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However, conventional graft\u2010versus\u2010host disease (GVHD) prophylaxis based on calcineurin inhibitors like ciclosporin (CsA) poses a potential neurotoxicity risk that may exacerbate neurological injury. We retrospectively analysed 26 cALD patients who underwent haplo\u2010HSCT with an optimized ciclosporin\u2010free GVHD prophylaxis regimen consisting of anti\u2010thymocyte globulin (ATG; 4\u2009mg\/kg), post\u2010transplant cyclophosphamide (PTCy) and mycophenolate mofetil. Neutrophil and platelet engraftment each occurred at a median of 15\u2009days (ranges, 13\u201326 and 9\u201333\u2009days respectively). The cumulative incidence (CI) of grades II\u2013IV acute GVHD by day 100 was 18.49%\u2009\u00b1\u20098.37%, and the 3\u2010year CI of moderate\u2010to\u2010severe chronic GVHD was 9.32%\u2009\u00b1\u20096.28%. After a median follow\u2010up of 29\u2009months, overall survival was 90.4%\u2009\u00b1\u20096.6% and major functional disability\u2010free survival was 69.0%\u2009\u00b1\u20099.1%. Post\u2010transplantation, plasma C26:0 levels and the C24:0\/C22:0 and C26:0\/C22:0 ratios were markedly reduced. Magnetic resonance imaging (MRI) at a mean of 7.3\u2009\u00b1\u20095.9\u2009months post\u2010HSCT showed largely preserved LOES scores without significant progression. Our experience supports the safety and efficacy of this optimized ciclosporin\u2010free haplo\u2010HSCT approach for patients with cALD, warranting further validation in prospective studies.<\/jats:p>","DOI":"10.1111\/bjh.70561","type":"journal-article","created":{"date-parts":[[2026,5,19]],"date-time":"2026-05-19T05:50:21Z","timestamp":1779169821000},"page":"660-669","update-policy":"https:\/\/doi.org\/10.1002\/crossmark_policy","source":"Crossref","is-referenced-by-count":0,"title":["Outcomes of an optimized ciclosporin\u2010free haploidentical\n                    <scp>HSCT<\/scp>\n                    protocol in paediatric patients with cerebral adrenoleukodystrophy"],"prefix":"10.1111","volume":"209","author":[{"given":"Fanghua","family":"Ye","sequence":"first","affiliation":[{"name":"Department of Pediatrics Xiangya Hospital Central South University  Changsha 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