TY - JOUR
T1 - Efficacy and safety of ruxolitinib in Asian patients with myelofibrosis
AU - Jung, Chul Won
AU - Shih, Lee Yung
AU - Xiao, Zhijian
AU - Jie, Jin
AU - Hou, Hsin An
AU - Du, Xin
AU - Wang, Ming Chung
AU - Park, Seonyang
AU - Eom, Ki Seong
AU - Oritani, Kenji
AU - Okamoto, Shinichiro
AU - Tauchi, Tetsuzo
AU - Kim, Jin Seok
AU - Zhou, Daobin
AU - Saito, Shigeki
AU - Li, Junmin
AU - Handa, Hiroshi
AU - Jianyong, Li
AU - Ohishi, Kohshi
AU - Hou, Ming
AU - Depei, Wu
AU - Takenaka, Katsuto
AU - Liu, Ting
AU - Hu, Yu
AU - Amagasaki, Taro
AU - Ito, Kazuo
AU - Gopalakrishna, Prashanth
AU - Akashi, Koichi
N1 - Funding 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.
Funding Information:
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.
Publisher Copyright:
© 2015 The Author(s).
PY - 2015/7/1
Y1 - 2015/7/1
N2 - 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.
AB - 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.
KW - Asian patient population
KW - JAK1/JAK2 inhibitor
KW - Myelofibrosis
KW - Phase 2
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UR - http://www.scopus.com/inward/citedby.url?scp=84938117732&partnerID=8YFLogxK
U2 - 10.3109/10428194.2014.969260
DO - 10.3109/10428194.2014.969260
M3 - Article
C2 - 25315076
AN - SCOPUS:84938117732
SN - 1042-8194
VL - 56
SP - 2067
EP - 2074
JO - Leukemia and Lymphoma
JF - Leukemia and Lymphoma
IS - 7
ER -
