Myeloproliferative Neoplasms

A machine learning, artificial intelligence algorithm analyzing diagnostic bone marrow biopsy digital whole-slide images was able to effectively differentiate with 92.3% accuracy between prefibrotic primary myelofibrosis and essential thrombocythemia.

Amer Zeidan, MBBS, discusses classification and treatment updates for patients with myelodysplastic syndrome.

Marina Kremyanskaya, MD, PhD, discusses disease and patient characteristics that influence the choice between hydroxyurea and interferon therapy for patients with polycythemia vera or essential thrombocythemia.

Treatment with the combination of pelabresib and ruxolitinib led to a statistically significant and clinically meaningful improvement in spleen volume reduction vs placebo plus ruxolitinib in patients with JAK inhibitor-naive myelofibrosis, meeting the primary end point of the phase 3 MANIFEST-2 trial.

Patients with myelodysplastic syndrome harboring a TP53 mutation experienced a survival benefit with allogeneic hematopoietic cell transplantation compared with non-HCT treatment regardless of TP53 allelic status.

Gaby Hobbs, MD, discusses how research into the biology of myelofibrosis has propelled the development of novel therapies for this patient population and highlights the role that biomarkers play in the diagnosis, prognosis, and treatment response evaluation for these patients.

The European Medicines Agency’s Committee for Medicinal Products for Human Use has adopted a positive opinion supporting the approval of momelotinib for the treatment of disease-related splenomegaly or symptoms in adult patients with moderate to severe anemia with primary myelofibrosis, post–polycythemia vera myelofibrosis, or post–essential thrombocytopenia myelofibrosis who have not been exposed to a JAK inhibitor or who had received prior ruxolitinib.

Douglas A. Tremblay, MD, discusses the prevalence of myelodysplastic syndrome/myeloproliferative neoplasm overlap syndromes and the evolving treatment paradigm for these diseases.

Andrew T. Kuykendall, MD, discusses the importance of the approval of momelotinib in the treatment of patients with anemic symptomatic myelofibrosis, key efficacy and safety data from the MOMENTUM trial that supported the decision, and unanswered questions regarding the agent’s potential role in other subsets within this population.

Anthony M. Hunter, MD, discusses the evolution of JAK inhibitors in myelofibrosis, a topic that was highlighted in a presentation at the 2023 SOHO Annual Meeting.

Courtney D. DiNardo, MD, MSCE, discusses the significance of the FDA approval of ivosidenib in patients with relapsed/refractory, IDH1-mutated myelodysplastic syndromes.

The FDA has approved ivosidenib (Tibsovo) for the treatment of adult patients with relapsed/refractory myelodysplastic syndromes with a susceptible IDH1 mutation, as detected by an FDA-approved test.

Andrew Kuykendall, MD, discusses frontline cytoreductive treatment options for patients with polycythemia vera.

Amy W. Zhou, MD, discusses ongoing and planned trials that may enhance therapeutic options for patients with myelofibrosis and anemia that are currently taking place at the Washington University School of Medicine in St. Louis.

Anthony M. Hunter, MD, discusses the roles for pacritinib, momelotinib, and fedratinib in patients with myelofibrosis; treatment sequencing with newer-generation JAK inhibitors after ruxolitinib; and the importance of having several treatment options in the second-line setting and beyond.

Amy W. Zhou, MD, discusses combination therapies with ruxolitinib that may contribute to the treatment paradigm for patients with myelofibrosis, highlighting the importance of continued research with phase 3 confirmatory trials for patients with this disease.

The addition of bexmarilimab to standard-of-care azacitidine or azacitidine plus venetoclax continued to elicit responses in patients with relapsed or refractory acute myeloid leukemia and those with myelodysplastic syndrome who were refractory to hypomethylating agents.

Ricardo D. Parrondo, MD, discusses the clinical benefit seen with momelotinib in anemic myelofibrosis, and where it fits into the current treatment paradigm in this disease landscape.

The European Medicines Agency has validated the marketing authorization application for the use of imetelstat in the treatment of transfusion-dependent anemia in patients with lower-risk myelodysplastic syndrome.

Amy W. Zhou, MD, discusses the investigation of ruxolitinib given in conjunction with novel or emerging agents such as pacritinib and momelotinib, highlighting these agents’ potential at contributing to the treatment paradigm in myelofibrosis.

The hematologic neoplasm systemic mastocytosis is marked by the clonal proliferation of mast cells in the skin, bone marrow, gastrointestinal tract, spleen, and/or liver, caused by an activating KIT mutation in the KIT gene.

Amy W. Zhou, MD, highlights the unique mechanism of action of the JAK1/2 and ACVR1 inhibitor momelotinib and the importance of ongoing investigations of JAK inhibitor combination therapies in myelofibrosis.

John Mascarenhas, MD, and Joe Scandura, MD, PhD, close their discussion by highlighting what to look forward to in the future of polycythemia vera treatment.

The FDA has granted fast track designation to the investigational, anti-TMPRSS6 monoclonal antibody, MWTX-003 (DISC-3405), for use in the treatment of patients with polycythemia vera, according to an announcement from Disc Medicine, Inc.

Uwe Platzbecker, MD, discusses the efficacy and safety data from the COMMANDS trial that supported the approval, and the next steps planned for luspatercept’s investigation in patients with lower-risk MDS who are transfusion independent.

Uwe Platzbecker, MD, discusses how the FDA approval of luspatercept for the treatment of anemia in patients who have not had prior treatment with erythropoiesis-stimulating agent and may require regular red blood cell transfusions could change the sequencing of agents for this patient population.

Andrew Kuykendall, MD, discusses the significance of the FDA approval of momelotinib in patients with myelofibrosis.

The FDA has approved momelotinib (Ojjaara) for the treatment of adult patients with intermediate or high-risk myelofibrosis, including primary myelofibrosis or secondary myelofibrosis, and anemia.

Key opinion leaders discuss data on the use of ruxolitinib in patients with polycythemia vera.

Joe Scandura, MD, PhD, explains how a patient with polycythemia vera and driver mutations responds to interferon treatment.