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Richard Francis Riedel

Associate Professor of Medicine
Medicine, Medical Oncology
Duke Box 3198, Durham, NC 27710
Dept of Medicine, 25178 Morris Bldg, Durham, NC 27710

Selected Publications


Onset and resolution of ovarian toxicity with nirogacestat treatment in females with desmoid tumors: Updated safety analyses from the DeFi phase 3 study.

Journal Article Cancer · May 4, 2024 INTRODUCTION: Nirogacestat is a targeted gamma secretase inhibitor approved in the United States for adults with progressing desmoid tumors. In the phase 3 DeFi study (NCT03785964) of nirogacestat, ovarian toxicity (OT) was identified as a safety signal am ... Full text Link to item Cite

Phase II Trial of nab-Sirolimus in Patients With Advanced Malignant Perivascular Epithelioid Cell Tumors (AMPECT): Long-Term Efficacy and Safety Update.

Journal Article J Clin Oncol · May 1, 2024 Clinical trials frequently include multiple end points that mature at different times. The initial report, typically based on the primary end point, may be published when key planned co-primary or secondary analyses are not yet available. Clinical Trial Up ... Full text Link to item Cite

A Single Arm Phase 2 Trial of Trametinib in Patients With Locally Advanced or Metastatic Epithelioid Hemangioendothelioma.

Journal Article Clin Cancer Res · March 6, 2024 PURPOSE: Epithelioid hemangioendothelioma (EHE) is a rare vascular cancer with pathogenic TAZ-CAMTA1 operating as an oncogenic driver through activation of MAPK pathway. Trametinib is an inhibitor of MEK, a critical kinase in the MAPK pathway. We sought to ... Full text Link to item Cite

Consensus recommendations in the management of Ewing sarcoma from the National Ewing Sarcoma Tumor Board.

Journal Article Cancer · November 1, 2023 Ewing sarcoma (ES) is a malignant tumor of bone and soft tissue that most often occurs in adolescents and young adults. Despite an international coordinated approach, several nuances, discrepancies, and debates remain in defining the standard of care for t ... Full text Link to item Cite

Atezolizumab for Advanced Alveolar Soft Part Sarcoma.

Journal Article N Engl J Med · September 7, 2023 BACKGROUND: Alveolar soft part sarcoma (ASPS) is a rare soft-tissue sarcoma with a poor prognosis and no established therapy. Recently, encouraging responses to immune checkpoint inhibitors have been reported. METHODS: We conducted an investigator-initiate ... Full text Link to item Cite

Emerging predictive biomarkers in the management of bone and soft tissue sarcomas.

Journal Article Expert Rev Anticancer Ther · May 2023 INTRODUCTION: Soft tissue and bone sarcomas are a heterogeneous group of malignancies, each with a unique biology and clinical course. As our understanding of individual sarcoma subtypes and their molecular landscapes increases, predictive biomarkers are e ... Full text Link to item Cite

Bad to the Bone: Emerging Approaches to Aggressive Bone Sarcomas.

Journal Article Am Soc Clin Oncol Educ Book · May 2023 Bone sarcomas are rare heterogeneous tumors that affect patients of all ages including children, adolescent young adults, and older adults. They include many aggressive subtypes and patient groups with poor outcomes, poor access to clinical trials, and lac ... Full text Link to item Cite

Nirogacestat, a γ-Secretase Inhibitor for Desmoid Tumors.

Journal Article N Engl J Med · March 9, 2023 BACKGROUND: Desmoid tumors are rare, locally aggressive, highly recurrent soft-tissue tumors without approved treatments. METHODS: We conducted a phase 3, international, double-blind, randomized, placebo-controlled trial of nirogacestat in adults with prog ... Full text Link to item Cite

A phase II trial of regorafenib in patients with advanced Ewing sarcoma and related tumors of soft tissue and bone: SARC024 trial results.

Journal Article Cancer Med · January 2023 BACKGROUND: Regorafenib is one of several FDA-approved cancer therapies targeting multiple tyrosine kinases. However, there are few subtype-specific data regarding kinase inhibitor activity in sarcomas. We report results of a single arm, phase II trial of ... Full text Link to item Cite

Evolution in Management of Upfront Treatment For Desmoid Tumors

Conference ANNALS OF SURGICAL ONCOLOGY · 2023 Cite

Bone Sarcomas

Chapter · January 1, 2023 This chapter provides answers to practice-based questions with case studies covering the new principles of diagnosis, classification, staging, treatment, and outcomes in the rapidly advancing field of bone sarcomas. Bone sarcomas are rare, mesenchymal neop ... Full text Cite

NCCN Guidelines® Insights: Gastrointestinal Stromal Tumors, Version 2.2022.

Journal Article J Natl Compr Canc Netw · November 2022 Gastrointestinal stromal tumors (GIST) are the most common type of soft tissue sarcoma that occur throughout the gastrointestinal tract. Most of these tumors are caused by oncogenic activating mutations in the KIT or PDGFRA genes. The NCCN Guidelines for G ... Full text Link to item Cite

Onset and resolution of ovarian toxicity with nirogacestat treatment in females with desmoid tumors: Updated safety analyses from the DeFi phase 3 study.

Journal Article Cancer · May 4, 2024 INTRODUCTION: Nirogacestat is a targeted gamma secretase inhibitor approved in the United States for adults with progressing desmoid tumors. In the phase 3 DeFi study (NCT03785964) of nirogacestat, ovarian toxicity (OT) was identified as a safety signal am ... Full text Link to item Cite

Phase II Trial of nab-Sirolimus in Patients With Advanced Malignant Perivascular Epithelioid Cell Tumors (AMPECT): Long-Term Efficacy and Safety Update.

Journal Article J Clin Oncol · May 1, 2024 Clinical trials frequently include multiple end points that mature at different times. The initial report, typically based on the primary end point, may be published when key planned co-primary or secondary analyses are not yet available. Clinical Trial Up ... Full text Link to item Cite

A Single Arm Phase 2 Trial of Trametinib in Patients With Locally Advanced or Metastatic Epithelioid Hemangioendothelioma.

Journal Article Clin Cancer Res · March 6, 2024 PURPOSE: Epithelioid hemangioendothelioma (EHE) is a rare vascular cancer with pathogenic TAZ-CAMTA1 operating as an oncogenic driver through activation of MAPK pathway. Trametinib is an inhibitor of MEK, a critical kinase in the MAPK pathway. We sought to ... Full text Link to item Cite

Consensus recommendations in the management of Ewing sarcoma from the National Ewing Sarcoma Tumor Board.

Journal Article Cancer · November 1, 2023 Ewing sarcoma (ES) is a malignant tumor of bone and soft tissue that most often occurs in adolescents and young adults. Despite an international coordinated approach, several nuances, discrepancies, and debates remain in defining the standard of care for t ... Full text Link to item Cite

Atezolizumab for Advanced Alveolar Soft Part Sarcoma.

Journal Article N Engl J Med · September 7, 2023 BACKGROUND: Alveolar soft part sarcoma (ASPS) is a rare soft-tissue sarcoma with a poor prognosis and no established therapy. Recently, encouraging responses to immune checkpoint inhibitors have been reported. METHODS: We conducted an investigator-initiate ... Full text Link to item Cite

Emerging predictive biomarkers in the management of bone and soft tissue sarcomas.

Journal Article Expert Rev Anticancer Ther · May 2023 INTRODUCTION: Soft tissue and bone sarcomas are a heterogeneous group of malignancies, each with a unique biology and clinical course. As our understanding of individual sarcoma subtypes and their molecular landscapes increases, predictive biomarkers are e ... Full text Link to item Cite

Bad to the Bone: Emerging Approaches to Aggressive Bone Sarcomas.

Journal Article Am Soc Clin Oncol Educ Book · May 2023 Bone sarcomas are rare heterogeneous tumors that affect patients of all ages including children, adolescent young adults, and older adults. They include many aggressive subtypes and patient groups with poor outcomes, poor access to clinical trials, and lac ... Full text Link to item Cite

Nirogacestat, a γ-Secretase Inhibitor for Desmoid Tumors.

Journal Article N Engl J Med · March 9, 2023 BACKGROUND: Desmoid tumors are rare, locally aggressive, highly recurrent soft-tissue tumors without approved treatments. METHODS: We conducted a phase 3, international, double-blind, randomized, placebo-controlled trial of nirogacestat in adults with prog ... Full text Link to item Cite

A phase II trial of regorafenib in patients with advanced Ewing sarcoma and related tumors of soft tissue and bone: SARC024 trial results.

Journal Article Cancer Med · January 2023 BACKGROUND: Regorafenib is one of several FDA-approved cancer therapies targeting multiple tyrosine kinases. However, there are few subtype-specific data regarding kinase inhibitor activity in sarcomas. We report results of a single arm, phase II trial of ... Full text Link to item Cite

Evolution in Management of Upfront Treatment For Desmoid Tumors

Conference ANNALS OF SURGICAL ONCOLOGY · 2023 Cite

Bone Sarcomas

Chapter · January 1, 2023 This chapter provides answers to practice-based questions with case studies covering the new principles of diagnosis, classification, staging, treatment, and outcomes in the rapidly advancing field of bone sarcomas. Bone sarcomas are rare, mesenchymal neop ... Full text Cite

NCCN Guidelines® Insights: Gastrointestinal Stromal Tumors, Version 2.2022.

Journal Article J Natl Compr Canc Netw · November 2022 Gastrointestinal stromal tumors (GIST) are the most common type of soft tissue sarcoma that occur throughout the gastrointestinal tract. Most of these tumors are caused by oncogenic activating mutations in the KIT or PDGFRA genes. The NCCN Guidelines for G ... Full text Link to item Cite

Results of NC-6300 (Nanoparticle Epirubicin) in an Expansion Cohort of Patients with Angiosarcoma.

Journal Article Oncologist · October 1, 2022 BACKGROUND: NC-6300 is a novel epirubicin (EPI) drug conjugated polymeric micelle developed using cutting-edge micellar nanoparticle technology. The nanoparticle epirubicin conjugates EPI to a polymer via a pH-sensitive linker which enables the selective E ... Full text Link to item Cite

Evolving strategies for management of desmoid tumor.

Journal Article Cancer · August 15, 2022 Desmoid tumors (DTs) are rare soft tissue mesenchymal neoplasms that may be associated with impairments, disfigurement, morbidity, and (rarely) mortality. DT disease course can be unpredictable. Most DTs are sporadic, harboring somatic mutations in the gen ... Full text Link to item Cite

Selinexor in Advanced, Metastatic Dedifferentiated Liposarcoma: A Multinational, Randomized, Double-Blind, Placebo-Controlled Trial.

Journal Article J Clin Oncol · August 1, 2022 PURPOSE: Antitumor activity in preclinical models and a phase I study of patients with dedifferentiated liposarcoma (DD-LPS) was observed with selinexor. We evaluated the clinical benefit of selinexor in patients with previously treated DD-LPS whose sarcom ... Full text Link to item Cite

Soft Tissue Sarcoma, Version 2.2022, NCCN Clinical Practice Guidelines in Oncology.

Journal Article J Natl Compr Canc Netw · July 2022 Soft tissue sarcomas (STS) are rare malignancies of mesenchymal cell origin that display a heterogenous mix of clinical and pathologic characteristics. STS can develop from fat, muscle, nerves, blood vessels, and other connective tissues. The evaluation an ... Full text Link to item Cite

Identified Enrollment Challenges of Adolescent and Young Adult Patients on the Nonchemotherapy Arm of Children's Oncology Group Study ARST1321.

Journal Article J Adolesc Young Adult Oncol · June 2022 ARST1321, a trial of patients with advanced soft tissue sarcoma, was the first National Clinical Trials Network study codeveloped by pediatric and adult consortia with two treatment cohorts. We report on the findings of a survey to identify barriers to enr ... Full text Link to item Cite

Phase II Randomized Study of CMB305 and Atezolizumab Compared With Atezolizumab Alone in Soft-Tissue Sarcomas Expressing NY-ESO-1.

Journal Article J Clin Oncol · April 20, 2022 PURPOSE: CMB305 is a heterologous prime-boost vaccination regimen created to prime NY-ESO-1-specific CD8 T-cell populations and then activate the immune response with a potent TLR-4 agonist. This open-label randomized phase II trial was designed to investi ... Full text Link to item Cite

Dual-Energy CT Vital Iodine Tumor Burden for Response Assessment in Patients With Metastatic GIST Undergoing TKI Therapy: Comparison With Standard CT and FDG PET/CT Criteria.

Journal Article AJR Am J Roentgenol · April 2022 BACKGROUND. CT-based criteria for assessing the gastrointestinal stromal tumor (GIST) response to tyrosine kinase inhibitor (TKI) therapy are limited in part because tumor attenuation is influenced by treatment-related changes including hemorrhage and calc ... Full text Link to item Cite

nab-Sirolimus for Patients With Malignant Perivascular Epithelioid Cell Tumors.

Journal Article J Clin Oncol · November 20, 2021 PURPOSE: Malignant perivascular epithelioid cell tumor (PEComa) is a rare aggressive sarcoma, with no approved treatment. To our knowledge, this phase II, single-arm, registration trial is the first prospective clinical trial in this disease, investigating ... Full text Link to item Cite

Health-related quality of life and pain with selinexor in patients with advanced dedifferentiated liposarcoma.

Journal Article Future Oncol · August 2021 Objective: Compare health-related quality of life (HRQoL) of selinexor versus placebo in patients with dedifferentiated liposarcoma. Materials & methods: HRQoL was assessed at baseline and day 1 of each cycle using the European Organization for Research an ... Full text Link to item Cite

Large scale multiomic analysis suggests mechanisms of resistance to immunotherapy in leiomyosarcoma.

Conference Journal of Clinical Oncology · May 20, 2021 11512 Background: Leiomyosarcomas (LMS) have been reported to have immunohistochemical (IHC) and gene expression signatures suggestive of an immune-responsive tumor microenvironment. Despite this, immune checkpoint inhibitors hav ... Full text Cite

Multiomic analysis to reveal distinct molecular profiles of uterine and nonuterine leiomyosarcoma.

Conference Journal of Clinical Oncology · May 20, 2021 11555 Background: Leiomyosarcoma (LMS) is a rare group of mesenchymal malignancies found in the uterus, retroperitoneum, skin, or other soft-tissue sites. Treatment for LMS is extrapolated from trials including both uterine (uLMS ... Full text Cite

Phase II study of atezolizumab in advanced alveolar soft part sarcoma (ASPS).

Conference Journal of Clinical Oncology · May 20, 2021 11519 Background: ASPS constitutes < 1% of soft tissue sarcomas and frequently presents in adolescents and young adults. There are no approved therapies for ASPS. We are currently evaluating the clinical activity of atezolizum ... Full text Cite

P10015/SARC033: A phase 2 trial of trametinib in patients with advanced epithelioid hemangioendothelioma (EHE).

Conference Journal of Clinical Oncology · May 20, 2021 11503 Background: EHE is a rare vascular cancer arising in liver, lung, soft tissue and bone. The natural history of metastatic disease varies considerably from indolent growth over years to rapid growth with fatal outcome in mon ... Full text Cite

Results of NC-6300 (nanoparticle epirubicin) in an expansion cohort of patients with angiosarcoma.

Conference Journal of Clinical Oncology · May 20, 2021 11543 Background: NC-6300 is a polymeric micelle exhibiting increased tumor accumulation compared to small-molecule epirubicin through enhanced pharmacokinetics and controlled release within the tumor through a pH-sensitive linke ... Full text Cite

TK216 for relapsed/refractory Ewing sarcoma: Interim phase 1/2 results.

Conference Journal of Clinical Oncology · May 20, 2021 11500 Background: Ewing Sarcoma (ES) is a rare cancer of the young with very few treatment options in the relapsed/refractory (R/R) setting. Fusions of the EWS gene and one of five different ETS transcription factors are dominant ... Full text Cite

Optimal Avapritinib Treatment Strategies for Patients with Metastatic or Unresectable Gastrointestinal Stromal Tumors.

Journal Article Oncologist · April 2021 BACKGROUND: Avapritinib, a novel inhibitor of KIT/PDGFRA, is approved in the U.S. for the treatment of adults with PDGFRA exon 18-mutant unresectable or metastatic gastrointestinal stromal tumors (U/M GISTs). We assessed the safety of avapritinib and provi ... Full text Link to item Cite

Recent advances in the understanding and management of liposarcoma.

Journal Article Fac Rev · 2021 Liposarcomas are a common subfamily of soft tissue sarcoma with several subtypes recognized by the World Health Organization: atypical lipomatous tumors (ALT)/well-differentiated liposarcoma (WDLPS), dedifferentiated liposarcoma (DDLPS), myxoid liposarcoma ... Full text Link to item Cite

NCCN Guidelines Insights: Soft Tissue Sarcoma, Version 1.2021.

Journal Article J Natl Compr Canc Netw · December 2, 2020 The NCCN Guidelines for Soft Tissue Sarcoma provide recommendations for the diagnosis, evaluation, treatment, and follow-up for patients with soft tissue sarcomas. These NCCN Guidelines Insights summarize the panel discussion behind recent important update ... Full text Link to item Cite

A Randomized, Double-Blind, Placebo-Controlled, Phase II Study of Regorafenib Versus Placebo in Advanced/Metastatic, Treatment-Refractory Liposarcoma: Results from the SARC024 Study.

Journal Article Oncologist · November 2020 LESSONS LEARNED: The results from the liposarcoma cohort of SARC024 confirm previously published data and do not support the routine use of regorafenib in this patient population. Continued exploration of novel therapies, including combination approaches, ... Full text Link to item Cite

A Phase 1b Dose Escalation Trial of NC-6300 (Nanoparticle Epirubicin) in Patients with Advanced Solid Tumors or Advanced, Metastatic, or Unresectable Soft-tissue Sarcoma.

Journal Article Clin Cancer Res · August 15, 2020 PURPOSE: NC-6300 is a novel nanoparticle formulation of epirubicin that has a pH-sensitive linker conjugated to epirubicin. It exhibits selective tumor accumulation owing to enhanced permeability and retention effect. We conducted a phase 1b trial to deter ... Full text Link to item Cite

Correlative Analyses of the SARC028 Trial Reveal an Association Between Sarcoma-Associated Immune Infiltrate and Response to Pembrolizumab.

Journal Article Clin Cancer Res · March 15, 2020 PURPOSE: We recently reported a 17.5% objective RECIST 1.1 response rate in a phase II study of pembrolizumab in patients with advanced sarcoma (SARC028). The majority of responses occurred in undifferentiated pleomorphic sarcoma (UPS) and dedifferentiated ... Full text Link to item Cite

Academic-Practice Partnership for Caregiver Training and Support: The Duke Elder Family/Caregiver Training (DEFT) Center.

Journal Article N C Med J · 2020 BACKGROUND After a hospital stay, many older adults rely on their caregivers for assistance at home. Empirical evidence demonstrates that caregiver support programs in hospital-to-home transitions are associated with favorable caregiver and patient outcome ... Full text Link to item Cite

Randomized Double-Blind Phase II Study of Regorafenib in Patients With Metastatic Osteosarcoma.

Journal Article J Clin Oncol · June 1, 2019 PURPOSE: SARC024 is a phase II clinical trial of the multikinase inhibitor regorafenib in specific sarcoma subtypes, including advanced osteosarcoma. We hypothesized that regorafenib would improve progression-free survival (PFS) in patients with sarcoma an ... Full text Link to item Cite

Assessing the Impact of a Novel Integrated Palliative Care and Medical Oncology Inpatient Service on Health Care Utilization before Hospice Enrollment.

Journal Article J Palliat Med · April 2019 BACKGROUND: Evidence increasingly supports the integration of specialist palliative care (PC) into routine cancer care. A novel, fully integrated PC and medical oncology inpatient service was developed at Duke University Hospital in 2011. OBJECTIVE: To ass ... Full text Link to item Cite

The experiences of older caregivers of cancer patients following hospital discharge.

Journal Article Support Care Cancer · February 2019 PURPOSE: This study addressed the experiences of older caregivers of cancer patients in the 2 weeks following a hospital discharge. It sought to understand the challenges they face in providing supportive care to patients at home. METHODS: Qualitative desc ... Full text Link to item Cite

Recurrent Extradural Myxopapillary Ependymoma With Oligometastatic Spread.

Journal Article Front Oncol · 2019 Myxopapillary ependymomas are a slow-growing, grade I type glial tumor in the lumbosacral region. More rarely, they can present as extradural, subcutaneous sacrococcygeal, or perisacral masses, and it is under these circumstances that they are more likely ... Full text Link to item Cite

Sorafenib for Advanced and Refractory Desmoid Tumors.

Journal Article N Engl J Med · December 20, 2018 BACKGROUND: Desmoid tumors (also referred to as aggressive fibromatosis) are connective tissue neoplasms that can arise in any anatomical location and infiltrate the mesentery, neurovascular structures, and visceral organs. There is no standard of care. ME ... Full text Link to item Cite

Systemic Anti-Cancer Therapy in Synovial Sarcoma: A Systematic Review.

Journal Article Cancers (Basel) · November 1, 2018 Synovial sarcoma (SS) is an aggressive malignancy which accounts for approximately 5⁻10% of all soft-tissue sarcomas. SS has pathologic and genomic characteristics that define it as a distinct subtype of soft tissue sarcoma (STS). STS subtypes continue to ... Full text Link to item Cite

Rationale and emerging strategies for immune checkpoint blockade in soft tissue sarcoma.

Journal Article Cancer · October 1, 2018 Soft tissue sarcomas (STS) are heterogeneous, mesenchymal malignancies with variable biologic behavior. The primary management for localized STS is surgical resection, which may be combined with neoadjuvant or adjuvant radiation therapy to increase the pro ... Full text Link to item Cite

A Phase I Trial of the IGF-1R Antibody Ganitumab (AMG 479) in Combination with Everolimus (RAD001) and Panitumumab in Patients with Advanced Cancer.

Journal Article Oncologist · July 2018 PURPOSE: This study evaluated the maximum tolerated dose or recommended phase II dose (RPTD) and safety and tolerability of the ganitumab and everolimus doublet regimen followed by the ganitumab, everolimus, and panitumumab triplet regimen. MATERIALS AND M ... Full text Link to item Cite

Soft Tissue Sarcoma, Version 2.2018, NCCN Clinical Practice Guidelines in Oncology.

Journal Article J Natl Compr Canc Netw · May 2018 Soft tissue sarcomas (STS) are rare solid tumors of mesenchymal cell origin that display a heterogenous mix of clinical and pathologic characteristics. STS can develop from fat, muscle, nerves, blood vessels, and other connective tissues. The evaluation an ... Full text Link to item Cite

Making progress in a rare disease: emerging therapeutics in soft tissue sarcomas.

Journal Article F1000Res · 2018 Sarcomas are rare tumors derived from mesenchymal connective tissues in the body. Because there are well over 50 histologic sarcoma subtypes, including malignant and non-malignant pathologies, clinical courses and therapeutic management are widely divergen ... Full text Link to item Cite

Comprehensive and Integrated Genomic Characterization of Adult Soft Tissue Sarcomas.

Journal Article Cell · November 2, 2017 Sarcomas are a broad family of mesenchymal malignancies exhibiting remarkable histologic diversity. We describe the multi-platform molecular landscape of 206 adult soft tissue sarcomas representing 6 major types. Along with novel insights into the biology ... Full text Link to item Cite

Pembrolizumab in advanced soft-tissue sarcoma and bone sarcoma (SARC028): a multicentre, two-cohort, single-arm, open-label, phase 2 trial.

Journal Article The Lancet. Oncology · November 2017 BackgroundPatients with advanced sarcomas have a poor prognosis and few treatment options that improve overall survival. Chemotherapy and targeted therapies offer short-lived disease control. We assessed pembrolizumab, an anti-PD-1 antibody, for s ... Full text Cite

Improvements in Patient and Health System Outcomes Using an Integrated Oncology and Palliative Medicine Approach on a Solid Tumor Inpatient Service.

Journal Article J Oncol Pract · September 2017 PURPOSE: Early palliative care (PC) improves outcomes for outpatients with advanced cancer. Its effect on hospitalized patients with cancer is unknown. Herein, we report on the influence of a novel, fully integrated inpatient medical oncology and PC partne ... Full text Link to item Cite

Doxorubicin plus evofosfamide versus doxorubicin alone in locally advanced, unresectable or metastatic soft-tissue sarcoma (TH CR-406/SARC021): an international, multicentre, open-label, randomised phase 3 trial.

Journal Article Lancet Oncol · August 2017 BACKGROUND: Evofosfamide is a hypoxia-activated prodrug of bromo-isophosphoramide mustard. We aimed to assess the benefit of adding evofosfamide to doxorubicin as first-line therapy for advanced soft-tissue sarcomas. METHODS: We did this international, ope ... Full text Link to item Cite

Multicenter phase II study of pembrolizumab (P) in advanced soft tissue (STS) and bone sarcomas (BS): Final results of SARC028 and biomarker analyses.

Conference Journal of Clinical Oncology · May 20, 2017 11008 Background: SARC028 is the first multicenter Phase II study of P monotherapy in patients (pts) with STS and BS. Designed to detect clinical efficacy signals in multiple histologies, the study collected blood & tissue sa ... Full text Cite

A phase II trial of regorafenib (REGO) in patients (pts) with advanced Ewing sarcoma and related tumors (EWS) of soft tissue and bone: SARC024 trial results.

Conference Journal of Clinical Oncology · May 20, 2017 11005 Background: Pazopanib is approved for soft tissue sarcoma pts after failure of other therapy, but there are few subtype-specific data regarding kinase inhibitor activity. We report on a single arm, phase II trial of REGO in ... Full text Cite

Phase I study of pazopanib plus TH-302 in advanced solid tumors.

Journal Article Cancer Chemother Pharmacol · March 2017 PURPOSE: To define the maximum tolerated dose (MTD), recommended phase II dose (RPTD), and assess safety and tolerability for the combination of pazopanib plus TH-302, an investigational hypoxia-activated prodrug (HAP), in adult patients with advanced soli ... Full text Link to item Cite

Correlation of circulating PD-L2 levels with outcomes of therapy with the anti-PD-1 antibody pembrolizumab (P) in patients (pts) with advanced soft tissue sarcomas (STS): Biomarker analysis of SARC028.

Conference Journal of Clinical Oncology · March 1, 2017 60 Background: The activity of the anti-PD-1 antibody P in pts with advanced STS was evaluated in a prospective multicenter Phase II study conducted by the Sarcoma Alliance for Research through Collaboration (SARC). SARC028 enrol ... Full text Cite

Healthcare utilization among cancer patients prior to hospice

Conference JOURNAL OF CLINICAL ONCOLOGY · October 9, 2016 Link to item Cite

Dose-escalation study of a second-generation non-ansamycin HSP90 inhibitor, onalespib (AT13387), in combination with imatinib in patients with metastatic gastrointestinal stromal tumour.

Journal Article Eur J Cancer · July 2016 BACKGROUND: Gastrointestinal stromal tumours (GIST) treated with the tyrosine kinase inhibitor (TKI) imatinib can become resistant when additional mutations in the receptor tyrosine kinases KIT or PDGFRA block imatinib activity. Mutated KIT requires the mo ... Full text Link to item Cite

Soft Tissue Sarcoma, Version 2.2016, NCCN Clinical Practice Guidelines in Oncology.

Journal Article J Natl Compr Canc Netw · June 2016 Soft tissue sarcomas (STS) are rare solid tumors of mesenchymal cell origin that display a heterogenous mix of clinical and pathologic characteristics. STS can develop from fat, muscle, nerves, blood vessels, and other connective tissues. The evaluation an ... Full text Link to item Cite

A mouse-human phase 1 co-clinical trial of a protease-activated fluorescent probe for imaging cancer.

Journal Article Sci Transl Med · January 6, 2016 Local recurrence is a common cause of treatment failure for patients with solid tumors. Intraoperative detection of microscopic residual cancer in the tumor bed could be used to decrease the risk of a positive surgical margin, reduce rates of reexcision, a ... Full text Link to item Cite

Effects of enhanced caregiver training program on cancer caregiver's self-efficacy, preparedness, and psychological well-being.

Journal Article Support Care Cancer · January 2016 PURPOSE: We examined the effects of an enhanced informal caregiver training (Enhanced-CT) protocol in cancer symptom and caregiver stress management to caregivers of hospitalized cancer patients. METHODS: We recruited adult patients in oncology units and t ... Full text Link to item Cite

Abstract SY36-03: Intraoperative molecular imaging with protease-activated fluorescent imaging agents

Conference Cancer Research · August 1, 2015 AbstractIntra-operative detection of residual cancer in the tumor bed can be used to decrease the risk of a positive surgical margin, reduce the rate of re-excision, and tailor adjuvant therapy. LUM015 is a ... Full text Cite

Rhabdomyosarcomatous Transformation of a Gastrointestinal Stromal Tumor following Treatment with Imatinib.

Journal Article Case Rep Oncol Med · 2015 Rhabdomyosarcomatous dedifferentiation of GIST following tyrosine kinase inhibitor (TKI) therapy is rare, with only a handful of cases previously reported in the literature. Herein we present a case of metastatic GIST initially treated with imatinib that d ... Full text Link to item Cite

Chemotherapy in soft-tissue sarcoma: where do we go from here?

Journal Article Oncology (Williston Park) · January 2015 Link to item Cite

Chemotherapy in soft-tissue sarcoma: where do we go from here?

Journal Article Oncology (Williston Park, N.Y.) · January 1, 2015 Cite

Chemotherapy in soft-tissue sarcoma: where do we go from here?

Journal Article Oncology (Williston Park, N.Y.) · January 1, 2015 Cite

Early palliative care on an inpatient oncology unit: Impact of a novel co-rounding partnership on patient and health system outcomes.

Journal Article J Clin Oncol · November 2014 3 Background: Early palliative care (PC) improves outcomes for outpatients with advanced cancer, but its impact on an inpatient oncology unit is unknown. We implemented a novel inpatient medical oncology (ONC) and PC co-rounding partnership on September 1, ... Full text Link to item Cite

Bone Sarcomas

Chapter · June 20, 2014 This chapter presents a series of case studies with questions related to bone sarcomas. The first case study is about a 17-year-old Caucasian male diagnosed with Ewing sarcoma of the left humerus. Other case studies include a 52-year-old Caucasian male wit ... Full text Cite

Gastrointestinal stromal tumors, version 2.2014.

Journal Article J Natl Compr Canc Netw · June 2014 Gastrointestinal stromal tumors (GIST) are the most common soft tissue sarcoma of the gastrointestinal tract, resulting most commonly from KIT or platelet-derived growth factor receptor α (PDGFRα)-activating mutations. These NCCN Guideline Insights highlig ... Full text Link to item Cite

Soft tissue sarcoma, version 2.2014.

Journal Article J Natl Compr Canc Netw · April 2014 These NCCN Guidelines Insights highlight the important updates to the NCCN Guidelines for Soft Tissue Sarcoma (STS) specific to the role of radiation therapy in the management of patients with retroperitoneal/intra-abdominal STS. The guidelines have also i ... Full text Link to item Cite

Bone sarcomas

Chapter · 2014 Cite

Abstract C61: Phase I Study of pazopanib in combination with the investigational hypoxia-targeted drug TH-302.

Journal Article Molecular Cancer Therapeutics · November 1, 2013 AbstractBackground: Preclinical data demonstrate anti-VEGF agents can induce hypoxia, which may mediate resistance and induce increased tumor invasiveness. TH-302 is an investigational hypoxia-targeted drug; ... Full text Cite

Practical radiation oncology for extremity sarcomas.

Journal Article Surg Oncol Clin N Am · July 2013 Soft tissue sarcomas are rare cancers. They should be managed by a multidisciplinary team with experience caring for these diverse malignancies. Local control is frequently achieved with a combination of radiation therapy and surgery. This article reviews ... Full text Link to item Cite

Results of an international randomized phase III trial of the mammalian target of rapamycin inhibitor ridaforolimus versus placebo to control metastatic sarcomas in patients after benefit from prior chemotherapy.

Journal Article J Clin Oncol · July 1, 2013 PURPOSE: Aberrant mammalian target of rapamycin (mTOR) signaling is common in sarcomas and other malignancies. Drug resistance and toxicities often limit benefits of systemic chemotherapy used to treat metastatic sarcomas. This large randomized placebo-con ... Full text Link to item Cite

Soft tissue sarcoma, version 2.2012: featured updates to the NCCN guidelines.

Journal Article J Natl Compr Canc Netw · August 2012 The major changes to the 2012 and 2011 NCCN Guidelines for Soft Tissue Sarcoma pertain to the management of patients with gastrointestinal stromal tumors (GISTs) and desmoid tumors (aggressive fibromatosis). Postoperative imatinib following complete resect ... Full text Link to item Cite

A phase I study of bevacizumab, everolimus and panitumumab in advanced solid tumors.

Journal Article Cancer Chemother Pharmacol · July 2012 PURPOSE: Preclinical data suggest concurrent inhibition of VEGF, mTOR and EGFR pathways may augment antitumor and antiangiogenic effects compared to inhibition of each pathway alone. This study evaluated the maximum tolerated dose/recommended phase II dose ... Full text Link to item Cite

Systemic therapy for advanced soft tissue sarcomas: highlighting novel therapies and treatment approaches.

Journal Article Cancer · March 15, 2012 Featured Publication Soft tissue sarcomas (STS) are a rare, heterogeneous group of solid tumors in need of improved therapeutic options. First-line chemotherapy is considered the current standard of care for patients with advanced, symptomatic STS, but the median survival is o ... Full text Link to item Cite

Targeted therapy in sarcoma: should we be lumpers or splitters?

Journal Article Am Soc Clin Oncol Educ Book · 2012 The identification of KIT as a critical driver in the pathogenesis of GI stromal tumor (GIST), and its subsequent inhibition with imatinib, have resulted in tremendous efforts to identify other potential therapeutic targets for the heterogeneous group of d ... Full text Link to item Cite

Efficacy of phosphatidylinositol-3 kinase inhibitors in a primary mouse model of undifferentiated pleomorphic sarcoma.

Journal Article Sarcoma · 2012 Recent advances in sarcoma genomics have identified novel mutations in the PI3K pathway in human sarcomas. Here, we use a mouse model of primary soft-tissue sarcoma for preclinical testing of doxorubicin and inhibitors of the PI3K pathway: BKM120 (PI3K inh ... Full text Cite

Targeted agents for sarcoma: is individualized therapy possible in such a diverse tumor type?

Journal Article Semin Oncol · October 2011 A wide variety of cytogenetic abnormalities and molecular pathways have been implicated in the pathogenesis of sarcoma, and significant progress has been made in the past decade toward identifying potential therapeutic targets. However, apart from gastroin ... Full text Link to item Cite

Emerging therapeutic targets for soft tissue sarcoma.

Journal Article Curr Oncol Rep · August 2011 Featured Publication Soft tissue sarcomas (STS) are malignancies of mesenchymal origin that represent approximately 1% of cancers in adults. Systematic research into the treatment of STS is challenging given its rarity and disease heterogeneity. Despite the ability to histolog ... Full text Link to item Cite

Results of the phase III, placebo-controlled trial (SUCCEED) evaluating the mTOR inhibitor ridaforolimus (R) as maintenance therapy in advanced sarcoma patients (pts) following clinical benefit from prior standard cytotoxic chemotherapy (CT).

Journal Article J Clin Oncol · May 20, 2011 10005 Background: The mTOR signaling pathway is activated in most sarcomas. R, an oral mTOR inhibitor, demonstrated activity in phase I-II trials in advanced sarcomas following failure of prior CT. METHODS: This trial is an international, double-blind stud ... Link to item Cite

Primary meningeal rhabdomyosarcoma.

Journal Article Sarcoma · 2011 Featured Publication Primary meningeal rhabdomyosarcoma is a rare primary brain malignancy, with scant case reports. While most reports of primary intracranial rhabdomyosarcoma occur in pediatric patients, a handful of cases in adult patients have been reported in the medical ... Full text Link to item Cite

Soft tissue sarcoma.

Journal Article J Natl Compr Canc Netw · June 2010 Featured Publication Full text Link to item Cite

NCCN Task Force report: update on the management of patients with gastrointestinal stromal tumors.

Journal Article J Natl Compr Canc Netw · April 2010 Featured Publication The standard of care for managing patients with gastrointestinal stromal tumors (GISTs) rapidly changed after the introduction of effective molecularly targeted therapies involving tyrosine kinase inhibitors (TKIs), such as imatinib mesylate and sunitinib ... Full text Link to item Cite

Cross species genomic analysis identifies a mouse model as undifferentiated pleomorphic sarcoma/malignant fibrous histiocytoma.

Journal Article PLoS One · November 30, 2009 Featured Publication Undifferentiated pleomorphic sarcoma/Malignant Fibrous Histiocytoma (MFH) is one of the most common subtypes of human soft tissue sarcoma. Using cross species genomic analysis, we define a geneset from the LSL-Kras(G12D); Trp53(Flox/Flox) mouse model of so ... Full text Link to item Cite

Bevacizumab (B) plus everolimus (E) and panitumumab (P) in refractory advanced solid tumors.

Journal Article J Clin Oncol · May 20, 2009 3551 Background: In preclinical models, VEGF, mTOR, and EGFR inhibitors have anti-tumor and anti-angiogenesis effects as monotherapies and in combination. B inhibits VEGF; E inhibits mTOR; P inhibits EGFR. There is also potential for interaction between th ... Link to item Cite

The clinical management of chondrosarcoma.

Journal Article Curr Treat Options Oncol · April 2009 Featured Publication Chondrosarcomas (CHS) represent a heterogeneous group of disorders ranging from indolent, low-grade tumors to aggressive, high-grade forms. Surgical resection represents the primary and preferred treatment modality for individuals with localized disease. R ... Full text Link to item Cite

Characterizing the developmental pathways TTF-1, NKX2-8, and PAX9 in lung cancer.

Journal Article Proc Natl Acad Sci U S A · March 31, 2009 We investigated the clinical implications of lung developmental transcription factors (TTF-1, NKX2-8, and PAX9) that we recently discovered as cooperating oncogenes activated by way of gene amplification at chromosome 14q13 in lung cancer. Using stable tra ... Full text Link to item Cite

A genomic approach to identify molecular pathways associated with chemotherapy resistance.

Journal Article Mol Cancer Ther · October 2008 Featured Publication Resistance to chemotherapy in cancer is common. As gene expression profiling has been shown to anticipate chemotherapeutic resistance, we sought to identify cellular pathways associated with resistance to facilitate effective combination therapy. Gene set ... Full text Link to item Cite

Use of co-activation of lung cancer specific developmental pathway genes, TTF-1, NKX2-8, and PAX9, to predict prognosis and guide therapeutic strategies.

Journal Article J Clin Oncol · May 20, 2008 7511 Background: We recently described a novel, recurring amplicon (14q13.3) specific to NSCLC that harbors three genes, TTF- 1, NKX2-8, and PAX9, important in lung development (Kendall et al. PNAS 2007). METHODS: Using stable transfectants of human bronch ... Link to item Cite

A genomic approach to identify therapeutic targets in histologic subtypes of soft tissue sarcoma.

Journal Article J Clin Oncol · May 20, 2008 10577 Background: Sarcomas are a rare, heterogeneous group of tumors with varying degrees of aggressiveness and responsiveness to therapy. In the advanced setting, chemotherapy results in unsatisfactory long-term outcomes. Novel therapeutic strategies that ... Link to item Cite

Pharmacogenomic strategies provide a rational approach to the treatment of cisplatin-resistant patients with advanced cancer.

Journal Article J Clin Oncol · October 1, 2007 Featured Publication PURPOSE: Standard treatment for advanced non-small-cell lung cancer (NSCLC) includes the use of a platinum-based chemotherapy regimen. However, response rates are highly variable. Newer agents, such as pemetrexed, have shown significant activity as second- ... Full text Link to item Cite

A phase II trial of carboplatin/vinorelbine with pegfilgrastim support for the treatment of patients with advanced non-small cell lung cancer.

Journal Article J Thorac Oncol · June 2007 Featured Publication INTRODUCTION: The impact of chemotherapy dose delivery has not been well studied in patients with non-small cell lung cancer (NSCLC). Overlapping hematologic toxicities commonly limit planned dose intensity of combination chemotherapy regimens. A phase II ... Full text Link to item Cite

Genomic signatures to guide the use of chemotherapeutics.

Other Nat Med · November 2006 Using in vitro drug sensitivity data coupled with Affymetrix microarray data, we developed gene expression signatures that predict sensitivity to individual chemotherapeutic drugs. Each signature was validated with response data from an independent set of ... Full text Link to item Cite

Impact of a multidisciplinary thoracic oncology clinic on the timeliness of care.

Journal Article J Thorac Oncol · September 2006 Featured Publication BACKGROUND: Multidisciplinary clinics have been recommended for the evaluation of patients with lung cancer. Evidence to support this recommendation, however, is limited. A single-center, retrospective review of lung cancer patients at a Veterans Affairs h ... Link to item Cite

Thymoma: benign appearance, malignant potential.

Journal Article Oncologist · September 2006 Featured Publication Thymoma is a rare tumor with a largely indolent growth pattern. It does, however, have malignant potential as a result of its ability to invade locally and metastasize regionally. Often associated with a number of immune- and nonimmune-mediated paraneoplas ... Full text Link to item Cite

Epidermal growth factor receptor mutations predict sensitivity to gefitinib in patients with non-small-cell lung cancer.

Journal Article Future Oncol · August 2005 Featured Publication Despite advances in chemotherapeutics, overall survival for advanced lung cancer patients remains poor. Consequently, efforts have focused on the use of targeted therapies to improve response rates and survival. The epidermal growth factor receptor (EGFR) ... Full text Link to item Cite

Small-cell lung cancer: a review of clinical trials.

Journal Article Semin Thorac Cardiovasc Surg · October 2003 Featured Publication Small-cell lung cancer (SCLC) is expected to account for 25% of the approximate 170,000 cases of lung cancer diagnosed in the United States in 2002. Although sensitive and responsive to chemotherapy, SCLC has an increased propensity for early metastases, w ... Full text Link to item Cite

Targeted inactivation of the EGF and amphiregulin genes reveals distinct roles for EGF receptor ligands in mouse mammary gland development.

Journal Article Development · June 1999 Featured Publication Targeted mice lacking functional EGF or amphiregulin (AR) were derived and bred to the TGFalpha-knockout to generate mice lacking various combinations of the three ligands. In contrast to EGF receptor (EGFR) knockout mice, triple null mice lacking half of ... Full text Link to item Cite

Expression of constitutively active Raf-1 in the mitochondria restores antiapoptotic and leukemogenic potential of a transformation-deficient BCR/ABL mutant.

Journal Article J Exp Med · June 15, 1998 Featured Publication The oncogenic BCR/ABL protein protects hematopoietic cells from apoptosis induced by growth factor deprivation, but the mechanisms are only partially understood. A BCR/ABL mutant lacking amino acids 176-426 in the BCR domain (p185DeltaBCR) failed to protec ... Full text Link to item Cite

Characterization of the mouse transforming growth factor alpha gene: its expression during eyelid development and in waved 1 tissues.

Journal Article Cell Growth Differ · September 1996 The spontaneous mouse waved 1 (wa1) mutation is allelic with the transforming growth factor alpha (TGF-alpha) gene and produces phenotypes similar to those of TGF-alpha knockout mice. Here, we show that TGF-alpha mRNA and protein levels are measurable in w ... Link to item Cite