Efficacy and safety of ruxolitinib in Asian patients with myelofibrosis

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

doi:10.3109/10428194.2014.969260

Efficacy and safety of ruxolitinib in Asian patients with myelofibrosis

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 HouWu Depei, Katsuto Takenaka, Ting Liu, Yu Hu, Taro Amagasaki, Kazuo Ito, Prashanth Gopalakrishna, Koichi Akashi

Department of Internal Medicine

Research output: Contribution to journal › Article › peer-review

24 Citations (Scopus)

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.

Original language	English
Pages (from-to)	2067-2074
Number of pages	8
Journal	Leukemia and Lymphoma
Volume	56
Issue number	7
DOIs	https://doi.org/10.3109/10428194.2014.969260
Publication status	Published - 2015 Jul 1

Bibliographical note

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).

All Science Journal Classification (ASJC) codes

Hematology
Oncology
Cancer Research

UN SDGs

This output contributes to the following UN Sustainable Development Goals (SDGs)

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10.3109/10428194.2014.969260

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Jung, C. W., Shih, L. Y., Xiao, Z., Jie, J., Hou, H. A., Du, X., Wang, M. C., Park, S., Eom, K. S., Oritani, K., Okamoto, S., Tauchi, T., Kim, J. S., Zhou, D., Saito, S., Li, J., Handa, H., Jianyong, L., Ohishi, K., ... Akashi, K. (2015). Efficacy and safety of ruxolitinib in Asian patients with myelofibrosis. Leukemia and Lymphoma, 56(7), 2067-2074. https://doi.org/10.3109/10428194.2014.969260

@article{b63ed0690aaf4dd5808d3febfa9948f2,

title = "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.",

author = "Jung, {Chul Won} and Shih, {Lee Yung} and Zhijian Xiao and Jin Jie and Hou, {Hsin An} and Xin Du and Wang, {Ming Chung} and Seonyang Park and Eom, {Ki Seong} and Kenji Oritani and Shinichiro Okamoto and Tetsuzo Tauchi and Kim, {Jin Seok} and Daobin Zhou and Shigeki Saito and Junmin Li and Hiroshi Handa and Li Jianyong and Kohshi Ohishi and Ming Hou and Wu Depei and Katsuto Takenaka and Ting Liu and Yu Hu and Taro Amagasaki and Kazuo Ito and Prashanth Gopalakrishna and Koichi Akashi",

note = "Funding Information: This study was sponsored by Novartis Pharmaceuticals and conducted in compliance with the guidelines from the US Food and Drug Administration {\textquoteright}s Office of Good Clinical Practice and the principles of the Declaration of Helsinki. The study protocol was reviewed by each participating center{\textquoteright}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: {\textcopyright} 2015 The Author(s).",

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doi = "10.3109/10428194.2014.969260",

language = "English",

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Jung, CW, Shih, LY, Xiao, Z, Jie, J, Hou, HA, Du, X, Wang, MC, Park, S, Eom, KS, Oritani, K, Okamoto, S, Tauchi, T, Kim, JS, Zhou, D, Saito, S, Li, J, Handa, H, Jianyong, L, Ohishi, K, Hou, M, Depei, W, Takenaka, K, Liu, T, Hu, Y, Amagasaki, T, Ito, K, Gopalakrishna, P & Akashi, K 2015, 'Efficacy and safety of ruxolitinib in Asian patients with myelofibrosis', Leukemia and Lymphoma, vol. 56, no. 7, pp. 2067-2074. https://doi.org/10.3109/10428194.2014.969260

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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.

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Efficacy and safety of ruxolitinib in Asian patients with myelofibrosis

Abstract

Bibliographical note

All Science Journal Classification (ASJC) codes

UN SDGs

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