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Stem cell transplants are not frequently offered to older patients with high-risk myelodysplastic syndromes (MDS). According to a study from the Blood and Marrow Transplant Clinical Trials Network (BMTCTN 1102), these patients may indeed achieve a survival benefit from stem cell transplant. As...
In a phase Ib/II study reported in the Journal of Clinical Oncology, David A. Sallman, MD, and colleagues found that the combination of azacitidine and the first-in-class small-molecule agent eprenetapopt produced high response rates in patients with TP53-mutant myelodysplastic syndromes (MDS)....
In a phase III U.S. Intergroup trial reported in the Journal of Clinical Oncology, Alan F. List, MD, and colleagues found that treatment with lenalidomide plus epoetin alfa increased the rate of major erythroid response vs lenalidomide alone in patients with lower-risk, non-del(5q) myelodysplastic...
In the phase II FIGARO trial reported in the Journal of Clinical Oncology, Craddock et al found that intensified reduced-intensity conditioning with fludarabine/amsacrine/cytarabine–busulfan (FLAMSA-Bu) did not improve outcomes following allogeneic stem cell transplantation vs standard...
The BCL2 inhibitor venetoclax can be safely added to standard therapies for some high-risk myeloid blood cancers, and in early studies, the combination showed improved outcomes, according to two reports presented by Jacqueline S. Garcia, MD, and colleagues at the 2020 American Society of Hematology ...
Stem cell transplants are not frequently offered to older patients with high-risk myelodysplastic syndromes (MDS). According to a study from the Blood and Marrow Transplant Clinical Trials Network (BMTCTN 1102), these patients may indeed achieve a survival benefit from stem cell transplant. As...
Corey Cutler, MD, MPH, of Dana-Farber Cancer Institute, discusses results from a multicenter trial that compared reduced-intensity allogeneic hematopoietic cell transplantation to hypomethylating therapy or best supportive care in patients aged 50 to 75 with advanced myelodysplastic syndromes (Abstract 75).
In the phase II portion of the phase II/III MDS3001 study reported in the Journal of Clinical Oncology, David P. Steensma, MD, and colleagues found that imetelstat—a first-in-class competitive inhibitor of telomerase enzymatic activity—markedly reduced the need for red blood cell transfusion in...
Patients with lower-risk myelodysplastic syndromes (MDS) who are dependent on red blood cell transfusions have limited options, especially if they are no longer responding to erythropoiesis-stimulating agents. Research presented during the virtual edition of the 25th European Hematology Association ...
Over the past few weeks, the U.S. Food and Drug Administration (FDA) has issued designations and accepted applications for novel agents, as well as approved companion diagnostics. We summarize these regulatory movements below. Breakthrough Therapy Designation for MK-6482 in von Hippel-Lindau...
Considered the “guardian of the genome,” TP53 is the most commonly mutated gene in patients with cancer. TP53's normal function is to detect DNA damage and prevent cells from passing this damage on to daughter cells. When TP53 is mutated, the protein made from this gene, called p53, can no longer...
In higher-risk myelodysplastic syndromes (MDS), a global phase II open-label study found that the combination of pevonedistat plus azacitidine was associated with a trend toward improved event-free survival and numerically longer overall survival, as compared with azacitidine alone, according to...
On July 7, the U.S. Food and Drug Administration (FDA) approved an oral combination of decitabine and cedazuridine (Inqovi) for adult patients with myelodysplastic syndromes (MDS), including the following: Previously treated and untreated de novo and secondary MDS with the following...
Advances in the treatment of hematologic malignancies and some of their associated symptoms were presented during EHA25 Virtual, an ongoing online conference from the European Hematology Association (EHA). Advances in the Treatment of High-Risk CLL: CLL2-GIVe Results In the CLL2-GIVe trial, the...
Mikkael A. Sekeres, MD, of the Cleveland Clinic, discusses data from a phase II study of pevonedistat plus azacitidine vs azacitidine alone in patients with higher-risk myelodysplastic syndromes, chronic myelomonocytic leukemia, or low-blast acute myeloid leukemia (Abstract 7506).
On April 3, 2020, luspatercept-aamt was approved in the treatment of anemia failing to respond to an erythropoiesis-stimulating agent and requiring 2 or more red blood cell (RBC) units over 8 weeks.1,2 The treatment is geared toward adult patients with very low– to intermediate-risk myelodysplastic ...
The 2019 American Society of Hematology (ASH) Annual Meeting & Exposition featured a cornucopia of sessions. It was impossible to attend all the lectures, symposia, oral presentations, poster presentations, and special events because many were concurrent. Below, we have selected some...
In a special report published by Malcovati et al in the journal Blood, an international working group of experts in myelodysplastic syndromes (MDS) has proposed the recognition of a distinct subtype of MDS based on the presence of a nonheritable genetic mutation that causes the disease. The...
On April 3, the U.S. Food and Drug Administration (FDA) approved luspatercept-aamt (Reblozyl) for the treatment of anemia that fails to respond to an erythropoiesis-stimulating agent and requires two or more red blood cell units over 8 weeks in adult patients with very low- to intermediate-risk...
This week, the U.S. Food and Drug Administration (FDA) granted Priority Review to a treatment for lung and thyroid cancers with a RET fusion or mutation; gave Breakthrough Therapy designation to a doublet therapy for TP53-mutated myelodysplastic syndromes; and issued an update to their...
In the phase III MEDALIST trial reported in The New England Journal of Medicine, Fenaux et al found that luspatercept significantly reduced the severity of anemia vs placebo in red blood cell transfusion–dependent patients with very low- to intermediate-risk myelodysplastic syndromes with ring...
Recently, the U.S. Food and Drug Administration (FDA) granted Priority Reviews for treatments in extensive-stage small cell lung cancer (SCLC), non–muscle invasive bladder cancer, and BRAF V600E–mutant colorectal cancer. The Agency also recently issued multiple Breakthrough Therapy designations and ...
Ilaria Iacobucci, PhD, of St. Jude Children’s Research Hospital, discusses her work to more accurately define mutation subtypes in acute myeloid leukemia and myelodysplastic syndromes, as well as the implications for diagnosis, prognosis, and treatment (Abstract LBA-4 ).
Researchers have completed the first comprehensive analysis combining full genomic sequencing and gene-expression profiles of more than 1,300 patients with acute myeloid leukemia (AML) or myelodysplastic syndrome (MDS). The research was presented by Iacobucci et al during the 2019 American Society...
Mikkael A. Sekeres, MD, of the Cleveland Clinic, discusses results of a phase Ib study of glasdegib in combination with azacitidine, which showed activity in patients with untreated myelodysplastic syndromes, acute myeloid leukemia, and chronic myelomonocytic leukemia who are ineligible for intensive chemotherapy (Abstract 177).
Despite the spate of recent drug approvals in blood cancer, it’s been more than 13 years since the U.S. Food and Drug Administration (FDA) has approved a treatment for myelodysplastic syndromes (MDS), a bone marrow disorder characterized by ineffective hematopoiesis. Nevertheless, data from a...
Rafael Bejar, MD, PhD, of the University of California, San Diego Moores Cancer Center, discusses the tailored nature of MDS treatment, including somatic mutations that could alter treatment decisions, and novel therapies with predictive biomarkers.
Recently, the U.S. Food and Drug Administration (FDA) granted Fast Track designation to magrolimab in myelodysplastic syndrome and acute myeloid leukemia (AML). The agency also granted Breakthrough Therapy designations in lung cancer and desmoid tumors, as well as Breakthrough Device designations...
Recently, the U.S. Food and Drug Administration (FDA) granted Fast Track designation for momelotinib in myelofibrosis, granted Priority Review to a biologics license application for luspatercept in myelodyslastic syndromes and beta-thalassemia, granted Orphan Drug designation for a new chemical...
MORE DATA are in to support early intervention for high-risk smoldering multiple myeloma—an early, asymptomatic entity lacking the presence of CRAB criteria (elevated calcium, renal failure, anemia, bone lesions). The latest come from two phase II studies presented at the 2018 American Society of...
PRESS BRIEFING moderator Joseph Mikhael, MD, Professor of Applied Cancer Research and Drug Discovery, Translational Genomics Research Institute, City of Hope Cancer Center in Phoenix, noted that traditional models are based on simplicity, and the scoring system contains few variables. “In an era...
A PERSONALIZED risk-prediction model for myelodysplastic syndromes (MDS) has been developed through the use of a machine-learning approach that analyzes genomic and clinical data for an individual patient. According to lead investigator Aziz Nazha, MD, of the Cleveland Clinic, the model provides...
Over the past week, the U.S. Food and Drug Administration (FDA) granted several Fast Track and Orphan Drug designations to treatments for myelodysplastic syndromes, triple-negative breast cancer, acute myeloid leukemia (AML), and Epstein-Barr virus (EBV)-associated cancers. Fast Track Designation...
Over the past week, the U.S. Food and Drug Administration (FDA) granted a Fast Track designation to a treatment for metastatic uveal melanoma and received a biologics application for an agent to control anemia. Fast Track Designation for Tebentafusp in Metastatic Uveal Melanoma The FDA granted...
This week, the U.S. Food and Drug Administration (FDA) announced policy changes to modernize mammography policies and issued a Breakthrough Therapy designation, an Orphan Drug designation, and an investigational new drug application. FDA Advances Policy Changes to Modernize Mammography Services...
TO ADD to our ongoing coverage of the 2018 American Society of Hematology (ASH) Annual Meeting & Exposition, we bring readers of The ASCO Post these summaries of an assortment of interesting studies. They focus on novel therapies under investigation in the treatment of acute lymphoblastic...
AS PART of The ASCO Post’s continued coverage of the 2018 American Society of Hematology (ASH) Annual Meeting & Exposition, here is an update on seven different studies on new therapeutics in myelodysplastic syndromes (MDS). Among the treatments highlighted here are the erythroid maturation...
In a phase I trial reported in the Journal of Clinical Oncology, Craddock et al found evidence that the sequential combination of azacitidine and lenalidomide may be an effective salvage therapy in patients with acute myeloid leukemia (AML) relapsing after allogeneic stem cell transplantation...
Findings from a new study by researchers at the National Cancer Institute (NCI) show that patients treated with chemotherapy for most solid tumors from 2000 to 2014 experienced an increased risk of therapy-related myelodysplastic syndrome/acute myeloid leukemia (MDS/AML). The study, which used U.S. ...
Mikkael A. Sekeres, MD, of the Cleveland Clinic, discusses phase III study findings on luspatercept to treat anemia in patients with very low-, low-, or intermediate-risk myelodysplastic syndromes with ring sideroblasts who require red blood cell transfusions (Abstract 1).
Suleika Jaouad, an Emmy Award–winning writer, advocate, and cancer survivor who was diagnosed at age 22 with myelodysplastic syndrome and acute myeloid leukemia, discusses what she has learned about coping with cancer, learning from it, and growing beyond it.
THE 22ND CONGRESS of the European Hematology Association (EHA) was held in June in Madrid, drawing hematologists and allied professionals from every subspecialty of hematology from around the world. Among the extensive educational and scientific program, the EHA Congress provides a forum for...
Although several treatment options are available for patients with myelodysplastic syndromes (MDS), hematopoietic stem cell transplantation (HSCT) remains the only curative therapy.1 The risks of complications and death from transplantation can be substantial. Determining which patients may...
In a study reported in The New England Journal of Medicine, R. Coleman Lindsley, MD, PhD, of Dana-Farber Cancer Institute, and colleagues found that a number of mutations present in patients with myelodysplastic syndromes (MDS) were associated with poorer clinical outcome after allogeneic...
On June 29, the U.S. Food and Drug Administration (FDA) allowed marketing of ClearLLab Reagents (T1, T2, B1, B2, M), the first agency-authorized test for use with flow cytometry to aid in the detection of several leukemias and lymphomas, including chronic leukemia, acute leukemia, non-Hodgkin...
In the phase III RICMAC trial, use of a dose-reduced vs a standard myeloablative conditioning regimen prior to allogeneic stem cell transplantation (ASCT) did not appear to reduce 2-year relapse-free or overall survival in patients with myelodysplastic syndrome. These findings were reported by...
Steven D. Gore, MD, Director of Hematologic Malignancies at Yale School of Medicine, New Haven, Connecticut, applauded Dr. Stein for “accruing a terrible patient population, mutation-wise” and called the preliminary findings for the benefit of enasidenib in patients with ASXL1 mutations...
At the 2016 American Society for Hematology (ASH) Annual Meeting & Exposition, researchers reported early success with two new experimental agents for high-risk myelodysplastic syndromes—enasidenib (also known as AG-221), a potent oral inhibitor of the isocitrate dehydrogenase 2 (IDH2) enzyme,...
As reported in the Journal of Clinical Oncology, Shaffer et al have developed a prognostic scoring system for patients undergoing allogeneic hematopoietic cell transplantation for myelodysplastic syndrome. Study Details The study involved data from 2,133 patients with myelodysplastic syndrome in...
As reported in The Lancet Oncology by Garcia-Manero et al, the phase III ONTIME trial showed that treatment with the Ras mimetic rigosertib did not significantly improve overall survival vs best supportive care in patients with high-risk myelodysplastic syndromes after failure of hypomethylating...