title: Clinical features and outcomes of patients with primary myelofibrosis in Japan: report of a 17-year nationwide survey by the Idiopathic Disorders of Hematopoietic Organs Research Committee of Japan.
abstract: We conducted a 17-year nationwide survey (1999-2015) to elucidate the clinical outcomes of patients with primary myelofibrosis (PMF) in Japan. Questionnaires were sent annually to approximately 500 hematology departments. Newly diagnosed patients with PMF were enrolled in this study, and were followed up annually to collect prognostic information. Approximately 50 patients were enrolled per year, yielding a total of 780 patients with PMF included in this study. The median age at diagnosis was 66 years. At the time of analysis, the median survival duration was 47 months, and the 3-year overall survival rate was 59 %. Infection and disease transformation into acute leukemia were the most frequent causes of death. Of the proposed prognostic models for predicting the outcomes of PMF patients in Japan, the Dynamic International Prognostic Scoring System of PMF plus model was the most feasible. Forty-three patients received allogeneic hematopoietic stem cell transplantation (alloSCT) at a median of 343 days after diagnosis. This treatment significantly prolonged the survival of PMF patients, and the 3-year overall survival rate after first alloSCT was 84 %. A long-term registration study is required for further evaluation of prognosis and the impact of treatments on survival.
authors.affiliation: Department of Medicine and Biosystemic Science, Kyushu University Graduate School of Medical Sciences, 3-1-1 Maidashi, Higashi-ku, Fukuoka, 812-8582, Japan.
authors.forename: Katsuto
authors.initials: K
authors.lastname: Takenaka
authors.affiliation: Department of Gastroenterology and Hematology, Faculty of Medicine, University of Miyazaki, Miyazaki, Japan.
authors.forename: Kazuya
authors.initials: K
authors.lastname: Shimoda
authors.affiliation: Department of Hematology, Toranomon Hospital, Tokyo, Japan.
authors.forename: Naoyuki
authors.initials: N
authors.lastname: Uchida
authors.affiliation: Department of Hematology, Kumamoto Shinto General Hospital, Kumamoto, Japan.
authors.forename: Taizo
authors.initials: T
authors.lastname: Shimomura
authors.affiliation: Division of Hematology and Oncology, Department of Medicine, Kurume University School of Medicine, Kurume, Japan.
authors.forename: Koji
authors.initials: K
authors.lastname: Nagafuji
authors.affiliation: Department of Hematology and Oncology, Graduate School of Medicine, Kyoto University, Kyoto, Japan.
authors.forename: Tadakazu
authors.initials: T
authors.lastname: Kondo
authors.affiliation: Department of Hematology and Oncology, Osaka University Graduate School of Medicine, Osaka, Japan.
authors.forename: Hirohiko
authors.initials: H
authors.lastname: Shibayama
authors.affiliation: Division of Hematology, Department of Medicine, Keio University, Tokyo, Japan.
authors.forename: Takehiko
authors.initials: T
authors.lastname: Mori
authors.affiliation: Department of Hematology, NTT Medical Center Tokyo, Tokyo, Japan.
authors.forename: Kensuke
authors.initials: K
authors.lastname: Usuki
authors.affiliation: Department of Hematology, Clinical Immunology, and Infectious Diseases, Ehime University Graduate School of Medicine, Ehime, Japan.
authors.forename: Taichi
authors.initials: T
authors.lastname: Azuma
authors.affiliation: Department of Hematology, Hakodate Municipal Hospital, Hokkaido, Japan.
authors.forename: Yutaka
authors.initials: Y
authors.lastname: Tsutsumi
authors.affiliation: Department of Hematology, Tokyo Women's Medical University, Tokyo, Japan.
authors.forename: Junji
authors.initials: J
authors.lastname: Tanaka
authors.affiliation: Department of Medicine and Biosystemic Science, Kyushu University Graduate School of Medical Sciences, 3-1-1 Maidashi, Higashi-ku, Fukuoka, 812-8582, Japan.
authors.forename: Hitomi
authors.initials: H
authors.lastname: Dairaku
authors.affiliation: Division of Molecular Medicine, Aichi Cancer Center Research Institute, Nagoya, Japan.
authors.forename: Keitaro
authors.initials: K
authors.lastname: Matsuo
authors.affiliation: The Institute of Medical Science, The University of Tokyo, Tokyo, Japan.
authors.forename: Keiya
authors.initials: K
authors.lastname: Ozawa
authors.affiliation: Department of Hematology and Oncology, Graduate School of Medicine, The University of Tokyo, Tokyo, Japan.
authors.forename: Mineo
authors.initials: M
authors.lastname: Kurokawa
authors.affiliation: Department of Hematology and Oncology, Graduate School of Medicine, The University of Tokyo, Tokyo, Japan.
authors.forename: Shunya
authors.initials: S
authors.lastname: Arai
authors.affiliation: Department of Medicine and Biosystemic Science, Kyushu University Graduate School of Medical Sciences, 3-1-1 Maidashi, Higashi-ku, Fukuoka, 812-8582, Japan. akashi@med.kyushu-u.ac.jp.
authors.forename: Koichi
authors.initials: K
authors.lastname: Akashi
mesh_terms: D000328:Adult; D000728:Androgens / Q000627:therapeutic use; D000970:Antineoplastic Agents / Q000627:therapeutic use; D015331:Cohort Studies; D017707:Erythrocyte Transfusion; D005260:Female; D018380:Hematopoietic Stem Cell Transplantation; D006801:Humans; D006918:Hydroxyurea / Q000627:therapeutic use; D007564:Japan / Q000453:epidemiology; D059785:Karyotype; D008297:Male; D008875:Middle Aged; D009570:Nitriles; D017713:Platelet Transfusion; D055728:Primary Myelofibrosis / Q000175:diagnosis* / Q000453:epidemiology / Q000235:genetics / Q000628:therapy*; D011379:Prognosis; D011720:Pyrazoles / Q000627:therapeutic use; D011743:Pyrimidines; D013156:Splenectomy; D011795:Surveys and Questionnaires; D015996:Survival Rate; D016896:Treatment Outcome
chemical_list: D000728:Androgens; D000970:Antineoplastic Agents; D009570:Nitriles; D011720:Pyrazoles; D011743:Pyrimidines; C540383:ruxolitinib; D006918:Hydroxyurea
keywords: Long-term outcome; Myelofibrosis; Nationwide survey