Efficacy and safety of ruxolitinib in Asian patients with myelofibrosis.

Abstract

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.

• Authors: Chul Won Jung, Lee-Yung Shih, Zhijian Xiao, Jin Jie, Hsin-An Hou, Xin Du, Ming-Chung Wang, Seonyang Park, Ki-Seong Eom, Kenji Oritani, Shinichiro Okamoto, Tetsuzo Tauchi, Jin Seok Kim, Daobin Zhou, Shigeki Saito, Junmin Li, Hiroshi Handa, Li Jianyong, Kohshi Ohishi, Ming Hou, Wu Depei, Katsuto Takenaka, Ting Liu, Yu Hu, Taro Amagasaki, Kazuo Ito, Prashanth Gopalakrishna, Koichi Akashi
• Journal: Leukemia & lymphoma (Leuk Lymphoma) Vol. 56 Issue 7 Pg. 2067-74 (Jul 2015) ISSN: 1029-2403 [Electronic] United States
• PMID: 25315076 (Publication Type: Clinical Trial, Phase II, Journal Article, Multicenter Study, Research Support, Non-U.S. Gov't)

Chemical References

• Nitriles
• Protein Kinase Inhibitors
• Pyrazoles
• Pyrimidines
• ruxolitinib
• JAK1 protein, human
• JAK2 protein, human
• Janus Kinase 1
• Janus Kinase 2

Topics

• Adult
• Aged
• Aged, 80 and over
• Asian People
• Female
• Follow-Up Studies
• Humans
• Janus Kinase 1 (antagonists & inhibitors)
• Janus Kinase 2 (antagonists & inhibitors)
• Male
• Middle Aged
• Neoplasm Staging
• Nitriles
• Primary Myelofibrosis (drug therapy, pathology)
• Prognosis
• Protein Kinase Inhibitors (therapeutic use)
• Pyrazoles (therapeutic use)
• Pyrimidines
• Quality of Life