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Harry Paul Erba CV

Professor of Medicine
Medicine, Hematologic Malignancies and Cellular Therapy
DUMC Box 3961, Durham, NC 27710
2400 Pratt St Suite 5000, DUMC Box 3961, Durham, NC 27710
CV

Scholarly Works - Journal articles


Quizartinib for patients with newly diagnosed FLT3-ITD-positive AML who received maintenance therapy in QuANTUM-First.

Journal article Blood Adv · June 23, 2026 QuANTUM-First demonstrated improved overall survival (OS) in patients with newly diagnosed acute myeloid leukemia with FMS-like receptor tyrosine kinase 3-internal tandem duplication (FLT3-ITD) treated with quizartinib + standard chemotherapy. Herein, we e ... Full text Link to item Cite

Ziftomenib with venetoclax and azacitidine in relapsed/refractory NPM1-mutated acute myeloid leukemia.

Journal article Blood · June 2, 2026 Ziftomenib - a potent, selective, oral menin inhibitor - is approved as monotherapy for adults with relapsed/refractory (R/R) NPM1-mutated acute myeloid leukemia (NPM1-m AML). The KOMET-007 phase 1 trial investigated clinical activity and tolerability of z ... Full text Link to item Cite

Pivekimab Sunirine in Blastic Plasmacytoid Dendritic Cell Neoplasm.

Journal article J Clin Oncol · April 2026 PURPOSE: Blastic plasmacytoid dendritic cell neoplasm (BPDCN) is a unique myeloid malignancy with CD123 interleukin-3 receptor-α overexpression and poor prognosis. METHODS: This phase I/II, open-label, multicenter study evaluated pivekimab sunirine (PVEK), ... Full text Link to item Cite

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

Journal article Blood Adv · February 10, 2026 QuANTUM-First was a randomized trial that demonstrated that the addition of quizartinib, a potent and selective FMS-like tyrosine kinase 3 (FLT3) inhibitor, to induction and consolidation chemotherapy, followed by monotherapy maintenance, improved the surv ... Full text Link to item Cite

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

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

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

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

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

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

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

Journal article Blood Adv · April 11, 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 Link to item 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

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

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

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

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

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 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

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

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

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

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

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

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

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

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

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

[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

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

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

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

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

Myelodysplastic syndromes clinical practice guidelines in oncology.

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