Myelofibrosis is characterized by progressive cytopenias, bone marrow fibrosis, splenomegaly and severe constitutional symptoms. In the phase 3 Controlled Myelofibrosis Study with Oral JAK Inhibitor Treatment (COMFORT) studies, ruxolitinib, a potent Janus kinase 1 (JAK1)/JAK2 inhibitor, provided substantial improvements in splenomegaly, symptoms, quality-of-life measures and overall survival compared with placebo or best available therapy. No assessments of the efficacy and safety of ruxolitinib have been conducted in Asian patients. Here, we describe results from an open-label, single-arm, phase 2 trial evaluating ruxolitinib in Asian patients with myelofibrosis (n = 120). The primary endpoint was met, with 31.7% of patients achieving a ≥ 35% reduction from baseline spleen volume at week 24. As measured by the 7-day Myelofibrosis Symptom Assessment Form v2.0, 49% of patients achieved a ≥ 50% reduction from baseline in total symptom score. Adverse events were consistent with those seen in the COMFORT studies. Ruxolitinib was well tolerated in Asian patients with myelofibrosis and provided substantial reductions in splenomegaly and improvements in symptoms.
Bibliographical noteFunding Information:
This study was sponsored by Novartis Pharmaceuticals and conducted in compliance with the guidelines from the US Food and Drug Administration ’s Office of Good Clinical Practice and the principles of the Declaration of Helsinki. The study protocol was reviewed by each participating center’s institutional review board and written informed consent was obtained from each patient. The first draft of the manuscript was provided by the first author with the assistance of a medical writer funded by the sponsor; the first author made the final decision for publication. All authors and sponsor representatives reviewed the manuscript and attest to the integrity of the data and adherence to the planned protocol and statistical analyses.
The authors wish to thank Dr. Mineo Kurokawa for his contributions as a study investigator (University of Tokyo Hospital, Tokyo, Japan). The authors would also like to thank the patients and their families for their participation, and medical staff from participating institutions for their contributions to this study. Editorial support was provided by Kia C. Walcott, PhD, and was funded by Novartis.
© 2015 The Author(s).
All Science Journal Classification (ASJC) codes
- Cancer Research