Clinical features of children with polycythemia vera, essential thrombocythemia, and primary myelofibrosis in Japan: A retrospective nationwide survey
Abstract Background Philadelphia‐negative (Ph‐negative) myeloproliferative neoplasms (MPNs), including polycythemia vera (PV), essential thrombocythemia (ET), and primary myelofibrosis (PMF), are exceptionally rare during childhood. Thus, clinical features of pediatric Ph‐negative MPNs remain largel...
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Wiley
2020-07-01
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| Series: | eJHaem |
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| Online Access: | https://doi.org/10.1002/jha2.39 |
| _version_ | 1797739939135225856 |
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| author | Hisashi Ishida Yuji Miyajima Nobuyuki Hyakuna Satoru Hamada Takeo Sarashina Risa Matsumura Katsutsugu Umeda Tetsuo Mitsui Naoto Fujita Daisuke Tomizawa Kevin Y. Urayama Yasushi Ishida Takashi Taga Masatoshi Takagi Souichi Adachi Atsushi Manabe Toshihiko Imamura Katsuyoshi Koh Akira Shimada Leukemia/Lymphoma Committee of the Japanese Society of Pediatric Hematology Oncology (JSPHO) |
| author_facet | Hisashi Ishida Yuji Miyajima Nobuyuki Hyakuna Satoru Hamada Takeo Sarashina Risa Matsumura Katsutsugu Umeda Tetsuo Mitsui Naoto Fujita Daisuke Tomizawa Kevin Y. Urayama Yasushi Ishida Takashi Taga Masatoshi Takagi Souichi Adachi Atsushi Manabe Toshihiko Imamura Katsuyoshi Koh Akira Shimada Leukemia/Lymphoma Committee of the Japanese Society of Pediatric Hematology Oncology (JSPHO) |
| author_sort | Hisashi Ishida |
| collection | DOAJ |
| description | Abstract Background Philadelphia‐negative (Ph‐negative) myeloproliferative neoplasms (MPNs), including polycythemia vera (PV), essential thrombocythemia (ET), and primary myelofibrosis (PMF), are exceptionally rare during childhood. Thus, clinical features of pediatric Ph‐negative MPNs remain largely unknown. This study was therefore performed to address this. Methods We performed a retrospective study to collect clinical information of children diagnosed with Ph‐negative MPNs from 2000 to 2016 using questionnaires in qualified institutions in Japan. The results obtained from the questionnaire survey were then combined with those from the national registry data. Results Among 50 children identified, five had PV, 44 had ET, and one had PMF. Median age at diagnosis was 14.0, 9.0, and 0 years, respectively. Male to female ratio was 4:1, 21:23, and 1:0, respectively. Detection rates of the JAK2 V617F variant were 0/5 in PV and 9/39 in ET. Frequencies of complications, such as thrombosis and subsequent leukemia, were lower than complication frequencies in adults. We identified two children who developed subsequent leukemia, which has not been reported previously, and one of them died. Conclusion This is the first nationally representative survey of pediatric Ph‐negative MPNs. Given its rarity, an international collaboration with comprehensive genetic analyses might be needed to fully elucidate the clinical and genetic features. |
| first_indexed | 2024-03-12T14:05:23Z |
| format | Article |
| id | doaj.art-0b18a337d6f4481ea754b98cbaa91e94 |
| institution | Directory Open Access Journal |
| issn | 2688-6146 |
| language | English |
| last_indexed | 2024-03-12T14:05:23Z |
| publishDate | 2020-07-01 |
| publisher | Wiley |
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| series | eJHaem |
| spelling | doaj.art-0b18a337d6f4481ea754b98cbaa91e942023-08-21T14:10:49ZengWileyeJHaem2688-61462020-07-0111869310.1002/jha2.39Clinical features of children with polycythemia vera, essential thrombocythemia, and primary myelofibrosis in Japan: A retrospective nationwide surveyHisashi Ishida0Yuji Miyajima1Nobuyuki Hyakuna2Satoru Hamada3Takeo Sarashina4Risa Matsumura5Katsutsugu Umeda6Tetsuo Mitsui7Naoto Fujita8Daisuke Tomizawa9Kevin Y. Urayama10Yasushi Ishida11Takashi Taga12Masatoshi Takagi13Souichi Adachi14Atsushi Manabe15Toshihiko Imamura16Katsuyoshi Koh17Akira Shimada18Leukemia/Lymphoma Committee of the Japanese Society of Pediatric Hematology Oncology (JSPHO)19Department of Pediatrics Okayama University Hospital Okayama JapanDepartment of Pediatrics Anjo Kosei Hospital Anjo JapanDepartment of Pediatrics University of the Ryukyus Hospital Nishihara JapanDepartment of Pediatrics University of the Ryukyus Hospital Nishihara JapanDepartment of Pediatrics Asahikawa Medical University Asahikawa JapanDepartment of Pediatrics Hiroshima University Hospital Hiroshima JapanDepartment of Pediatrics Graduate School of Medicine Kyoto University Kyoto JapanDepartment of Pediatrics Yamagata University Hospital Yamagata JapanDepartment of Pediatrics Hiroshima Red Cross Hospital and Atomic‐bomb Survivors Hospital Hiroshima JapanChildren's Cancer Centre National Centre for Child Health and Development Tokyo JapanDepartment of Social Medicine National Centre for Child Health and Development Tokyo JapanPediatric Medical Centre Ehime Prefectural Central Hospital Matsuyama JapanDepartment of Pediatrics Shiga University of Medical Science Otsu JapanDepartment of Pediatrics and Developmental Biology Tokyo Medical and Dental University Tokyo JapanDepartment of Human Health Science Kyoto University Kyoto JapanDepartment of Pediatrics Hokkaido University Hospital Sapporo JapanDepartment of Pediatrics Graduate School of Medical Science Kyoto Prefectural University of Medicine Kyoto JapanDepartment of Hematology/Oncology Saitama Children's Medical Centre Saitama JapanDepartment of Pediatrics Okayama University Hospital Okayama JapanDepartment of Pediatrics Okayama University Hospital Okayama JapanAbstract Background Philadelphia‐negative (Ph‐negative) myeloproliferative neoplasms (MPNs), including polycythemia vera (PV), essential thrombocythemia (ET), and primary myelofibrosis (PMF), are exceptionally rare during childhood. Thus, clinical features of pediatric Ph‐negative MPNs remain largely unknown. This study was therefore performed to address this. Methods We performed a retrospective study to collect clinical information of children diagnosed with Ph‐negative MPNs from 2000 to 2016 using questionnaires in qualified institutions in Japan. The results obtained from the questionnaire survey were then combined with those from the national registry data. Results Among 50 children identified, five had PV, 44 had ET, and one had PMF. Median age at diagnosis was 14.0, 9.0, and 0 years, respectively. Male to female ratio was 4:1, 21:23, and 1:0, respectively. Detection rates of the JAK2 V617F variant were 0/5 in PV and 9/39 in ET. Frequencies of complications, such as thrombosis and subsequent leukemia, were lower than complication frequencies in adults. We identified two children who developed subsequent leukemia, which has not been reported previously, and one of them died. Conclusion This is the first nationally representative survey of pediatric Ph‐negative MPNs. Given its rarity, an international collaboration with comprehensive genetic analyses might be needed to fully elucidate the clinical and genetic features.https://doi.org/10.1002/jha2.39essential thrombocythemiamyeloproliferative neoplasmpediatricpolycythemia veraprimary myelofibrosis |
| spellingShingle | Hisashi Ishida Yuji Miyajima Nobuyuki Hyakuna Satoru Hamada Takeo Sarashina Risa Matsumura Katsutsugu Umeda Tetsuo Mitsui Naoto Fujita Daisuke Tomizawa Kevin Y. Urayama Yasushi Ishida Takashi Taga Masatoshi Takagi Souichi Adachi Atsushi Manabe Toshihiko Imamura Katsuyoshi Koh Akira Shimada Leukemia/Lymphoma Committee of the Japanese Society of Pediatric Hematology Oncology (JSPHO) Clinical features of children with polycythemia vera, essential thrombocythemia, and primary myelofibrosis in Japan: A retrospective nationwide survey eJHaem essential thrombocythemia myeloproliferative neoplasm pediatric polycythemia vera primary myelofibrosis |
| title | Clinical features of children with polycythemia vera, essential thrombocythemia, and primary myelofibrosis in Japan: A retrospective nationwide survey |
| title_full | Clinical features of children with polycythemia vera, essential thrombocythemia, and primary myelofibrosis in Japan: A retrospective nationwide survey |
| title_fullStr | Clinical features of children with polycythemia vera, essential thrombocythemia, and primary myelofibrosis in Japan: A retrospective nationwide survey |
| title_full_unstemmed | Clinical features of children with polycythemia vera, essential thrombocythemia, and primary myelofibrosis in Japan: A retrospective nationwide survey |
| title_short | Clinical features of children with polycythemia vera, essential thrombocythemia, and primary myelofibrosis in Japan: A retrospective nationwide survey |
| title_sort | clinical features of children with polycythemia vera essential thrombocythemia and primary myelofibrosis in japan a retrospective nationwide survey |
| topic | essential thrombocythemia myeloproliferative neoplasm pediatric polycythemia vera primary myelofibrosis |
| url | https://doi.org/10.1002/jha2.39 |
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