{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,9,21]],"date-time":"2026-09-21T16:59:14Z","timestamp":1790009954954,"version":"4.0.1"},"reference-count":28,"publisher":"Wiley","issue":"3","license":[{"start":{"date-parts":[[2024,4,11]],"date-time":"2024-04-11T00:00:00Z","timestamp":1712793600000},"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":["Pediatric Transplantation"],"published-print":{"date-parts":[[2024,5]]},"abstract":"<jats:title>Abstract<\/jats:title><jats:sec><jats:title>Objective<\/jats:title><jats:p>We investigated the safety and efficacy of haploidentical stem cell transplantation (SCT) in pediatric patients with X\u2010linked adrenoleukodystrophy (ALD).<\/jats:p><\/jats:sec><jats:sec><jats:title>Methods<\/jats:title><jats:p>A retrospective analysis of transplantation data from 29 cases of ALD, treated between December 2014 and April 2022, was conducted. Neurologic function scores (NFS) were assessed. The conditioning regimen was busulfan 9.6\u2009mg\/kg, cyclophosphamide 200\u2009mg\/kg, and fludarabine 90\u2009mg\/m<jats:sup>2<\/jats:sup> (BFC). Graft\u2010versus\u2010host disease prophylaxis consisted of anti\u2010human thymocyte globulin, cyclosporine A, mycophenolate mofetil, and short course of methotrexate.<\/jats:p><\/jats:sec><jats:sec><jats:title>Results<\/jats:title><jats:p>Among the 29 cases, 14 cases (NFS\u2009=\u20090) were asymptomatic, and 15 (NFS\u2009\u2265\u20091) were symptomatic. The median age at SCT was 8\u2009years (range: 4\u201316\u2009years); the median follow\u2010up time was 1058\u2009days (range: 398\u20133092\u2009days); 28 cases were father donors and 1 case was a grandfather donor. Hematopoietic reconstitution was successful in all patients, and all of them achieved complete donor chimerism at the time of engraftment. The leading cause of death was still primary disease progression (<jats:italic>n<\/jats:italic>\u2009=\u20094). Survival free of major functional disabilities was 100% in asymptomatic patients versus 66.67% in the symptomatic group (<jats:italic>p<\/jats:italic>\u2009=\u2009.018).<\/jats:p><\/jats:sec><jats:sec><jats:title>Conclusion<\/jats:title><jats:p>BFC regimen used in haploidentical SCT was administered safely without major transplant\u2010related complications even in symptomatic patients, and neurological symptoms were stabilized after SCT.<\/jats:p><\/jats:sec>","DOI":"10.1111\/petr.14735","type":"journal-article","created":{"date-parts":[[2024,4,11]],"date-time":"2024-04-11T09:39:23Z","timestamp":1712828363000},"update-policy":"https:\/\/doi.org\/10.1002\/crossmark_policy","source":"Crossref","is-referenced-by-count":5,"title":["Haploidentical hematopoietic stem cell transplantation with busulfan, cyclophosphamide, and fludarabine conditioning for X\u2010linked adrenal cerebral leukodystrophy"],"prefix":"10.1111","volume":"28","author":[{"given":"Yao","family":"Chen","sequence":"first","affiliation":[{"name":"Peking University People's Hospital  Beijing China"},{"name":"Peking University Institute of Hematology  Beijing China"},{"name":"National Clinical Research Center for Hematologic Disease  Beijing China"},{"name":"Beijing Key Laboratory of Hematopoietic Stem Cell Transplantation  Beijing China"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Lan\u2010ping","family":"Xu","sequence":"additional","affiliation":[{"name":"Peking University People's Hospital  Beijing China"},{"name":"Peking University Institute of Hematology  Beijing China"},{"name":"National Clinical Research Center for Hematologic Disease  Beijing China"},{"name":"Beijing Key Laboratory of Hematopoietic Stem Cell Transplantation  Beijing 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