Stephen T. Oh, MD, PhD, co-chief, Division of Hematology, associate professor, Department of Medicine, Hematology Division, associate professor, Department of Pathology and Immunology, Washington University School of Medicine in St. Louis, discusses the anemia benefit of pacritinib (Vonjo) in patients with myelofibrosis.

The phase 3 PERSIST-2 study (NCT02055781) evaluated the safety and efficacy of oral pacritinib compared with best available therapy in patients with thrombocytopenia and primary or secondary myelofibrosis. At the 2022 ASH Annual Meeting, investigators presented an analysis of pacritinib as a potent ACVR1 inhibitor and its clinical impact on transfusion independence in patients with myelofibrosis.

Although pacritinib is approved by the FDA for the treatment of adult patients with intermediate or high-risk primary or secondary (post-polycythemia vera or post-essential thrombocythemia) myelofibrosis with a platelet count below 50 × 109/L, data from the analysis showed that the agent provides an anemia benefit for other patients with myelofibrosis, Oh says.

Pacritinib displayed activity as an inhibitor of ACVR1 and reduced hepcidin levels in vitro. Treatment with the agent can lead to transfusion independence for patients with myelofibrosis who require red blood cell transfusions. These data, along with the efficacy findings from PERSIST-2, should be taken into consideration for deciding considering pacritinib, Oh concludes.

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