Pacritinib for the treatment of patients with myelofibrosis and thrombocytopenia.

INTRODUCTION

Myelofibrosis (MF) is a rare myeloproliferative neoplasm characterized by a complex symptom profile, cytopenias, splenomegaly, and potential for leukemic progression. Severe thrombocytopenia is common in patients with MF and correlates with poor prognosis; however, until recently, treatment options for these patients were limited. Pacritinib, a potent Janus kinase (JAK) 2/interleukin-1 receptor-associated kinase 1 (IRAK1) inhibitor, has demonstrated significant reduction in splenomegaly, improved symptom control, and a manageable safety profile in patients with MF regardless of the severity of thrombocytopenia.

AREAS COVERED: This review will outline the pacritinib drug profile and summarize key efficacy and safety data, focusing on the 200 mg twice daily dose from phase 2 and 3 studies that formed the basis for the recent US Food and Drug Administration approval of pacritinib in patients with MF and severe thrombocytopenia (platelet counts <50 × 109/L).

EXPERT OPINION: Pacritinib, with its unique mechanism of action targeting both JAK2 and IRAK1, offers patients with MF and severe thrombocytopenia a new treatment option, providing consistent disease and symptom control. Adverse events are easily manageable. Further analyses to identify ideal patient characteristics for pacritinib and other JAK inhibitors along with studies of pacritinib combinations are warranted, including in related myeloid malignancies.