{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,9,16]],"date-time":"2026-09-16T17:09:24Z","timestamp":1789578564605,"version":"build-2803163510"},"reference-count":22,"publisher":"Wiley","issue":"4","license":[{"start":{"date-parts":[[2023,2,14]],"date-time":"2023-02-14T00:00:00Z","timestamp":1676332800000},"content-version":"vor","delay-in-days":0,"URL":"http:\/\/onlinelibrary.wiley.com\/termsAndConditions#vor"}],"content-domain":{"domain":["onlinelibrary.wiley.com"],"crossmark-restriction":true},"short-container-title":["Br J Haematol"],"published-print":{"date-parts":[[2023,5]]},"abstract":"<jats:title>Summary<\/jats:title><jats:p>Patients with acute megakaryoblastic leukaemia of Down syndrome (DS\u2010AMKL) have an excellent survival rate; however, patients with non\u2010DS\u2010AMKL experience poor outcomes. Therefore, this study retrospectively analysed 203 children with non\u2010DS\u2010AMKL who underwent their first haematopoietic cell transplantation (HCT) from 1986 to 2015 using a nationwide Japanese HCT registry data to assess HCT outcomes for non\u2010DS\u2010AMKL. The 5\u2010year overall survival (OS) and event\u2010free survival (EFS) rates were 43% and 38% respectively. The 5\u2010year OS rate was significantly higher for patients who underwent HCT in the first complete remission (CR1, 72%) than for those in the second CR (CR2, 23%) and non\u2010CR (16%) (<jats:italic>p<\/jats:italic>\u2009&lt;\u20090.001), and for those from a human leukocyte antigen (HLA)\u2010matched (52%) than for those from an HLA\u2010mismatched donor (27%) (<jats:italic>p<\/jats:italic>\u2009&lt;\u20090.001). Multivariate analysis for OS revealed that HCT in CR2 and non\u2010CR was a significant risk factor (hazard ratio, 5.86; 95% confidence interval, 3.56\u20139.53; <jats:italic>p<\/jats:italic>\u2009&lt;\u20090.001). The 3\u2010year EFS in patients who received HCT in CR1 using reduced\u2010intensity conditioning (RIC, 35%) was significantly lower than in those using myeloablative conditioning (busulfan\u2010based, 71%; total body irradiation\u2010based, 58%) (<jats:italic>p<\/jats:italic>\u2009&lt;\u20090.001). Risk stratification in patients with non\u2010DS\u2010AMKL should be established to determine HCT indication in CR1.<\/jats:p>","DOI":"10.1111\/bjh.18691","type":"journal-article","created":{"date-parts":[[2023,2,14]],"date-time":"2023-02-14T09:40:26Z","timestamp":1676367626000},"page":"747-756","update-policy":"https:\/\/doi.org\/10.1002\/crossmark_policy","source":"Crossref","is-referenced-by-count":8,"title":["Haematopoietic cell transplantation for children with acute megakaryoblastic leukaemia without Down syndrome"],"prefix":"10.1111","volume":"201","author":[{"ORCID":"https:\/\/orcid.org\/0000-0002-7582-0201","authenticated-orcid":false,"given":"Asahito","family":"Hama","sequence":"first","affiliation":[{"name":"Department of Haematology and Oncology Children's Medical Centre, Japanese Red Cross Aichi Medical Centre Nagoya First Hospital  Nagoya 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Hospital  Izumi Japan"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Keiko","family":"Okada","sequence":"additional","affiliation":[{"name":"Department of Paediatric Hematology\/Oncology Osaka City General Hospital  Osaka Japan"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Katsuyoshi","family":"Koh","sequence":"additional","affiliation":[{"name":"Department of Haematology\/Oncology Saitama Children's Medical Centre  Saitama Japan"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Tetsuo","family":"Mitsui","sequence":"additional","affiliation":[{"name":"Department of Paediatrics Yamagata University Hospital  Yamagata Japan"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Yoshiyuki","family":"Takahashi","sequence":"additional","affiliation":[{"name":"Department of Paediatrics Nagoya University Graduate School of Medicine  Nagoya 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Aichi Medical University School of Medicine  Nagakute Japan"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0003-4412-6441","authenticated-orcid":false,"given":"Yasuhiro","family":"Okamoto","sequence":"additional","affiliation":[{"name":"Department of Paediatrics Kagoshima University Graduate School of Medical and Dental Sciences  Kagoshima 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