Myeloproliferative Neoplasms

Mikkael A. Sekeres, MD, MS, discussed the changes to the classifications of MDS, the evolution of targeted therapies for patients with acute myeloid leukemia, and approaches for patients with bone marrow failure syndromes.

The European Commission has granted an orphan drug designation to selinexor for the treatment of patients with myelofibrosis.

Luspatercept generated a statistically significant improvement in red blood cell transfusion independence with concurrent hemoglobin increase vs epoetin alfa in patients with very low–, low-, or intermediate-risk myelodysplastic syndromes who require RBC transfusions, meeting the primary end point of the phase 3 COMMANDS trial.

Andrew Kuykendall, MD, discusses treatment considerations with ruxolitinib in myelofibrosis.

Haris Ali, MD, discusses a phase 1 dose-escalation trial investigating the administration of selinexor in combination with ruxolitinib for patients with treatment-naïve myelofibrosis.

Luspatercept produced favorable safety and long-term efficacy in patients with untreated lower-risk myelodysplastic syndromes, irrespective of subtype.

Patients with polycythemia vera, a myeloproliferative neoplasm associated with JAK2 mutations and overproduction of red blood cells, often require regular therapeutic phlebotomies to avoid thrombosis.

Momelotinib induced superior 24-week overall survival rates compared with danazol in thrombocytopenic, symptomatic, and anemic patients with myelofibrosis.

Haris Ali, MD, discusses the investigation of ruxolitinib and navitoclax combination therapy in myelofibrosis.

Treatment with GB2064 monotherapy for at least 6 months led to a reduction in collagen fibrosis of the bone marrow of at least 1 grade in 4 of 5 evaluable patients with myelofibrosis, according to topline findings from a planned intermediate assessment of the phase 2a MYLOX-1 trial.

Treatment with rusfertide generated sustained hematocrit control at levels below 45% in patients with polycythemia vera, leading to a reduced need for repeated phlebotomy and eliminating this need in some patients, according to findings from 2 phase 2 clinical trials.

Andrew Kuykendall, MD, discusses the evolution of prognostication factors in primary and secondary myelofibrosis.

John O. Mascarenhas, MD, discusses the investigation of pelabresib in myelofibrosis.

Dr Verstovsek discusses the FDA approval of pemigatinib in myeloid/lymphoid neoplasms with FGFR1 rearrangements, the transformative effects of pemigatinib, and the importance of identifying chromosomal abnormalities in patients with this aggressive disease.

Azacitidine plus venetoclax showed encouraging activity in patients with high-risk myelodysplastic syndromes or chronic myelomonocytic leukemia.

Emavusertib elicited antitumor activity when given as a monotherapy in patients with relapsed/refractory acute myeloid leukemia or high-risk myelodysplastic syndrome that harbored a spliceosome or FLT3 mutation.

A panel of hematology cancer experts discuss the current treatment landscape and novel emerging therapies for patients with myelofibrosis.

The FDA has granted permission for enrollment to resume in the monotherapy phase 1a portion of the phase 1/2 TakeAim Leukemia trial evaluating emavusertib in relapsed/refractory acute myeloid leukemia or high-risk myelodysplastic syndromes.

Rami Komrokji, MD, expands on the disease factors and treatment options for lower-risk and higher-risk myelodysplastic syndromes, and highlights other key updates in chronic lymphocytic leukemia, mantle cell lymphoma, diffuse large B-cell lymphoma, and myelofibrosis.

Liquid biopsy using targeted next-generation sequencing for early diagnosis and monitoring of patients with myeloid neoplasms is effective and detects chromosomal structural abnormalities.

The panelists conclude their discussion by highlighting investigational agents to look forward to.

Rami Komrokji, MD, presents to the panel a second clinical scenario of a 74-year-old with cytopenic myelofibrosis.

The FDA has placed a full clinical hold on a phase 1 dose escalation trial investigating the BRG1/BRM inhibitor FHD-286 in patients with relapsed/refractory acute myeloid leukemia or myelodysplastic syndrome.

Jacqueline S. Garcia, MD, discusses the role of ruxolitinib (Jakafi) in myelofibrosis.

John Mascarenhas, MD, discussed the effect of the data from MANIFEST on the treatment landscape and the future of adding active agents to ruxolitinib for treatment of myelofibrosis.