Myeloproliferative Neoplasms

Next-generation sequencing plays a critical role in the diagnosis of patients with myeloid malignancies, but it also plays a necessary role in the identification of passenger mutations and subclonal events that go beyond founding drivers.

Key opinion leaders in the management of myeloproliferative neoplasms review factors to consider when selecting treatments for patients and highlight the role of fedratinib in the first- or second-line settings.

A panel of experts in myeloid malignancies begins with a discussion on risk assessment criteria, next-generation sequencing, and symptom burden questionnaires to assess prognosis for patients with myelofibrosis.

Ruben A. Mesa, MD, discusses the unique characteristics of pacritinib in the treatment of patients with myelofibrosis.

Jean-Jacques Kiladjian, MD, PhD, discusses, the potential utility of momelotinib in myelofibrosis.

Achieving transfusion independence after 24 weeks of treatment with momelotinib is associated with clinical benefit in patients with myelofibrosis irrespective of the degree of anemia, platelet count, or transfusion status at baseline.

John O. Mascarenhas, MD, discusses the potential for pacritinib to address an unmet need in the treatment of patients with myelofibrosis.

Srdan Verstovsek, MD, PhD, discusses the efficacy demonstrated with pacritinib in the treatment of patients with myelofibrosis.

Momelotinib can provide clinically relevant and comparable improvement in overall symptom burden and individual symptom items compared with ruxolitinib in patients with intermediate- and high-risk JAK inhibitor–naïve myelofibrosis.

The FDA has approved avapritinib (Ayvakit) for the treatment of adult patients with advanced systemic mastocytosis, including those with aggressive systemic mastocytosis, systemic mastocytosis with an associated hematological neoplasm, and mast cell leukemia.

Although cardiovascular health of patients with myeloproliferative neoplasms was relatively good, an estimated 11% to 22% of patients were not prescribed appropriate medications for management of comorbidities associated with thrombotic risk.

Ruben A. Mesa, MD, discusses results seen with momelotinib in patients with transfusion-independent myelofibrosis, as demonstrated in the phase 3 SIMPLIFY-1 and SIMPLIFY-2 trials.

Ruben A. Mesa, MD, discusses the importance of achieving transfusion independence with momelotinib in myelofibrosis.

Jan Philipp Bewersdorf, MD, discusses the role of allogeneic hematopoietic cell transplant in myelofibrosis.

Week 24 transfusion independence was associated with an improvement in overall survival vs week 24 transfusion dependence in patients with myelofibrosis who were randomized to momelotinib in the phase 3 SIMPLIFY 1 and SIMPLIFY 2 trials.

In an effort to build on the efficacy demonstrated with ruxolitinib, investigators are evaluating combination therapies—specifically the dual inhibition of the JAK-STAT and BET pathways using pelabresib, a novel BET inhibitor.

The FDA has granted breakthrough therapy designation to rusfertide as a potential therapeutic option for patients with polycythemia vera to reduce erythrocytosis in those who do not require further treatment for thrombocytosis and/or leukocytosis.

Abdulraheem Yacoub, MD, discusses next steps with the combination of parsaclisib and ruxolitinib in myelofibrosis.

The FDA has granted priority review to a new drug application for pacritinib as a potential treatment option for patients with myelofibrosis and severe thrombocytopenia, defined as platelet counts less than 50 x 109/L.

Abdulraheem Yacoub, MD, discusses the rationale for combining PI3K inhibitors with ruxolitinib in myelofibrosis, the results of the phase 2 study with parsaclisib, and ongoing phase 3 trials evaluating this novel combination.

Srdan Verstovsek, MD, PhD, discusses the rationale for utilizing ruxolitinib and navitoclax in patients with relapsed/refractory myelofibrosis.

The biologics license application seeking the approval of ropeginterferon alfa-2b-njft for use in the treatment of patients with polycythemia vera has been resubmitted to the FDA.

Srdan Verstovsek, MD, PhD, discusses future research efforts for patients with myeloproliferative neoplasms.

Srdan Verstovsek, MD, PhD, discusses real-world survival data with ruxolitinib in myelofibrosis.

Abdulraheem Yacoub, MD, discusses efficacy and safety results with parsaclisib in myelofibrosis.