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Myelodysplastic Syndromes

Updates in Lower-Risk Myelodysplastic Syndromes/Neoplasms

  Myelodysplastic syndromes (MDS) are a heterogeneous group of clonal hematopoietic stem cell disorders that are characterized by ineffective hematopoiesis, resulting in cytopenias, and they carry a risk of progression to acute myeloid leukemia (AML). In 2022, the fifth edition of the World Health ...

Myelodysplastic Syndromes

FDA Approves Ivosidenib for Myelodysplastic Syndromes

On October 24, the U.S. Food and Drug Administration (FDA) approved ivosidenib (Tibsovo) for adult patients with relapsed or refractory myelodysplastic syndromes (MDS) with a susceptible isocitrate dehydrogenase-1 (IDH1) mutation, as detected by an FDA-approved test. The FDA also approved the...

Myelodysplastic Syndromes
Genomics/Genetics

Allogeneic HCT Outcomes in Patients With MDS and High-Risk Genetics

In a genetic analysis from the Blood and Marrow Transplant Clinical Trials Network 1102 Study reported in the Journal of Clinical Oncology, Versluis et al found that allogeneic hematopoietic cell transplantation (HCT) improved overall survival compared with non-HCT treatment in patients with...

Myelodysplastic Syndromes

FDA Approves Luspatercept-aamt as First-Line Treatment for Adult Patients With Lower-Risk MDS and Anemia Who May Require Transfusions

On August 28, the U.S. Food and Drug Administration (FDA) approved luspatercept-aamt (Reblozyl) to treat anemia in adult patients with very low– to intermediate-risk myelodysplastic syndromes (MDS) who may require regular red blood cell transfusions and who have not received previous...

Myelodysplastic Syndromes
Hematologic Malignancies
Issues in Oncology

Do MDS Diagnoses Require Second Opinions?

Investigators have identified a need for strong coordination between clinicians and skilled pathologists to ensure accurate, timely diagnosis of myelodysplastic syndromes (MDS). The study findings were recently published by Gorak et al in Blood Advances. Background MDS—a collection of chronic...

Myelodysplastic Syndromes

Expert Point of View: Anand Ashwin Patel, MD

As presented at the 2023 ASCO Annual Meeting, the phase III IMerge trial of imetelstat1 and the phase III ­COMMANDS trial of luspatercept2 met their primary endpoints of transfusion independence. The findings suggest that lower-risk patients with transfusion-dependent, non-del(5q) myelodysplastic...

Myelodysplastic Syndromes

IMerge Trial: Use of Imetelstat Reduces Transfusion Dependence in Lower-Risk MDS

In the primary analysis of the phase III randomized, double-blind, placebo-controlled IMerge trial, in those achieving the primary endpoint of 8-week transfusion independence, treatment with imetelstat significantly reduced transfusion dependence—for a median duration of 1 year—and improved a...

myelodysplastic syndromes
supportive care

Aaron T. Gerds, MD, on Anemia in Myelofibrosis: New Data on Treatment With Luspatercept

Aaron T. Gerds, MD, of Cleveland Clinic Taussig Cancer Institute, talks about treating the anemia many patients with myelofibrosis experience because of JAK inhibitor therapy. The ACE-536-MF-001 study showed that luspatercept improved anemia and transfusion burden in this population, with a safety profile consistent with that in previous studies (Abstract 7016).

Myelodysplastic Syndromes

Use of Eltrombopag in Low-Risk Myelodysplastic Syndrome With Thrombocytopenia

Interim results of a phase II study (EQOL-MDS) reported in the Journal of Clinical Oncology by Oliva et al showed that eltrombopag, an orally bioavailable small molecule acting as a thrombopoietin receptor agonist, significantly increased the rate of platelet response vs placebo in patients with...

myelodysplastic syndromes

Amer Methqal Zeidan, MBBS, MHS, on Myelodysplastic Syndromes: New Data From the IMerge Study of Imetelstat

Amer Methqal Zeidan, MBBS, MHS, of Yale University and Yale Cancer Center, discusses phase III findings on the first-in-class telomerase inhibitor imetelstat, which was given to patients with heavily transfusion-dependent non-del(5q) lower-risk myelodysplastic syndromes that are resistant to erythropoiesis-stimulating agents. Imetelstat resulted in a significant and sustained red blood cell (RBC) transfusion independence in 40% of these heavily transfused patients. The response was also durable and accompanied by an impressive median hemoglobin rise of 3.6 g/dL, and seen in patients with and without ring sideroblasts. Importantly, reduced variant allele frequency was observed in the most commonly mutated myeloid genes which correlated with duration of transfusion independence and hemoglobin rise, therefore suggesting a disease-modifying potential of this agent (Abstract 7004).

myelodysplastic syndromes

Guillermo Garcia-Manero, MD, on Myelodysplastic Syndromes: Luspatercept and Epoetin Alfa in Lower-Risk Disease

Guillermo Garcia-Manero, MD, of The University of Texas MD Anderson Cancer Center, discusses phase III findings from the COMMANDS trial. Compared with epoetin alfa, luspatercept improved red blood cell transfusion independence and erythroid response, as well as the duration of response in erythropoiesis-stimulating agent–naive, transfusion-dependent patients with lower‐risk myelodysplastic syndromes (Abstract 7003).

Myelodysplastic Syndromes

Luspatercept Improves Reliance on Blood Transfusions for Patients With Lower-Risk MDS

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 were presented by...

Myelodysplastic Syndromes

Prognostic Performance of a Molecular International Prognostic Scoring System for MDS

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...

Myelodysplastic Syndromes

Magrolimab Plus Azacitidine in Higher-Risk Myelodysplastic Syndromes

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...

Myelodysplastic Syndromes
Leukemia

Vitamin B5 May Help Improve Red Blood Cell Production in Patients With Myelodysplastic Syndromes

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....

Hematologic Malignancies
Survivorship
Gastrointestinal Cancer
Lymphoma
Leukemia
Myelodysplastic Syndromes

Risk of Subsequent Gastrointestinal Tract Malignancies After Blood or Marrow Transplantation

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...

Myelodysplastic Syndromes

Phase III IMerge Trial of Imetelstat in Lower-Risk MDS

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...

Myelodysplastic Syndromes
Symptom Management

Luspatercept for Anemia in Patients With Lower-Risk Myelodysplastic Syndromes: Long-Term Follow-up

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...

Leukemia
Lymphoma
Myelodysplastic Syndromes

FDA Approves Pemigatinib for Patients With Myeloid/Lymphoid Neoplasms and FGFR1 Rearrangement

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...

Myelodysplastic Syndromes

Low-Dose Decitabine and Azacitidine in Lower-Risk MDS

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...

Leukemia
Myelodysplastic Syndromes

Eprenetapopt/Azacitidine Maintenance After Allogeneic HSCT for TP53-Mutant AML and MDS

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”...

Genomics/Genetics
Myelodysplastic Syndromes
Leukemia

Demethylation and Upregulation of the Oncogene SALL4 After Hypomethylating Therapy

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...

Leukemia
Myelodysplastic Syndromes
Genomics/Genetics

Targeted Therapy Emavusertib Shows Activity in Patients With MDS or AML With Specific Mutations

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...

myelodysplastic syndromes

Ruben A. Mesa, MD, on Myelofibrosis: Phase III Results on Momelotinib vs Danazol

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).

Hematologic Malignancies
Leukemia
Lymphoma
Myelodysplastic Syndromes

Allogeneic Transplantation for Hematologic Neoplasms in Adults

“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...

Myelodysplastic Syndromes

Study Defines Stem Cell Groups That May Drive Development of Myelodysplastic Syndromes

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...

Myelodysplastic Syndromes

PANTHER: No Significant Benefit for Pevonedistat Plus Azacitidine in Higher-Risk Myelodysplastic Syndrome

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...

Myelodysplastic Syndromes

PANTHER: No Significant Benefit for Pevonedistat Plus Azacitidine in Higher-Risk Myelodysplastic Syndrome

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...

leukemia
myelodysplastic syndromes

Alba Rodriguez-Meira, DPhil, on TP53-Mutated Leukemic Transformation in Myeloproliferative Neoplasms

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).

Leukemia
Myelodysplastic Syndromes

Study Identifies Factors for Severe COVID-19 Illness Among Patients With Acute Leukemia or MDS

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...

Myelodysplastic Syndromes
Leukemia
COVID-19

Antibody Response to Second Dose of mRNA COVID-19 Vaccine in Patients With AML and MDS

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 ...

Myelodysplastic Syndromes

Personalized Risk Prediction Model for Patients With Myelodysplastic Syndromes

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,...

Myelodysplastic Syndromes

Continuous Azacitidine vs Reduced-Intensity Conditioning HSCT in Older Patients With Advanced Myelodysplastic Syndrome

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 ...

Breast Cancer
Myelodysplastic Syndromes

FDA Pipeline: Designations for Therapies in Myelodysplastic Syndromes and Triple-Negative Breast Cancer

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...

Myelodysplastic Syndromes

Reduced-Intensity Conditioning Allogeneic HCT Based on Donor Availability in Older Patients With Advanced Myelodysplastic Syndrome

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...

Myelodysplastic Syndromes

Novel Insights in Myelodysplastic Syndromes: Highlights From 2020 ASH Meeting

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...

Myelodysplastic Syndromes

Oral Azacitidine in Lower-Risk Myelodysplastic Syndromes

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...

Myelodysplastic Syndromes
Immunotherapy

Identification of Genomic Subgroups of Patients With Myelodysplastic Syndromes: New Prognostic Modeling

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 ...

Leukemia
Myelodysplastic Syndromes
Genomics/Genetics

Whole-Genome Sequencing vs Cytogenetic Analysis in Myeloid Cancers

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...

Leukemia
Myelodysplastic Syndromes
Genomics/Genetics

Eprenetapopt Plus Azacitidine in TP53-Mutated MDS and AML

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) ...

Myelodysplastic Syndromes
Geriatric Oncology

Transplant May Improve Survival in Older Patients With High-Risk Myelodysplastic Syndrome

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...

Myelodysplastic Syndromes

First-in-Class Eprenetapopt Plus Azacitidine for TP53-Mutant Myelodysplastic Syndromes

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)....

Myelodysplastic Syndromes

Lenalidomide Plus Epoetin Alfa vs Lenalidomide Alone in Refractory Non-del(5q) 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...

Leukemia
Myelodysplastic Syndromes

Intensified Reduced-Intensity Conditioning for Allogeneic Stem Cell Transplantation in AML or MDS

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...

Leukemia
Myelodysplastic Syndromes

ASH 2020: Role of Venetoclax in High-Risk Myeloid Malignancies

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 ...

Myelodysplastic Syndromes

Reduced-Intensity Allogeneic Hematopoietic Stem Cell Transplant May Improve Survival in Older Patients With High-Risk 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...

myelodysplastic syndromes

Corey Cutler, MD, MPH, on MDS: Evaluating Treatments in Older Patients With Advanced Disease

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).

Myelodysplastic Syndromes

Imetelstat in Lower-Risk MDS With High Transfusion Dependence

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...

Myelodysplastic Syndromes

Novel Treatments Emerge for Patients With Lower-Risk Myelodysplastic Syndromes

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 ...

Kidney Cancer
Lung Cancer
Myelodysplastic Syndromes
Solid Tumors
Skin Cancer
Lymphoma
Pancreatic Cancer
Breast Cancer
Immunotherapy

FDA Pipeline: Designations in Kidney and Lung Cancers, Myelodysplastic Syndromes, and More

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...

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