Dr. Mascarenhas on the Potential Clinical Significance of Pacritinib in Myelofibrosis

John O. Mascarenhas, MD, professor, Medicine, Hematology, and Medical Oncology, Icahn School of Medicine at Mount Sinai, director, the Center of Excellence for Blood Cancers and Myeloid Disorders, member, the Tisch Cancer Institute, discusses the potential clinical significance of pacritinib in patients with myelofibrosis.

The phase 3 PERSIST-2 trial (NCT02055781) compared the safety and efficacy of pacritinib with best available therapy (BAT) in patients with myelofibrosis and thrombocytopenia. In a retrospective analysis of the study presented at the 2022 SOHO Annual Meeting, findings showed that treatment with pacritinib led to an improvement in transfusion independence and hemoglobin.

The percentage of non–transfusion independent patients who achieved transfusion independence over any 12-week period through week 24 proved to be higher with 200-mg or 400-mg doses of pacritinib vs BAT, at 27% and 25% vs 5%, respectively. Additionally, a greater percentage of patients experienced “improved” or “very much improved” symptoms with 200 mg of pacritinib vs BAT, at 50% and 16%, respectively. More patients who received pacritinib experienced a decrease in modified total symptom score of at least 50% compared with those who received BAT, at 46% and 16%, respectively. Lastly, a higher percentage of those with hemoglobin less than 10 g/dL at baseline experienced an improvement of at least 1 g/dL at any time through week 24 if they received 200 or 400 mg of pacritinib vs BAT, at 15% and 23% vs 7%, respectively.

Although pacritinib is currently approved by the FDA for the treatment of patients with intermediate or high-risk primary or secondary myelofibrosis with a platelet count below 50 × 10⁹/L, data from PERSIST-2 suggest that pacritinib could have a role for patients with platelet counts up to 100 × 10⁹/L, Mascarenhas says. For patients with bicytopenias, anemia, and thrombocytopenia, pacritinib can be administered at a full dose and provide a spleen and symptom benefit without treatment-emergent thrombocytopenia, Mascarenhas explains. Additionally, pacritinib can eliminate or significantly lessen a patient’s transfusion burden, Mascarenhas concludes.

Funding supported by CTI BioPharma. Content independently developed by OncLive.