Myeloproliferative Neoplasms

The National Institute for Health and Care Excellence has published its Final Draft Guidance recommending the use of ruxolitinib as treatment for eligible patients in England and Wales with polycythemia vera that is resistant or intolerant to hydroxycarbamide/hydroxyurea.

Following the FDA approvals of the JAK inhibitors ruxolitinib, fedratinib, and pacritinib, the treatment landscape of myelofibrosis continues to grow with the use of these agents with an additional FDA review planned for momelotinib in September 2023.

Anthony M Hunter, MD, discusses the current treatment landscape with JAK inhibitors in myelofibrosis.

Guillermo Garcia-Manero, MD, discusses the key efficacy findings from the phase 3 COMMANDS trial evaluating luspatercept-aamt in patients with myelodysplastic syndrome who were erythropoiesis-stimulating agent naïve and red blood cell transfusion dependent.

A retrospective analysis of the Surveillance, Epidemiology, and End Results database emphasized the potential impact of specific socio-racial factors, such as race, sex, and age, on survival outcomes in patients with primary myelofibrosis.

Drs Joe Scandura and John Mascarenhas discuss their treatment approaches for high-risk patients with polycythemia vera.

Experts review critical trial data in the treatment of patients with polycythemia vera.

Findings from a systematic review of several observational studies reveal that increasing disparities in survival outcomes within hematologic malignancies can be primarily attributed to 5 social determinants of health: lack of access to health insurance, treatment at a non-academic facility, low income or education level, and unmarried status.

The phase 3 COMMANDS trial investigating the use of luspatercept in patients with myelodysplastic syndrome who had not received an erythropoiesis-stimulating agent and were red blood cell transfusion dependent achieved its primary end point of superiority over treatment with an erythropoiesis-stimulating agent.

A predictive system developed using data from United States and European stem cell transplant registries was prognostic of survival in patients with myelofibrosis undergoing allogeneic hematopoietic cell transplantation.

Patients with essential thrombocythemia and polycythemia vera who also had arterial hypertension experienced a higher cumulative incidence of thrombotic adverse effects compared with those without hypertension and fewer thrombotic complications following treatment with renin angiotensin system inhibitors.

Aaron T. Gerds, MD, PhD, expands on the potential role of momelotinib in the treatment of patients with myelofibrosis who present with anemia, details the data from MOMENTUM, and explains what FDA approval of momelotinib could mean for the treatment of this patient population.

Joe Scandura, MD, PhD, summarizes the toxicity profile of interferon therapy in patients with polycythemia vera.

Drs John Mascarenhas and Joe Scandura review the toxicities seen with the non-specific chemotherapeutic agent hydroxyurea in patients with polycythemia vera.

Anna B. Halpern, MD, discusses ongoing efforts to address unmet needs in patients with primary myelofibrosis.

Uwe Platzbecker, MD, discusses the significance of the FDA approval of luspatercept for patients with lower-risk myelodysplastic syndrome with anemia.

The FDA has approved luspatercept-aamt (Reblozyl) for the treatment of anemia without prior erythropoiesis stimulating agent (ESA) use in adult patients with very low– to intermediate-risk myelodysplastic syndrome (MDS) who may require regular red blood cell (RBC) transfusions.

Anna B. Halpern, MD, discusses investigational efforts to utilize ruxolitinib and navitoclax in earlier treatment lines for patients with myelofibrosis.

Rami Komrokji, MD, discusses an international dataset analysis of the 2 current classification systems for patients with myelodysplastic syndromes, as well as the next steps being taken to develop a more harmonized classification system.

Marina Kremyanskaya, MD, PhD, discusses the safety profile and future implications of rusfertide in the treatment of patients with phlebotomy-dependent polycythemia vera, as seen in the phase 2 REVIVE trial.

Joe Scandura, MD, PhD, explains how he considers individual symptom burden when deciding on a treatment regimen for a patient with polycythemia vera.

Key opinion leaders discuss their treatment targets for low- and high-risk patients with polycythemia vera.

Marina Kremyanskaya, MD, PhD, discusses the potential advantages of using rusfertide to treat patients with phlebotomy-dependent polycythemia vera, as seen in the phase 2 REVIVE trial.

John Mascarenhas, MD, discusses the goals of the phase 1/2 KRT-232-109 study evaluating the addition of the first-in-class MDM2 inhibitor navtemadlin to ruxolitinib in patients with primary or secondary myelofibrosis and highlights the eligibility criteria of this trial.

The FDA has accepted a new drug application seeking the approval of imetelstat for the treatment of transfusion-dependent anemia in patients with lower-risk myelodysplastic syndrome.

Individualized myelofibrosis treatment begins with correctly identifying a patient’s disease subtype and considering their symptoms, from which accurate decisions regarding the use of JAK inhibitors vs radiation vs hypomethylating agents can lead to spleen and symptom burden reductions.

Experts explain the critical importance of obtaining bone marrow biopsies for accurately diagnosing polycythemia vera.

John Mascarenhas, MD, and Joe Scandura, MD, PhD, detail the typical presentation of a patient with polycythemia vera in their clinical practices.

John Mascarenhas, MD, discusses the rationale for adding the first-in-class MDM2 inhibitor navtemadlin to ruxolitinib in patients receiving treatment for primary or secondary myelofibrosis who have previously had suboptimal responses with ruxolitinib.

Closing their discussion, the panel shares some parting comments on the recent updates in the MDS treatment landscape and hopes for the future.