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Patients with lower-risk myelodysplastic syndromes (MDS) who received luspatercept to treat anemia instead of erythropoiesis-stimulating agents (ESAs)—the current standard of care—needed fewer blood transfusions and clinic visits. These findings from the phase III COMMANDS trial will be presented...
In a European study reported in the Journal of Clinical Oncology, Sauta et al found that the recently devised Molecular International Prognostic Scoring System (IPSS-M) had improved prognostic ability vs the standard Revised International Prognostic Scoring System (IPSS-R) in patients with...
In a phase Ib trial (5F9005) reported in the Journal of Clinical Oncology, David A. Sallman, MD, and colleagues found that the combination of magrolimab and azacitidine showed activity in previously untreated patients with intermediate- to very high–risk myelodysplastic syndromes (MDS). As stated...
Researchers have discovered that vitamin B5 in combination with existing drugs may be the key to improving outcomes in patients with myelodysplastic syndromes (MDS) and ineffective red blood cell production, according to a novel study published by Mian et al in Science Translational Medicine....
In a study reported in JAMA Oncology, McDonald et al found that receipt of blood or marrow transplantation (BMT) was associated with an increased risk of subsequent malignant neoplasms of the gastrointestinal (GI) tract. As stated by the investigators, “Survivors of BMT are at increased risk of...
Positive results were announced from the phase III IMerge trial (ClinicalTrials.gov identifier NCT02598661) evaluating the first-in-class telomerase inhibitor imetelstat in patients with lower-risk myelodysplastic syndromes (MDS) who are relapsed, refractory, or ineligible for...
In a long-term follow-up of the German phase II PACE-MDS trial reported in the Journal of Clinical Oncology, Uwe Platzbecker, MD, and colleagues described erythroid, neutrophil, and platelet hematologic improvement rates with luspatercept treatment for anemia in patients with lower-risk...
On August 26, the U.S. Food and Drug Administration (FDA) approved pemigatinib (Pemazyre), a selective fibroblast growth factor receptor (FGFR) inhibitor, for the treatment of adults with relapsed or refractory myeloid/lymphoid neoplasms with FGFR1 rearrangement. Myeloid/lymphoid neoplasms with...
As reported in NEJM Evidence by Sasaki et al, extended follow-up of a phase II trial showed good outcomes with low doses of the hypomethylating agents decitabine and azacitidine in previously untreated patients with lower-risk myelodysplastic syndromes (MDS). Study Details The study included 113...
In a phase II trial reported in the Journal of Clinical Oncology, Mishra et al found that maintenance treatment with the first-in-class small-molecule p53 reactivator eprenetapopt plus azacitidine following allogeneic hematopoietic stem cell transplantation (HSCT) was associated with “encouraging”...
In a study reported in The New England Journal of Medicine, Liu et al found that hypomethylating therapy was associated with upregulation of the oncogene SALL4 in a high proportion of patients with myelodysplastic syndrome. They also identified the gene region that is demethylated and responsible...
Mutations in SF3B1 and U2AF1 can drive overexpression of activated IRAK4—which regulates inflammation and promotes cancer cell growth and survival—and are associated with a poor prognosis for patients with high-risk myelodysplastic syndrome (MDS) and acute myeloid leukemia (AML). Emavusertib is a...
Ruben A. Mesa, MD, of Mays Cancer Center at UT Health San Antonio MD Anderson Cancer Center, discusses new findings from the MOMENTUM study. This trial showed that in symptomatic and anemic patients with myelofibrosis, momelotinib was superior to danazol for symptom and spleen responses, as well as transfusion requirements (Abstract 7002).
“The value of experience is not in seeing much, but in seeing wisely.” —Sir William Osler To complement The ASCO Post’s continued comprehensive coverage of the 2021 American Society of Hematology (ASH) Annual Meeting & Exposition, here are several abstracts selected from the meeting...
Researchers have discovered that treatment resistance in patients with myelodysplastic syndromes (MDS) may be caused by two distinct classes of stem cells and identified possible therapeutic approaches that target these cells. Their findings, which could have significant benefits for patients with...
The phase III PANTHER trial, which evaluated pevonedistat plus azacitidine vs azacitidine alone in higher-risk myelodysplastic syndromes, chronic myelomonocytic leukemia, and low–blast count acute myeloid leukemia (AML) failed to meet its primary endpoint of event-free survival,1 though lessons...
The phase III PANTHER trial, which evaluated pevonedistat plus azacitidine vs azacitidine alone in higher-risk myelodysplastic syndromes, chronic myelomonocytic leukemia, and low–blast count acute myeloid leukemia (AML), failed to meet its primary endpoint of event-free survival, though lessons...
Alba Rodriguez-Meira, DPhil, of the University of Oxford, discusses a comprehensive analysis of the genetic, cellular, and molecular landscape of TP53-mediated transformation, providing insights into the evolution of chronic hematologic malignancies toward an aggressive acute leukemia. Because TP53 is the most commonly mutated gene in human cancer, these findings may well be of broad relevance (Abstract 3).
In separate analyses of 257 patients with acute leukemia or myelodysplastic syndrome (MDS) who developed COVID-19 and are part of the American Society of Hematology (ASH) RC COVID-19 Registry for Hematology, both neutropenia and having active MDS or leukemia (vs being in remission) were found to...
In one of the largest studies to date of the antibody response to vaccination against COVID-19 in people who had been treated for acute myeloid leukemia (AML) and myelodysplastic syndrome (MDS), patients responded well to two doses of the Moderna mRNA vaccine and saw a pronounced increase in levels ...
As reported in the Journal of Clinical Oncology, Aziz Nazha, MD, and colleagues have developed a model for predicting the risk of disease progression for individual patients with myelodysplastic syndromes (MDS). To develop the prediction model for overall survival and leukemic transformation,...
In the German phase II VidazaAllo study reported in the Journal of Clinical Oncology, Kröger et al found that switching from azacitidine to reduced-intensity conditioning allogeneic hematopoietic stem cell transplantation (HSCT) contingent upon donor availability was associated with better outcomes ...
Recently, the U.S. Food and Drug Administration (FDA) granted special designation status to treatments for myelodysplastic syndromes and advanced triple-negative breast cancer. Breakthrough Therapy Designation for Venetoclax in Combination With Azacitidine for Myelodysplastic Syndromes Venetoclax...
In a study reported in the Journal of Clinical Oncology, Nakamura et al found that use of reduced-intensity conditioning allogeneic hematopoietic cell transplantation (HCT) in older patients with advanced myelodysplastic syndrome (MDS) with an available matched donor improved overall survival vs...
To complement The ASCO Post’s comprehensive coverage of the 2020 American Society of Hematology (ASH) Annual Meeting & Exposition, here are several abstracts selected from the meeting proceedings focusing on novel clinical trial findings in myelodysplastic syndromes (MDS). For full details of...
In a phase III trial reported in the Journal of Clinical Oncology, Guillermo Garcia-Manero, MD, and colleagues found that oral azacitidine significantly improved red blood cell (RBC) transfusion independence vs placebo in patients with lower-risk myelodysplastic syndromes. An increase in early...
In a study reported in the Journal of Clinical Oncology, Bersanelli et al reported that researchers in the EuroMDS Project found that patients with myelodysplastic syndromes (MDS) could be classified into eight distinct subtypes based on genomic characteristics. They also developed a new prognostic ...
In a study reported in The New England Journal of Medicine, Duncavage et al found that whole-genome sequencing in patients with acute myeloid leukemia (AML) or myelodysplastic syndromes (MDS) provided rapid genomic profiling that improved diagnostic yield vs conventional cytogenetic analysis, as...
In a Groupe Francophone des Myélodysplasies phase II study reported in the Journal of Clinical Oncology, Cluzeau et al found that the combination of eprenetapopt and azacitidine produced high response rates in previously untreated patients with high-risk TP53-mutated myelodysplastic syndromes (MDS) ...
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...