes for older AML pts in CR1 receiving HCT reported to the CIBMTR to older AML ... Full text Cite

Results of SWOG 1318: A Phase 2 Trial of Blinatumomab Followed By Pomp (Prednisone, Vincristine, Methotrexate, 6-Mercaptopurine) Maintenance in Elderly Patients with Newly Diagnosed Philadelphia Chromosome Negative B-Cell Acute Lymphoblastic Leukemia

Conference Blood · November 29, 2018 AbstractThe prognosis of elderly patients (pts) with acute lymphoblastic leukemia (ALL) remains poor, and novel therapeutic approaches are clearly needed. CD19 is expressed on the majority of precursor-B ALL ... Full text Cite

Predicting Induction Toxicity with 7+3: Analysis of SWOG Trial S1203

Conference Blood · November 29, 2018 AbstractBackground: Prior work has shown that multivariable models can have reasonably high accuracy in predicting early deaths (deaths within 28 days of starting induction, defined as treatment-related mort ... Full text Cite

Ivosidenib (AG-120) Induced Durable Remissions and Transfusion Independence in Patients with IDH1-Mutant Untreated AML: Results from a Phase 1 Dose Escalation and Expansion Study

Conference Blood · November 29, 2018 AbstractBACKGROUND: Isocitrate dehydrogenase 1 (IDH1) mutations are seen in 6-10% of patients with acute myeloid leukemia (AML). Ivosidenib (AG-120), an oral, potent, targeted inhibitor of the mutant IDH1 pr ... Full text Cite

A Randomized Phase III Study of Ibrutinib (PCI-32765)-Based Therapy Vs. Standard Fludarabine, Cyclophosphamide, and Rituximab (FCR) Chemoimmunotherapy in Untreated Younger Patients with Chronic Lymphocytic Leukemia (CLL): A Trial of the ECOG-ACRIN Cancer Research Group (E1912)

Conference Blood · November 29, 2018 AbstractBACKGROUND: Chronic lymphocytic leukemia (CLL) is one of the most common lymphoid malignancies, accounting for ~11% of all hematologic neoplasms. Over the last 15 years, a series of phase 3 trials ha ... Full text Cite

A Phase I, First-in-Human Study Evaluating the Safety and Preliminary Antileukemia Activity of IMGN632, a Novel CD123-Targeting Antibody-Drug Conjugate, in Patients with Relapsed/Refractory Acute Myeloid Leukemia and Other CD123-Positive Hematologic Malignancies

Conference Blood · November 29, 2018 AbstractINTRODUCTION: Overexpression of CD123, the alpha subunit of the IL-3 receptor, in multiple hematological malignancies, including acute myeloid leukemia (AML), acute lymphoblastic leukemia (ALL), and ... Full text Cite

The Impact of Cumulative Dose of Cytarabine Consolidation on Outcomes in Older Patients with Acute Myeloid Leukemia

Conference Blood · November 29, 2018 AbstractIntroduction: The optimum number of cycles and dose of Cytarabine consolidation in older patients (age 60 and above) with AML in first remission remains unclear.Metho ... Full text Cite

Outcomes of Acute Myeloid Leukemia Patients with Indeterminate Day 14 Bone Marrow Results Treated with and without Re-Induction Chemotherapy

Conference Blood · November 29, 2018 AbstractIntroduction: The initial response to standard induction chemotherapy for acute myeloid leukemia (AML) is commonly assessed with day 14 bone marrow evaluation; patients with residual disease may rece ... Full text Cite

Maturing Clinical Profile of IMGN779, a Next-Generation CD33-Targeting Antibody-Drug Conjugate, in Patients with Relapsed or Refractory Acute Myeloid Leukemia

Conference Blood · November 29, 2018 AbstractINTRODUCTION: The myeloid differentiation antigen CD33 is an established therapeutic target in acute myeloid leukemia (AML). In addition to its broad expression on leukemic cells of most patients wit ... Full text Cite

Phase 1/2 trial of GCLAM with dose-escalated mitoxantrone for newly diagnosed AML or other high-grade myeloid neoplasms.

Journal Article Leukemia · November 2018 Outcomes with "7 + 3" are often unsatisfactory in acute myeloid leukemia (AML). Trials demonstrating improved outcomes with high-dose cytarabine, addition of cladribine, or escalated anthracycline doses prompted a phase 1/2 study (NCT02044796) of G-CSF, cl ... Full text Open Access Link to item Cite

Outcomes of fludarabine, high dose cytarabine and granulocyte-colony stimulating factor (FLAG) as re-induction for residual acute myeloid leukemia on day 14 bone marrow.

Journal Article Leuk Res · November 2018 BACKGROUND: Patients with acute myeloid leukemia (AML) treated with intensive chemotherapy may require re-induction based on the evaluation of day 14 bone marrow biopsy. METHODS: A retrospective chart review was performed to evaluate adult patients with AM ... Full text Link to item Cite

A phase 1 trial of vadastuximab talirine combined with hypomethylating agents in patients with CD33-positive AML.

Journal Article Blood · September 13, 2018 Treatment of acute myeloid leukemia (AML) among the elderly is challenging because of intolerance of intensive therapy and therapy-resistant biology. Hypomethylating agents (HMAs) are commonly used, with suboptimal outcomes. Vadastuximab talirine is a CD33 ... Full text Link to item Cite

Durable Remissions with Ivosidenib in IDH1-Mutated Relapsed or Refractory AML.

Journal Article N Engl J Med · June 21, 2018 BACKGROUND: Mutations in the gene encoding isocitrate dehydrogenase 1 ( IDH1) occur in 6 to 10% of patients with acute myeloid leukemia (AML). Ivosidenib (AG-120) is an oral, targeted, small-molecule inhibitor of mutant IDH1. METHODS: We conducted a phase ... Full text Link to item Cite

Comparable outcomes of patients eligible vs ineligible for SWOG leukemia studies.

Journal Article Blood · June 21, 2018 Patients may be deemed ineligible for a clinical trial for reasons that do not directly impact efficacy or safety. We identified reasons for ineligibility and compared outcomes of ineligible with eligible patients treated on Southwest Oncology Group (SWOG) ... Full text Link to item Cite

Report of the relapsed/refractory cohort of SWOG S0919: A phase 2 study of idarubicin and cytarabine in combination with pravastatin for acute myelogenous leukemia (AML).

Journal Article Leuk Res · April 2018 Inhibition of cholesterol synthesis and uptake sensitizes acute myeloid leukemia (AML) blasts to chemotherapy. A Phase 2 study of high dose pravastatin given in combination with idarubicin and cytarabine demonstrated an impressive response rate [75% comple ... Full text Link to item Cite

Pevonedistat, a first-in-class NEDD8-activating enzyme inhibitor, combined with azacitidine in patients with AML.

Journal Article Blood · March 29, 2018 Pevonedistat (TAK-924/MLN4924) is a novel inhibitor of NEDD8-activating enzyme (NAE) with single-agent activity in relapsed/refractory acute myeloid leukemia (AML). We performed a phase 1b study of pevonedistat (PEV) with azacitidine (AZA) based on synergi ... Full text Link to item Cite

Impact of Specimen Heterogeneity on Biomarkers in Repository Samples from Patients with Acute Myeloid Leukemia: A SWOG Report.

Journal Article Biopreserv Biobank · February 2018 INTRODUCTION: Current prognostic models for acute myeloid leukemia (AML) are inconsistent at predicting clinical outcomes for individual patients. Variability in the quality of specimens utilized for biomarker discovery and validation may contribute to thi ... Full text Link to item Cite

A phase 1 trial of vadastuximab talirine as monotherapy in patients with CD33-positive acute myeloid leukemia.

Journal Article Blood · January 25, 2018 Vadastuximab talirine (SGN-CD33A, 33A) is an antibody-drug conjugate consisting of pyrrolobenzodiazepine dimers linked to a monoclonal antibody targeting CD33, which is expressed in the majority of acute myeloid leukemia (AML) patients. This phase 1 study ... Full text Link to item Cite

Randomized Phase II Study of Azacitidine Alone or in Combination With Lenalidomide or With Vorinostat in Higher-Risk Myelodysplastic Syndromes and Chronic Myelomonocytic Leukemia: North American Intergroup Study SWOG S1117.

Conference J Clin Oncol · August 20, 2017 Purpose Azacitidine is standard, first-line therapy in higher-risk myelodysplastic syndromes (MDS). Whether azacitidine-based combinations with lenalidomide or vorinostat produce superior overall response rates (ORRs) to azacitidine is not known. Patients ... Full text Link to item Cite

Selective inhibition of FLT3 by gilteritinib in relapsed or refractory acute myeloid leukaemia: a multicentre, first-in-human, open-label, phase 1-2 study.

Journal Article Lancet Oncol · August 2017 BACKGROUND: Internal tandem duplication mutations in FLT3 are common in acute myeloid leukaemia and are associated with rapid relapse and short overall survival. The clinical benefit of FLT3 inhibitors in patients with acute myeloid leukaemia has been limi ... Full text Open Access Link to item Cite

Prognostic methylation markers for overall survival in cytogenetically normal patients with acute myeloid leukemia treated on SWOG trials.

Journal Article Cancer · July 1, 2017 BACKGROUND: Aberrant DNA methylation is known to occur in patients with acute myeloid leukemia (AML), whereas methylation signatures and prognostic markers have been proposed. The objective of the current study was to evaluate all CpG sites of the genome a ... Full text Link to item Cite

Molecular genetic testing patterns for patients with newly diagnosed acute myeloid leukemia (AML) enrolled in the CONNECT MDS/AML disease registry.

Conference Journal of Clinical Oncology · May 20, 2017 7022 Background: Recurrent mutations in AML-associated genes have prognostic value and may help guide treatment decisions. Molecular genetic testing patterns for AML in clinical practice are largely unknown. Previously the CONNEC ... Full text Cite

Overall survival (OS) with CPX-351 versus 7+3 in older adults with newly diagnosed, therapy-related acute myeloid leukemia (tAML): Subgroup analysis of a phase III study.

Conference Journal of Clinical Oncology · May 20, 2017 7035 Background: tAML may occur as a late complication of cytotoxic therapy and is associated with a poor prognosis. CPX-351 is a liposomal formulation that delivers a synergistic 5:1 molar ratio of cytarabine (C) and daunorubici ... Full text Cite

Overall survival (OS) and stem cell transplant (SCT) in patients with FLT3 mutations treated with CPX-351 versus 7+3: Subgroup analysis of a phase III study of older adults with newly diagnosed, high-risk acute myeloid leukemia (AML).

Conference Journal of Clinical Oncology · May 20, 2017 e18507 Background: FLT3+ AML patients (pts; 20%-30% of AML pts) often have rapid post-induction relapse, highlighting the need for therapies that improve the bridge to SCT. CPX-351 is a liposomal formulation that delivers a syner ... Full text Cite

Dose escalation results of a phase 1b study of the MDM2 inhibitor AMG 232 with or without trametinib in patients (Pts) with relapsed/refractory (r/r) acute myeloid leukemia (AML).

Conference Journal of Clinical Oncology · May 20, 2017 7027 Background: The ubiquitin ligase MDM2 inhibits the tumor suppressor p53. In preclinical AML models, MDM2 inhibitors have antitumor activity as monotherapy that is synergistic when combined with MEK inhibitors. This open-labe ... Full text Cite

Determinants of fatal bleeding during induction therapy for acute promyelocytic leukemia in the ATRA era.

Journal Article Blood · March 30, 2017 Acute promyelocytic leukemia (APL) is commonly complicated by a complex coagulopathy. Uncertainty remains as to which markers of bleeding risk are independent predictors. Drawing from 5 large clinical trials that included all-trans retinoic acid (ATRA) as ... Full text Link to item Cite

Downward Platelet Utilization Trends in Acute Leukemia

Conference American Journal of Clinical Pathology · March 1, 2017 Full text Cite

Wide variations in blood product transfusion practices among providers who care for patients with acute leukemia in the United States.

Journal Article Transfusion · February 2017 BACKGROUND: Transfusion of blood products is a key component of the supportive management in patients with acute leukemia (AL). However high-quality trial evidence and clinical outcome data to support specific transfusion goals for blood products for patie ... Full text Link to item Cite

Seven-year follow-up for energy/vitality outcomes in early stage Hodgkin's disease patients treated with subtotal lymphoid irradiation versus chemotherapy plus radiation: SWOG S9133 and its QOL companion study, S9208.

Journal Article J Cancer Surviv · February 2017 PURPOSE: We describe 7 years of follow-up for the energy/vitality outcome in early-stage Hodgkin's disease patients treated on a randomized clinical trial that compared subtotal lymphoid irradiation (STLI) with combined modality treatment (CMT) (SWOG 9133) ... Full text Link to item Cite

US Intergroup Study of Chemotherapy Plus Dasatinib and Allogeneic Stem Cell Transplant in Philadelphia Chromosome Positive ALL.

Journal Article Blood Adv · December 27, 2016 This multicenter trial was conducted to determine whether the addition of dasatinib to chemotherapy followed by an allogeneic hematopoietic cell transplant (HCT) in patients with Philadelphia chromosome positive (Ph+) acute lymphoblastic leukemia (ALL) was ... Full text Link to item Cite

ENESTgoal Treatment-Free Remission Study: Updated Preliminary Results and Digital Polymerase Chain Reaction Analysis in Patients with Chronic Myeloid Leukemia in Chronic Phase Who Switched from Imatinib to Nilotinib

Conference Blood · December 2, 2016 AbstractBackground: Treatment-free remission (TFR; ie, stopping tyrosine kinase inhibitor [TKI] therapy without loss of response) has been demonstrated in multiple trials in patients (pts) with chronic myelo ... Full text Cite

A Phase 1 Trial of Inotuzumab in Combination with CVP (Cyclophosphamide, Vincristine, Prednisone) for Relapsed/ Refractory CD22+ Acute Leukemia (SWOG 1312)

Conference Blood · December 2, 2016 AbstractThe prognosis of patients (pts) with relapsed/ refractory acute lymphoblastic leukemia (ALL) remains poor and novel therapies are needed. The anti-CD22 immunoconjugate inotuzumab ozogamicin (INO) has ... Full text Cite

Improved Prognostic Significance of Genomic and Transcriptional Biomarkers By Examining Enriched Populations of AML Blasts: A SWOG Report

Conference Blood · December 2, 2016 AbstractINTRODUCTION. Most studies of AML biomarkers have examined cryopreserved mononuclear cells (MNCs) obtained from repositories. These MNCs typically include non-leukemic cells (e.g., lymphocytes) and h ... Full text Cite

Current Diagnosis Patterns for Acute Myeloid Leukemia (AML) in Clinical Practice Compared with World Health Organization (WHO) 2008 Recommendations: Outcomes from the CONNECT® Myelodysplastic Syndromes (MDS) and AML Disease Registry

Conference Blood · December 2, 2016 AbstractBackground: The diagnosis of AML requires an integrated approach of clinical, morphologic, cytogenetic, and molecular assessments. The CONNECT MDS/AML Disease Registry (NCT01688011) is a prospective, ... Full text Cite

SWOG S1203: A Randomized Phase III Study of Standard Cytarabine Plus Daunorubicin (7+3) Therapy Versus Idarubicin with High Dose Cytarabine (IA) with or without Vorinostat (IA+V) in Younger Patients with Previously Untreated Acute Myeloid Leukemia (AML)

Conference Blood · December 2, 2016 AbstractBackground: A majority of younger pts with AML receive initial therapy with a 7+3 scheme. Escalated doses of daunorubicin (dauno) in combination with standard doses of ara-C ... Full text Cite

Comparable Outcomes of Patients Eligible Versus Ineligible for Southwest Oncology Group (SWOG) Leukemia Studies

Conference Blood · December 2, 2016 AbstractBackground: Clinical trial eligibility criteria balance identifying a specific patient population who may benefit from an intervention with restricting allowable comorbidities to maximize safety and ... Full text Cite

Complete Remissions (CRs) with Azacitidine Regimens Compared to Crs with 7+3 Induction Chemotherapy and the Effect on Overall Survival

Conference Blood · December 2, 2016 AbstractBackground: CR and CR with incomplete count recovery (CRi) are associated with prolonged overall survival (OS) for acute myeloid leukemia (AML) patients (pts) treated with curative-intent, induction ... Full text Cite

A Phase 1/2 Study of G-CSF, Cladribine, Cytarabine, and Dose-Escalated Mitoxantrone (G-CLAM) in Adults with Newly Diagnosed Acute Myeloid Leukemia (AML) or High-Risk Myelodysplastic Syndrome (MDS)

Conference Blood · December 2, 2016 AbstractIntroduction:"7+3" with standard doses of cytarabine and an anthracycline has remained the mainstay of induction chemotherapy for newly diagnosed AML. Since some studies have shown improved outcomes ... Full text Cite

Feasibility of Allogeneic Hematopoietic Cell Transplantation Among High-Risk AML Patients in First Complete Remission: Results of the Transplant Objective from the SWOG (S1203) Randomized Phase III Study of Induction Therapy Using Standard 7+3 Therapy or Idarubicin with High-Dose Cytarabine (IA) Versus IA Plus Vorinostat

Conference Blood · December 2, 2016 AbstractBackground: Adult acute myeloid leukemia (AML) patients with high-risk cytogenetics have a significantly worse survival compared to similarly treated intermediate- or favorable-risk patients. Althoug ... Full text Cite

Vadastuximab Talirine Monotherapy in Older Patients with Treatment Naive CD33-Positive Acute Myeloid Leukemia (AML)

Conference Blood · December 2, 2016 AbstractBackgroundCD33is expressed in approximately 90% of AML cases, representing a promising target despite age, prior therapies, or mutational heterogeneity. Vadastuximab ... Full text Cite

Results of a Clinical Study of Pevonedistat (Pev), a First-in-Class NEDD8-Activating Enzyme (NAE) Inhibitor, Combined with Azacitidine (Aza) in Older Patients (Pts) with Acute Myeloid Leukemia (AML)

Conference Blood · December 2, 2016 AbstractBackground: Pev (TAK-924/MLN4924), a novel investigational NAE inhibitor, enhances the anti-leukemic effects of aza in AML cell lines and murine xenografts (Smith et al, Blood 2011). Single-agent pev ... Full text Cite

A Phase 1b Study of Vadastuximab Talirine in Combination with 7+3 Induction Therapy for Patients with Newly Diagnosed Acute Myeloid Leukemia (AML)

Conference Blood · December 2, 2016 AbstractBackgroundFor patients who are less than 65 years with newly diagnosed AML, standard induction treatment is continuous infusion cytarabine for 7 days and an anthracyc ... Full text Cite

Vadastuximab Talirine Plus Hypomethylating Agents: A Well-Tolerated Regimen with High Remission Rate in Frontline Older Patients with Acute Myeloid Leukemia (AML)

Conference Blood · December 2, 2016 AbstractBackgroundTreatment of AML among the elderly is challenging due to intolerance of intensive therapy and greater prevalence of therapy-resistant biology. Hypomethylati ... Full text Cite

A Phase 1b Study of Vadastuximab Talirine As Maintenance and in Combination with Standard Consolidation for Patients with Acute Myeloid Leukemia (AML)

Conference Blood · December 2, 2016 AbstractBackgroundPost-remission therapies for patients with AML such as high-dose cytarabine (HiDAC) and allogeneic stem cell transplant (alloSCT) have led to improved outco ... Full text Cite

Final Results of the Chrysalis Trial: A First-in-Human Phase 1/2 Dose-Escalation, Dose-Expansion Study of Gilteritinib (ASP2215) in Patients with Relapsed/Refractory Acute Myeloid Leukemia (R/R AML)

Conference Blood · December 2, 2016 AbstractBackground: Gilteritinib (ASP2215) is a novel, highly selective, potent oral FLT3/AXL inhibitor with preclinical activity against FLT3-ITD activating and FLT3-D835 resistanc ... Full text Cite

Durable Overall Survival Benefit in Patients ≥ 60 Years with Relapsed or Refractory AML Treated with Vosaroxin/Cytarabine Vs Placebo/Cytarabine: Updated Results from the Valor Trial

Conference Blood · December 2, 2016 AbstractBackground: Prognosis for older patients with relapsed or refractory (R/R) acute myeloid leukemia (AML) is poor, with lower response rates and shorter survival compared with ... Full text Cite

Connect MDS/AML: design of the myelodysplastic syndromes and acute myeloid leukemia disease registry, a prospective observational cohort study.

Journal Article BMC Cancer · August 19, 2016 BACKGROUND: Myelodysplastic syndromes (MDS) and acute myeloid leukemia (AML) are myeloid neoplasms in which outgrowth of neoplastic clones disrupts normal hematopoiesis. Some patients with unexplained persistent cytopenias may not meet minimal diagnostic c ... Full text Link to item Cite

Association between early promoter-specific DNA methylation changes and outcome in older acute myeloid leukemia patients.

Journal Article Leuk Res · March 2016 Treatment options for older patients with acute myeloid leukemia (AML) range from supportive care alone to full-dose chemotherapy. Identifying factors that predict response to therapy may help increase efficacy and avoid toxicity. The phase II SWOG S0703 s ... Full text Link to item Cite

Azacitidine with or without Entinostat for the treatment of therapy-related myeloid neoplasm: further results of the E1905 North American Leukemia Intergroup study.

Journal Article Br J Haematol · February 2016 Therapy-related myeloid neoplasms (tMN) are serious late effects of the treatment of cancer with poor response to conventional treatment. Azacitidine (AZA) has been used to treat patients with tMN but current data are retrospective. We present here 47 tMN ... Full text Link to item Cite

In Vitro Pre-Clinical Validation of Suicide Gene Modified Anti-CD33 Redirected Chimeric Antigen Receptor T-Cells for Acute Myeloid Leukemia.

Journal Article PLoS One · 2016 BACKGROUND: Approximately fifty percent of patients with acute myeloid leukemia can be cured with current therapeutic strategies which include, standard dose chemotherapy for patients at standard risk of relapse as assessed by cytogenetic and molecular ana ... Full text Link to item Cite

Multi-Center US Intergroup Study of Intensive Chemotherapy Plus Dasatinib Followed By Allogeneic Stem Cell Transplant in Patients with Philadelphia Chromosome Positive Acute Lymphoblastic Leukemia Younger Than 60

Conference Blood · December 3, 2015 AbstractBackground- Introduction of tyrosine kinase inhibitors in the treatment of patients with Philadelphia (Ph) chromosome positive (Ph+) acute lymphoblastic leukemia (ALL) has l ... Full text Cite

Vosaroxin plus cytarabine versus placebo plus cytarabine in patients with first relapsed or refractory acute myeloid leukaemia (VALOR): a randomised, controlled, double-blind, multinational, phase 3 study.

Journal Article Lancet Oncol · September 2015 BACKGROUND: Safe and effective treatments are urgently needed for patients with relapsed or refractory acute myeloid leukaemia. We investigated the efficacy and safety of vosaroxin, a first-in-class anticancer quinolone derivative, plus cytarabine in patie ... Full text Link to item Cite

Oral Debio1143 (AT406), an antagonist of inhibitor of apoptosis proteins, combined with daunorubicin and cytarabine in patients with poor-risk acute myeloid leukemia--results of a phase I dose-escalation study.

Journal Article Clin Lymphoma Myeloma Leuk · July 2015 BACKGROUND: Treatment of acute myeloid leukemia (AML) remains difficult owing to the development of treatment resistance, which might be overcome through antagonists of inhibitors of apoptosis proteins (IAPs). PATIENTS AND METHODS: The present multicenter, ... Full text Link to item Cite

Pevonedistat (MLN4924), a First-in-Class NEDD8-activating enzyme inhibitor, in patients with acute myeloid leukaemia and myelodysplastic syndromes: a phase 1 study.

Journal Article Br J Haematol · May 2015 This trial was conducted to determine the dose-limiting toxicities (DLTs) and maximum tolerated dose (MTD) of the first in class NEDD8-activating enzyme (NAE) inhibitor, pevonedistat, and to investigate pevonedistat pharmacokinetics and pharmacodynamics in ... Full text Link to item Cite

Phase III open-label randomized study of cytarabine in combination with amonafide L-malate or daunorubicin as induction therapy for patients with secondary acute myeloid leukemia.

Journal Article J Clin Oncol · April 10, 2015 PURPOSE: Secondary acute myeloid leukemia (sAML), defined as AML arising after a prior myelodysplastic syndrome or after antineoplastic therapy, responds poorly to current therapies. It is often associated with adverse karyotypic abnormalities and overexpr ... Full text Link to item Cite

Cytogenetic heterogeneity negatively impacts outcomes in patients with acute myeloid leukemia.

Journal Article Haematologica · March 2015 Clonal heterogeneity is a hallmark of malignant transformation. In acute myeloid leukemia, acquired cytogenetic abnormalities are important independent predictors of initial response to therapy, remission duration, and overall survival. However, whether th ... Full text Link to item Cite

Recent developments in the treatment of older individuals with acute myeloid leukemia: 2014.

Journal Article Curr Opin Hematol · March 2015 PURPOSE OF REVIEW: The treatment of acute myeloid leukemia (AML) in older persons remains a tremendous clinical challenge. AML in older patients is more often associated with biologically unfavorable features, and these patients are less likely to tolerate ... Full text Link to item Cite

Molecular monitoring to improve outcomes in patients with chronic myeloid leukemia in chronic phase: importance of achieving treatment-free remission.

Journal Article Am J Hematol · March 2015 Imatinib was the first BCR-ABL1 tyrosine kinase inhibitor (TKI) developed for the treatment of patients with chronic myeloid leukemia (CML); subsequently, the introduction of more potent BCR-ABL1 TKIs has raised expectations regarding the speed and depth o ... Full text Link to item Cite

Fate of patients with newly diagnosed acute myeloid leukemia who fail primary induction therapy.

Journal Article Biol Blood Marrow Transplant · March 2015 The aim of this study was to describe the fate of patients with newly diagnosed acute myeloid leukemia (AML) who did not achieve an initial remission while being treated on a contemporary cooperative group trial. We analyzed the outcome of patients entered ... Full text Link to item Cite

Acute myeloid leukemia ontogeny is defined by distinct somatic mutations.

Journal Article Blood · February 26, 2015 Acute myeloid leukemia (AML) can develop after an antecedent myeloid malignancy (secondary AML [s-AML]), after leukemogenic therapy (therapy-related AML [t-AML]), or without an identifiable prodrome or known exposure (de novo AML). The genetic basis of the ... Full text Link to item Cite

Finding the optimal combination therapy for the treatment of newly diagnosed AML in older patients unfit for intensive therapy.

Journal Article Leuk Res · February 2015 There is no standard of care for older patients with newly diagnosed acute myeloid leukemia (AML) unfit for intensive therapy, and prognosis with currently recommended low-intensity therapies (decitabine, azacitidine, and low-dose cytarabine [LDAC]) remain ... Full text Link to item Cite

Favorable Outcomes for Older Adolescents and Young Adults (AYA) with Acute Lymphoblastic Leukemia (ALL): Early Results of U.S. Intergroup Trial C10403

Conference Blood · December 6, 2014 AbstractBackground: Retrospective analyses have demonstrated significantly improved survival for AYA ALL patients (pts) aged 16-21 years (yrs) when treated on pediatric versus adult ... Full text Cite

Platelet count doubling after the first cycle of azacitidine therapy predicts eventual response and survival in patients with myelodysplastic syndromes and oligoblastic acute myeloid leukaemia but does not add to prognostic utility of the revised IPSS.

Journal Article Br J Haematol · October 2014 Reliable clinical or molecular predictors of benefit from azacitidine therapy in patients with myelodysplastic syndromes (MDS) are not defined. Doubling of platelet count at start of second cycle of azacitidine therapy compared to baseline was associated w ... Full text Link to item Cite

Comparison of the prognostic utility of the revised International Prognostic Scoring System and the French Prognostic Scoring System in azacitidine-treated patients with myelodysplastic syndromes.

Journal Article Br J Haematol · August 2014 The revised International Prognostic Scoring System (IPSS-R) was developed in a cohort of untreated myelodysplastic syndromes (MDS) patients. A French Prognostic Scoring System (FPSS) was recently reported to identify differential survival among azacitidin ... Full text Link to item Cite

Cytogenetics and Prognostic Models in Myelodysplastic Syndromes

Chapter · June 20, 2014 Myelodysplastic syndromes (MDS), discussed in this chapter, are a heterogeneous group of clonal hematopoietic disorders that have an increased risk of progression to acute myeloid leukemia (AML). The prognosis of MDS patients varies significantly based on ... Full text Cite

Prolonged administration of azacitidine with or without entinostat for myelodysplastic syndrome and acute myeloid leukemia with myelodysplasia-related changes: results of the US Leukemia Intergroup trial E1905.

Journal Article J Clin Oncol · April 20, 2014 PURPOSE: Although azacitidine (AZA) improves survival in patients with high-risk myelodysplastic syndrome, the overall response remains approximately 50%. Entinostat is a histone deacetylase inhibitor that has been combined with AZA with significant clinic ... Full text Link to item Cite

Four different regimens of farnesyltransferase inhibitor tipifarnib in older, untreated acute myeloid leukemia patients: North American Intergroup Phase II study SWOG S0432.

Journal Article Leuk Res · March 2014 We report on 348 patients ≥ 70 years (median age 78 years) with acute myeloid leukemia (>50% with secondary AML) randomized to receive either 600 mg or 300 mg of tipifarnib orally twice daily on days 1-21 or days 1-7 and 15-21, repeated every 28 days (4 tr ... Full text Link to item Cite

Declining rates of treatment-related mortality in patients with newly diagnosed AML given 'intense' induction regimens: a report from SWOG and MD Anderson.

Journal Article Leukemia · February 2014 Less-intense remission induction regimens for adults with newly diagnosed acute myeloid leukemia (AML) aim to reduce treatment-related mortality (TRM), here defined as death within 4 weeks after starting induction therapy. This assumes that TRM rates are s ... Full text Link to item Cite

Phase II study of the oral MEK inhibitor selumetinib in advanced acute myelogenous leukemia: a University of Chicago phase II consortium trial.

Journal Article Clin Cancer Res · January 15, 2014 PURPOSE: The clinical relevance of targeting the RAS/RAF/MEK/ERK pathway, activated in 70% to 80% of patients with acute myelogenous leukemia (AML), is unknown. EXPERIMENTAL DESIGN: Selumetinib is an oral small-molecule inhibitor of MAP-ERK kinase (MEK)-1/ ... Full text Link to item Cite

A multicenter phase I/II study of obatoclax mesylate administered as a 3- or 24-hour infusion in older patients with previously untreated acute myeloid leukemia.

Journal Article PLoS One · 2014 PURPOSE: An open-label phase I/II study of single-agent obatoclax determined a maximum tolerated dose (MTD) and schedule, safety, and efficacy in older patients (≥ 70 yr) with untreated acute myeloid leukemia (AML). EXPERIMENTAL DESIGN: Phase I evaluated t ... Full text Open Access Link to item Cite

Azacitidine With Or Without Entinostat For The Treatment Of Therapy-Related Myeloid Neoplasm: Further Results Of The E1905 North American Leukemia Intergroup Study

Conference Blood · November 15, 2013 AbstractBackgroundTherapy-related myeloid neoplasm (tMN) includes t-myelodysplasia (tMDS) and t-acute myeloi ... Full text Cite

Alteration Of Cytogenetic Risk Stratification In New Diagnosis Of Leukemia By FISH Testing – Retrospective Review Of The SWOG Experience

Conference Blood · November 15, 2013 AbstractBackgroundFluorescence in situ hybridization (FISH) is an invaluable tool for complementing conventi ... Full text Cite

MLN4924, a Novel Investigational Inhibitor Of NEDD8-Activating Enzyme (NAE), In Adult Patients With Acute Myeloid Leukemia (AML) and Myelodysplastic Syndrome (MDS): Results From Multiple Dosing Schedules In a Phase 1 Study

Conference Blood · November 15, 2013 AbstractBackgroundNEDD8-activating enzyme (NAE) regulates the NEDD8 conjugation pathway, and is required for activity of the cullin-RI ... Full text Cite

A phase 2 trial of azacitidine and gemtuzumab ozogamicin therapy in older patients with acute myeloid leukemia.

Journal Article Blood · November 14, 2013 This trial tested the safety and efficacy of a regimen consisting of hydroxyurea followed by azacitidine, 75 mg/m(2) for 7 days, and gemtuzumab ozogamicin, 3 mg/m(2) on day 8, in older patients with newly diagnosed acute myeloid leukemia. Those achieving a ... Full text Link to item Cite

Safety and pharmacokinetics of the antisense oligonucleotide (ASO) LY2181308 as a single-agent or in combination with idarubicin and cytarabine in patients with refractory or relapsed acute myeloid leukemia (AML).

Journal Article Invest New Drugs · August 2013 Survivin is expressed in tumor cells, including acute myeloid leukemia (AML), regulates mitosis, and prevents tumor cell death. The antisense oligonucleotide sodium LY2181308 (LY2181308) inhibits survivin expression and may cause cell cycle arrest and rest ... Full text Link to item Cite

A phase 3 study of gemtuzumab ozogamicin during induction and postconsolidation therapy in younger patients with acute myeloid leukemia.

Journal Article Blood · June 13, 2013 This randomized phase 3 clinical trial evaluated the potential benefit of the addition of gemtuzumab ozogamicin (GO) to standard induction and postconsolidation therapy in patients with acute myeloid leukemia. Patients were randomly assigned to receive dau ... Full text Link to item Cite

North American Cooperative Trials in AML

Conference ANNALS OF HEMATOLOGY · February 1, 2013 Link to item Cite

[Acute myeloid leukemia].

Journal Article Nihon Rinsho · November 2012 Link to item Cite

Potential of whole-genome sequencing for determining risk and personalizing therapy: focus on AML.

Journal Article Expert Rev Anticancer Ther · October 2012 In spite of recent advances in molecular diagnostic techniques and expanded indications for allogeneic hematopoietic stem cell transplantation, treatment of acute myeloid leukemia (AML) remains a major challenge. In the last decade, several recurrent genet ... Full text Link to item Cite

Outpatient consolidation treatment with clofarabine in a phase 2 study of older adult patients with previously untreated acute myelogenous leukemia.

Journal Article Leuk Lymphoma · March 2012 This report describes outpatient (OP) administration of clofarabine in older patients (≥60 years) with untreated acute myelogenous leukemia (AML). Overall, 112 patients underwent clofarabine induction. Clofarabine was administered to 35 OPs for a total of ... Full text Link to item Cite

Somatic mutations in the transcriptional corepressor gene BCORL1 in adult acute myelogenous leukemia.

Journal Article Blood · November 24, 2011 To further our understanding of the genetic basis of acute myelogenous leukemia (AML), we determined the coding exon sequences of ∼ 18 000 protein-encoding genes in 8 patients with secondary AML. Here we report the discovery of novel somatic mutations in t ... Full text Link to item Cite

Clofarabine and busulfan conditioning facilitates engraftment and provides significant antitumor activity in nonremission hematologic malignancies.

Journal Article Blood · October 13, 2011 Patients with hematologic malignancies not in remission before allogeneic hematopoietic stem cell transplantation (HSCT) have a poor prognosis. To improve the antitumor activity of conditioning, we combined clofarabine with myeloablative doses of busulfan ... Full text Link to item Cite

Improving frontline treatment for chronic myeloid leukemia: emerging evidence for use of nilotinib and dasatinib.

Journal Article Clin Adv Hematol Oncol · October 2011 The approval of imatinib in 2001 changed the landscape of chronic myeloid leukemia (CML) management, becoming the standard of care and improving the survival rates of patients. With the prevalent use of imatinib worldwide, it was observed that up to one-th ... Link to item Cite

A pathobiological role of the insulin receptor in chronic lymphocytic leukemia.

Journal Article Clin Cancer Res · May 1, 2011 PURPOSE: The chromosomal deletion 11q affects biology and clinical outcome in chronic lymphocytic leukemia (CLL) but del11q-deregulated genes remain incompletely characterized. EXPERIMENTAL DESIGN: We have employed integrated genomic profiling approaches o ... Full text Link to item Cite

Results from a randomized trial of salvage chemotherapy followed by lestaurtinib for patients with FLT3 mutant AML in first relapse.

Journal Article Blood · March 24, 2011 In a randomized trial of therapy for FMS-like tyrosine kinase-3 (FLT3) mutant acute myeloid leukemia in first relapse, 224 patients received chemotherapy alone or followed by 80 mg of the FLT3 inhibitor lestaurtinib twice daily. Endpoints included complete ... Full text Link to item Cite

A Phase 2 study of combination therapy with arsenic trioxide and gemtuzumab ozogamicin in patients with myelodysplastic syndromes or secondary acute myeloid leukemia.

Journal Article Cancer · March 15, 2011 BACKGROUND: Higher-risk myelodysplastic syndromes (MDS) are similar pathobiologically to acute myeloid leukemia (AML), particularly in older adults. AML therapies thus may have activity in MDS. In the current study, phase 2 study data of arsenic trioxide ( ... Full text Link to item Cite

Targeting protein neddylation: a novel therapeutic strategy for the treatment of cancer.

Journal Article Expert Opin Ther Targets · March 2011 INTRODUCTION: The NEDD8 (neural precursor cell-expressed developmentally downregulated 8) conjugation pathway regulates the post-translational modification of oncogenic proteins. This pathway has important potential for cancer therapeutics. Several protein ... Full text Link to item Cite

Acquired genomic copy number aberrations and survival in adult acute myelogenous leukemia.

Journal Article Blood · December 2, 2010 Genomic aberrations are of predominant importance to the biology and clinical outcome of patients with acute myelogenous leukemia (AML), and conventional karyotype-based risk classifications are routinely used in clinical decision making in AML. One of the ... Full text Link to item Cite

Has there been progress in the treatment of older patients with acute myeloid leukemia?

Journal Article Best Pract Res Clin Haematol · December 2010 The treatment of older patients with acute myeloid leukemia (AML) has become increasingly important as the population ages. Progress, measured by overall survival rates, has improved in younger patients, perhaps due to the use of intensive post-remission t ... Full text Link to item Cite

Allogeneic Hematopoietic Stem Cell Transplantation with Clofarabine/Busulfan × 4 (CloBu4) Conditioning Exhibits Significant Anti-Tumor Activity In Non - Remission Hematologic Malignancies, Especially In AML

Conference Blood · November 19, 2010 AbstractAbstract 35Patients with hematologic malignancies who are not in remission prior to allogeneic hematopoi ... Full text Cite

NF1 inactivation in adult acute myelogenous leukemia.

Journal Article Clin Cancer Res · August 15, 2010 PURPOSE: This study was conducted to identify novel genes with importance to the biology of adult acute myelogenous leukemia (AML). EXPERIMENTAL DESIGN: We analyzed DNA from highly purified AML blasts and paired buccal cells from 95 patients for recurrent ... Full text Link to item Cite

Multiple distinct molecular mechanisms influence sensitivity and resistance to MDM2 inhibitors in adult acute myelogenous leukemia.

Journal Article Blood · July 8, 2010 The survival of most patients with acute myelogenous leukemia (AML) remains poor, and novel therapeutic approaches are needed to improve outcomes. Given that the fraction of AML with mutated p53 is small ( approximately 10%), it appears rational to study M ... Full text Link to item Cite

Phase II study of clofarabine monotherapy in previously untreated older adults with acute myeloid leukemia and unfavorable prognostic factors.

Journal Article J Clin Oncol · February 1, 2010 PURPOSE: This phase II study assessed clofarabine monotherapy in older adults (>or= 60 years of age) with untreated acute myeloid leukemia (AML) and at least one unfavorable baseline prognostic factor. PATIENTS AND METHODS: Clofarabine was administered int ... Full text Link to item Cite

Sequential phase II Southwest Oncology Group studies (S0112 and S0301) of daunorubicin and cytarabine by continuous infusion, without and with ciclosporin, in older patients with previously untreated acute myeloid leukaemia.

Journal Article Br J Haematol · January 2010 Attempts to overcome multi-drug resistance in acute myeloid leukaemia (AML) have been limited by toxicities. To investigate the effect of reducing peak drug levels, we performed sequential phase II studies using continuous infusion daunorubicin and cytarab ... Full text Link to item Cite

Phase II Study of the Oral MEK Inhibitor AZD6244 in Advanced Acute Myeloid Leukemia (AML).

Conference Blood · November 20, 2009 AbstractAbstract 2081Poster Board II-58AZD6244 is an orally bioavailable small molecule inhibitor of the MEK kinase. MEK is downstream of the ... Full text Cite

NCCN task force report: molecular markers in leukemias and lymphomas.

Journal Article J Natl Compr Canc Netw · July 2009 The introduction of targeted therapies has revolutionized treatment and improved outcomes in patients with leukemias and lymphomas. However, many patients experience relapse caused by the persistence of residual malignant cells. Cytogenetic and molecular t ... Full text Link to item Cite

Myeloid growth factors.

Journal Article J Natl Compr Canc Netw · January 2009 Full text Link to item Cite

Acute myeloid leukemia.

Journal Article J Natl Compr Canc Netw · November 2008 Full text Link to item Cite

Myelodysplastic syndromes.

Journal Article J Natl Compr Canc Netw · October 2008 Link to item Cite

Genomic complexity identifies patients with aggressive chronic lymphocytic leukemia.

Journal Article Blood · September 1, 2008 Chronic lymphocytic leukemia (CLL) has a variable clinical course. Presence of specific genomic aberrations has been shown to impact survival outcomes and can help categorize CLL into clinically distinct subtypes. We studied 178 CLL patients enrolled in a ... Full text Link to item Cite

Integrated genomic profiling of chronic lymphocytic leukemia identifies subtypes of deletion 13q14.

Journal Article Cancer Res · February 15, 2008 Chronic lymphocytic leukemia (CLL) is a biologically heterogeneous illness with a variable clinical course. Loss of chromosomal material on chromosome 13 at cytoband 13q14 is the most frequent genetic abnormality in CLL, but the molecular aberrations under ... Full text Link to item Cite

Comprehensive biomarker and genomic analysis identifies p53 status as the major determinant of response to MDM2 inhibitors in chronic lymphocytic leukemia.

Journal Article Blood · February 1, 2008 Chronic lymphocytic leukemia (CLL) is the most common leukemia in the Western world and remains incurable with conventional therapies. Patients with relapsed or resistant CLL have a significantly shortened lifespan. MDM2 inhibitors have been developed and ... Full text Link to item Cite

Phase II Studies of Different Schedules and Doses of the Farnesyl Transferase Inhibitor Tipifarnib (R115777, Zarnestra, NSC-702818) for Patients of Age 70 or Older with Previously Untreated Acute Myeloid Leukemia (AML): A North American Intergroup Study (S0432).

Conference Blood · November 16, 2007 AbstractThe prognosis of older adults with acute myeloid leukemia (AML) has not improved over the last three decades, despite the improvement in the outcome of younger adults during this same time due to the ... Full text Cite

In vitro and in silico analysis of annexin V binding to lymphocytes as a biomarker in emergency department sepsis studies.

Journal Article Acad Emerg Med · September 2007 BACKGROUND: Peripheral blood lymphocyte apoptosis is a recognized feature of serious infection and sepsis and can be easily quantified by flow cytometric measurement of annexin V binding to the cell surface. Use of apoptosis as a biomarker in emergency dep ... Full text Link to item Cite

Institutional experience with voriconazole compared with liposomal amphotericin B as empiric therapy for febrile neutropenia.

Journal Article Pharmacotherapy · July 2007 STUDY OBJECTIVE: To assess the effectiveness, safety, and cost of empiric treatment of febrile neutropenia before and after implementing an algorithm in which voriconazole was substituted for liposomal amphotericin B (L-AmB). DESIGN: Retrospective cohort a ... Full text Link to item Cite

Amonafide and ara-C treatment for secondary acute myeloid leukemia (sAML)

Conference JOURNAL OF CLINICAL ONCOLOGY · June 20, 2007 Link to item Cite

Chronic myelogenous leukemia.

Journal Article J Natl Compr Canc Netw · May 2007 Full text Link to item Cite

Prognostic factors in elderly patients with AML and the implications for treatment.

Journal Article Hematology Am Soc Hematol Educ Program · 2007 The outcome of older patients with acute myeloid leukemia (AML) has not improved in the last three decades. These patients are more likely to have comorbid illness, poor performance status, and impaired organ function. These clinical features limit their a ... Full text Link to item Cite

A Phase II Trial of Combination Therapy with Arsenic Trioxide (ATO) and Gemtuzumab Ozogamicin (GO) in Patients with High-Risk Myelodysplastic Syndromes (MDS) or Acute Myeloid Leukemia (AML) Arising from MDS.

Conference Blood · November 16, 2006 AbstractBackground: Bone marrow findings in high-risk MDS are similar to those found in AML. MDS-associated myeloblasts are just as likely to express CD33 as AML myeloblasts, and cytogenetic abnormalities ar ... Full text Cite

Genomic Profiling of Chronic Lymphocytic Leukemia Identifies Novel Genetic Subtypes.

Conference Blood · November 1, 2006 AbstractCLL is the most common leukemia in the Western world and displays considerable clinical heterogeneity. Genetic approaches to elucidate defects in CLL genomes have resulted in the identification of re ... Full text Cite

Myelodysplastic syndromes clinical practice guidelines in oncology.

Journal Article J Natl Compr Canc Netw · January 2006 Link to item Cite

Combination of Daunorubicin and Cytarabine Chemotherapy with Gemtuzumab Ozogamicin as Initial Therapy for Elderly Patients with Previously Untreated Acute Myeloid Leukemia.

Conference Blood · November 16, 2005 AbstractAcute myeloid leukemia (AML) is a hematopoietic neoplasm that primarily affects older adults. The prognosis for elderly patients with AML remains poor. Treatment with standard induction chemotherapy ... Full text Cite

Chronic myelogenous leukemia.

Journal Article J Natl Compr Canc Netw · November 2005 Full text Link to item Cite

Treatment options for newly diagnosed patients with chronic lymphocytic leukemia.

Journal Article Curr Hematol Rep · January 2004 Chronic lymphocytic leukemia (CLL) is an indolent B-cell malignancy for which early intervention has not been shown to extend survival. However, there are many agents available that are active in this disease. Alkylating agents, the purine analogs, and mon ... Link to item Cite

Health status and quality of life in patients with early-stage Hodgkin's disease treated on Southwest Oncology Group Study 9133.

Journal Article J Clin Oncol · September 15, 2003 PURPOSE: We describe the short and intermediate-term quality-of-life (QOL) outcomes in patients treated on a randomized clinical trial in early-stage Hodgkin's disease (Southwest Oncology Group [SWOG] 9133) comparing subtotal lymphoid irradiation (STLI) wi ... Full text Link to item Cite

Recent progress in the treatment of myelodysplastic syndrome in adult patients.

Journal Article Curr Opin Oncol · January 2003 Peripheral blood cytopenias and dysplastic hematopoietic progenitors characterize the myelodysplastic syndrome. Patients suffer from the consequences of chronic cytopenias or progression to acute myelogenous leukemia. Although allogeneic hematopoietic stem ... Full text Link to item Cite

Antibody-targeted chemotherapy of older patients with acute myeloid leukemia in first relapse using Mylotarg (gemtuzumab ozogamicin).

Journal Article Leukemia · September 2002 We analyzed the safety and efficacy of Mylotarg (gemtuzumab ozogamicin, an antibody-targeted chemotherapy consisting of a humanized anti-CD33 antibody linked to calicheamicin, a potent antitumor antibiotic) in the treatment of 101 patients > or =60 years o ... Full text Link to item Cite

NCCN Practice Guidelines for Chronic Myelogenous Leukemia.

Journal Article Oncology (Williston Park) · November 2000 Link to item Cite

NCCN Practice Guidelines for Acute Myelogenous Leukemia.

Journal Article Oncology (Williston Park) · November 2000 Link to item Cite

NCCN Practice Guidelines for acute myelogenous leukemia

Journal Article ONCOLOGY-NEW YORK · November 1, 2000 Link to item Cite

NCCN Practice Guidelines for chronic myelogenous leukemia

Journal Article ONCOLOGY-NEW YORK · November 1, 2000 Link to item Cite

Reversible pulmonary hypertension in POEMS syndrome--another etiology of triggered pulmonary vasculopathy?

Journal Article Can J Cardiol · August 2000 Polyneuropathy, organomegaly, endocrinopathy, monoclonal gammopathy and skin changes (POEMS) syndrome, a plasma cell dyscrasia associated with pulmonary hypertension, has been treated in the past with anticytokine strategies with a poor outcome. A patient ... Link to item Cite

Nucleotide sequence and expression of the human skeletal alpha-actin gene: evolution of functional regulatory domains.

Journal Article Genomics · November 1988 Regulation of the actin multigene family involves the recognition of regulatory sequences that specify the tissue type and developmental program of expression for each actin isotype. In order to investigate the underlying regulatory mechanisms, the human s ... Full text Link to item Cite

Modulation of microfilament protein composition by transfected cytoskeletal actin genes.

Journal Article Mol Cell Biol · April 1988 HuT-14T is a highly tumorigenic fibroblast cell line which exhibits a reduced steady-state level of beta-actin due to coding mutations in one of two beta-actin alleles. The normal rate of total actin synthesis could be restored in some clones of cells foll ... Full text Link to item Cite

Structure, chromosome location, and expression of the human gamma-actin gene: differential evolution, location, and expression of the cytoskeletal beta- and gamma-actin genes.

Journal Article Mol Cell Biol · April 1988 The accumulation of the cytoskeletal beta- and gamma-actin mRNAs was determined in a variety of mouse tissues and organs. The beta-isoform is always expressed in excess of the gamma-isoform. However, the molar ratio of beta- to gamma-actin mRNA varies from ... Full text Link to item Cite

Cytoskeletal actin gene families of Xenopus borealis and Xenopus laevis.

Journal Article J Mol Evol · 1988 We have sequenced the coding and leader regions, as well as part of the 3' untranslated region, of a Xenopus borealis type 1 cytoskeletal actin gene [defined according to the arrangement of acidic residues at the N-terminus; Vandekerckhove et al. (1981) J ... Full text Link to item Cite

Nucleotide sequence of the human gamma cytoskeletal actin mRNA: anomalous evolution of vertebrate non-muscle actin genes.

Journal Article Nucleic Acids Res · July 11, 1986 Two distinct, but iso-coding, gamma non-muscle actin cDNAs were isolated from an SV40-transformed human fibroblast library. The complete nucleotide sequence of the human gamma non-muscle actin cDNAs indicates that they may have arisen from polymorphic alle ... Full text Link to item Cite

Histone gene clusters of the newt notophthalmus are separated by long tracts of satellite DNA.

Journal Article Cell · June 1981 The genomic organization of the histone genes of the newt Notophthalmus viridescens is described. Genes for the five proteins are clustered on a 9.0 kb segment of cloned DNA which is part of a homogeneous family of sequences containing 600--800 members per ... Full text Link to item Cite

Characterization of a cloned histone gene cluster of the newt Notophthalamus viridescens.

Journal Article Nucleic Acids Res · May 25, 1981 We report the cloning and characterization of a histone gene cluster of the newt Notophthalamus viridescens. Fragments containing newt histone genes were identified in whole genome Southern blots; these fragments were cloned into a bacteriophage lambda clo ... Full text Link to item Cite

Histone genes are located at the sphere loci of newt lampbrush chromosomes.

Journal Article Chromosoma · 1981 In situ hybridization experiments on mitotic and lampbrush chromosomes show that histone genes and an associated 222 bp repeated sequence, satellite 1, are located at or near the sphere loci of chromosomes 2 and 6 of the newt Notophthalmus viridescens. Dur ... Full text Link to item Cite

Satellite DNA is transcribed on lampbrush chromosomes.

Journal Article Nature · February 14, 1980 During the lampbrush stage of oogenesis there is widespread transcription, and it has been estimated that the total amount of DNA transcribed may be an order of magnitude greater than that required to produce the necessary functional RNA for the oocyte. We ... Full text Link to item Cite

Cytological evidence of transcription of highly repeated DNA sequences during the lampbrush stage in Triturus cristatus carnifex.

Journal Article Chromosoma · 1980 Highly repeated, or satellite, DNA fractions have been isolated from total Triturus cristatus carnifex DNA by renaturation kinetics, caesium salt centrifugation and restriction endonuclease digestion. We have shown by DNA/DNA in situ hybridisation and auto ... Full text Link to item Cite