Bomedemstat as an investigative treatment for myeloproliferative neoplasms

作者：Hugh Young Rienhoff, Harinder Singh Gill · 发表于：Expert Opinion on Investigational Drugs · 年份：2023 · DOI：10.1080/13543784.2023.2267980 · 被引用次数：13 · 研究领域：Myeloproliferative Neoplasms: Diagnosis and Treatment、Acute Myeloid Leukemia Research、Eosinophilic Disorders and Syndromes

INTRODUCTION: Myeloproliferative neoplasm (MPN) is a heterogeneous group of hematopoietic stem cell disorders characterized by clonal proliferation of one of more of the hematopoietic stem cell lineages. Clinical manifestations result from uncontrolled myeloproliferation, extramedullary hematopoiesis with splenomegaly and excessive inflammatory cytokine production. Currently available therapy improves hematologic parameters and symptoms but does not adequately address the underlying neoplastic biology. Bomedemstat has thus far demonstrated clinical efficacy and tolerability in the treatment of MPNs with recent evidence of impacting the malignant stem cell population. AREAS COVERED: This review summarizes the mechanisms of action, pharmacokinetics and pharmacodynamics, safety and efficacy of bomedemstat in MPN with specific emphasis on essential thrombocythemia (ET) and myelofibrosis (MF). EXPERT OPINION: In patients with MPNs, bomedemstat appears effective and well tolerated. The signs and symptoms of these diseases are managed as a reduction in the frequency of mutant cells was demonstrated in patients with ET and MF. Ongoing and planned studies of bomedemstat in MPN will establish the position of bomedemstat in MPNs and may help to redefine treatment endpoints of MPNs in the future.