Ten years after ruxolitinib approval for myelofibrosis: A review of clinical efficacy Review


Authors: Pemmaraju, N.; Bose, P.; Rampal, R.; Gerds, A. T.; Fleischman, A.; Verstovsek, S.
Review Title: Ten years after ruxolitinib approval for myelofibrosis: A review of clinical efficacy
Abstract: Myelofibrosis (MF) is a chronic myeloproliferative neoplasm characterized by splenomegaly, abnormal cytokine expression, cytopenias, and progressive bone marrow fibrosis. The disease often manifests with burdensome symptoms and is associated with reduced survival. Ruxolitinib, an oral Janus kinase (JAK) 1 and JAK2 inhibitor, was the first agent approved for MF. As a first-in-class targeted treatment, ruxolitinib approval transformed the MF treatment approach and remains standard of care. In addition, targeted inhibition of JAK1/JAK2 signaling, a key molecular pathway underlying MF pathogenesis, and the large volume of literature evaluating ruxolitinib, have led to a better understanding of the disease and improved management in general. Here we review ruxolitinib efficacy in patients with MF in the 10 years following approval, including demonstration of clinical benefit in the phase 3 COMFORT-I/II trials, real-world evidence, translational studies, and expanded access data. Lastly, future directions for MF treatment are discussed, including ruxolitinib-based combination therapies. © 2023 The Author(s). Published by Informa UK Limited, trading as Taylor & Francis Group.
Keywords: myelofibrosis; janus kinase; myeloproliferative neoplasm; ruxolitinib
Journal Title: Leukemia and Lymphoma
Volume: 64
Issue: 6
ISSN: 1042-8194
Publisher: Taylor & Francis Group  
Date Published: 2023-06-01
Start Page: 1063
End Page: 1081
Language: English
DOI: 10.1080/10428194.2023.2196593
PUBMED: 37081809
PROVIDER: scopus
DOI/URL:
Notes: Review -- Source: Scopus
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  1. Raajit Kumar Rampal
    339 Rampal