Validation of the revised International Prognostic Scoring System in patients with myelodysplastic syndrome in Japan: results from a prospective multicenter registry

Abstract

The Japanese National Research Group on Idiopathic Bone Marrow Failure Syndromes has been conducting prospective registration, central review, and follow-up study for patients with aplastic anemia and myelodysplastic syndrome (MDS) since 2006. Using this database, we retrospectively analyzed the prognosis of patients with MDS. As of May 2016, 351 cases were registered in this database, 186 of which were eligible for the present study. Kaplan–Meier analysis showed that overall survival (OS) curves of the five risk categories stipulated by the revised international prognostic scoring system (IPSS-R) were reasonably separated. 2-year OS rates for the very low-, low-, intermediate-, high-, and very high-risk categories were 95, 89, 79, 35, and 12%, respectively. In the same categories, incidence of leukemic transformation at 2 years was 0, 10, 8, 56, and 40%, respectively. Multivariate analysis revealed that male sex, low platelet counts, increased blast percentage (>2%), and high-risk karyotype abnormalities were independent risk factors for poor OS. Based on these data, we classified Japanese MDS patients who were classified as intermediate-risk in IPSS-R, into the lower risk MDS category, highlighting the need for careful assessment of treatments within low- and high-risk treatment protocols.

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Acknowledgements

The authors thank the patients, their families, all the investigators including Drs Yukiharu Nakabo (The Center for Hematological Diseases, Takeda General Hospital), Takahiko Utsumi (Department of Hematology and Oncology, Shiga Medical Center for Adults), Tatsuo Ichinohe (Department of Hematology and Oncology, Research Institute for Radiation Biology and Medicine, Hiroshima University), Hirohiko Shibayama (Department of Hematology and Oncology, Osaka University Graduate School of Medicine), Yasuyoshi Morita (Division of Hematology and Rheumatology, Department of Internal Medicine, Kindai University Faculty of Medicine), Masayuki Shisek (Department of Hematology, Tokyo Women’s Medical University), Masayoshi Kobune (Department of Hematology, Sapporo Medical University School of Medicine), Naoshi Obara (Department of Hematology, Graduate School of Comprehensive Human Sciences, Tsukuba University), Wataru Takahashi (Department of Hematology and Oncology, Dokkyo Medical University Hospital), and nurses in the participating institutions of this study. The authors also thank Drs. Maki Shindo-Ueda, Kazue Miyamoto-Arimoto, Tasuki Uchiyama, and Ms. Yukiko Takada, and Nana Kawabe for their technical assistance with the construction of the database. This study was supported in part by the National Research Group on Idiopathic Bone Marrow Failure Syndromes, granted by the Ministry of Health, Labor and Welfare, Japan (H26-Nanchi-Ippan-062).

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Correspondence to Hiroshi Kawabata.

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Dr. Kawabata reports Grants from the Ministry of Health, Labor and Welfare, Japan, during the conduct of the study; personal fees from Nippon Shinyaku Co., Ltd., personal fees from Celgene Corporation, outside the submitted work; Dr. Matsuda reports personal fees from Kyowa Hakko Kirin Co., Ltd., personal fees from Nippon Shinyaku Co., Ltd., personal fees from Alexion Pharmaceuticals Inc., personal fees from Sanofi K.K., personal fees from GlaxoSmithKline K.K., outside the submitted work; Dr. Suzuki reports Grants from the Ministry of Health, Labor and Welfare, Japan, during the conduct of the study; personal fees from Nippon Shinyaku Co., Ltd., Grants and personal fees from Kyowa Hakko Kirin Co., Ltd., outside the submitted work; Dr. Usuki reports Grants from Fujimoto Pharmaceutical Corporation, Grants from Astellas Pharma Inc., Grants from Otsuka Pharmaceutical Co., Ltd., Grants from Dainippon Sumitomo Pharma Co., Ltd., Grants from Kyowa Hakko Kirin Co., Ltd., Grants from Daiichi Sankyo Co., Ltd., personal fees from Novartis Pharma K.K., outside the submitted work; Dr. Chiba reports grants from the Ministry of Health, Labor and Welfare, Japan, during the conduct of the study; Grants from Nippon Shinyaku Co., Ltd., personal fees from Celgene Corporation, outside the submitted work; Dr. Arima reports Grants from Nippon Shinyaku Co., Ltd., Grants from Kyowa Hakko Kirin Co., Ltd., Grants from Chugai Pharmaceutical Co., Ltd., Grants from Teijin Pharma Ltd., Grants from CSL Behring K.K., Grants from Japan Blood Products Organization, Grants from Taiho Pharmaceutical Co., Ltd., outside the submitted work; Dr. Ohta reports Grants from the Ministry of Health, Labor and Welfare, Japan, during the conduct of the study; Dr. Miyazaki reports Grants from the Ministry of Health Labour and Welfare, Japan, during the conduct of the study; personal fees from Nippon Shinyaku Co., Ltd., personal fees from Kyowa Hakko Kirin Co., Ltd., personal fees from Celgene Corporation, personal fees from Dainippon Sumitomo Pharma Co., Ltd., personal fees from Novartis Pharma K.K., personal fees from Chugai Pharmaceutical Co. Ltd., outside the submitted work; Dr. Mitani reports Grants and personal fees from Kyowa-Hakko Kirin, Grants and personal fees from Chugai Pharmaceutical Co., Ltd., Grants and personal fees from Novartis Pharma K.K., Grants from Teijin Pharma Ltd., Grants from Japan Blood Products Organization, Grants from Ono Pharmaceutical Co., Ltd., Grants from Takeda Pharmaceutical Co., Ltd., Grants from Toyama Chemical Co., Ltd., Grants from Asteras Pharma Inc., Grants from Taiho Pharmaceutical Co., Ltd., Grants from Pfizer Japan Inc., Grants from Dainippon Sumitomo Pharma Co., Ltd., Grants from MSD K.K., Grants from Mochida Pharmaceutical Co., Ltd., Grants from Nihon Pharmaceutical Co., Ltd., outside the submitted work; Dr. Arai reports Grants from the Ministry of Health, Labor and Welfare, Japan, during the conduct of the study; Grants from Kyowa Hakko Kirin Co., Ltd., Grants from Nippon Shinyaku, Co., Ltd., outside the submitted work; Dr. Kurokawa reports Grants from the Ministry of Health, Labor and Welfare, Japan, during the conduct of the study, Grants from Kyowa Hakko Kirin Pharma Inc., Grants from Nippon Shinyaku, Co., Ltd., Grants from Chugai Pharmaceutical Co., Ltd., outside the submitted work; personal fees from Kyowa Hakko Kirin Pharma Inc., Nippon Shinyaku, Co., Ltd., Celgene Corporation outside the submitted work; Dr. Takaori-Kondo reports Grants from Ministry of Health, Labor and Welfare, Japan, during the conduct of the study; personal fees from Bristol-Myers Squibb, personal fees from Yanssen Pharmaceutical K.K., Grants from Kyowa Hakko Kirin Co., Ltd., Grants from Chugai Pharmaceutical Co., Ltd., Grants from Takeda Pharmaceutical Co., Ltd., outside the submitted work.

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Kawabata, H., Tohyama, K., Matsuda, A. et al. Validation of the revised International Prognostic Scoring System in patients with myelodysplastic syndrome in Japan: results from a prospective multicenter registry. Int J Hematol 106, 375–384 (2017). https://doi.org/10.1007/s12185-017-2250-0

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Keywords

  • Myelodysplastic syndrome
  • International prognostic scoring system
  • Leukemic transformation