{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,5,19]],"date-time":"2026-05-19T15:59:08Z","timestamp":1779206348391,"version":"3.51.4"},"reference-count":31,"publisher":"Wiley","issue":"4","license":[{"start":{"date-parts":[[2021,3,29]],"date-time":"2021-03-29T00:00:00Z","timestamp":1616976000000},"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":[[2021,6]]},"abstract":"<jats:title>Abstract<\/jats:title><jats:sec><jats:title>Background<\/jats:title><jats:p>ALD is a rare X\u2010linked peroxisomal metabolic disorder with many distinct phenotypes of disease that emerge on a wide scale from adrenal insufficiency to fatal cALD which progresses to a vegetative state within a few years. Currently, HSCT is the only treatment method known to stabilize disease progression in patients with cALD. In this study, we aim to report our HSCT experience in patients with cALD and the factors that determine the success of HSCT, as a single\u2010center experience.<\/jats:p><\/jats:sec><jats:sec><jats:title>Methods<\/jats:title><jats:p>The study cohort involves 23 boys with cALD and three patients with ALD trait and new\u2010onset abnormal behavior who underwent allogeneic HSCT between January 2012 and September 2019 in our transplantation center. Loes scoring, NFS, scale and MFD were performed for evaluating the severity of the cerebral disease. The study cohort was divided into two groups according to baseline NFS and Loes score: early\u2010stage (NFS\u00a0\u2264\u00a01 and Loes score &lt;9) and advanced stage (NFS\u00a0&gt;\u00a01 or Loes score \u22659).<\/jats:p><\/jats:sec><jats:sec><jats:title>Results<\/jats:title><jats:p>The pretransplant stage of disease impacted both OS and MFD\u2010free survival. The estimated OS and MFD\u2010free survival at 3\u00a0years in patients with advanced disease were 46.1% (95% CI 19.0\u201373.2) and 23.1% (95% CI 0.2\u201346.0), respectively, and all patients with the early disease were alive (<jats:italic>p<\/jats:italic>: .004) and MFD\u2010free (<jats:italic>p<\/jats:italic>\u00a0&lt;\u00a0.001) at 3\u00a0years.<\/jats:p><\/jats:sec><jats:sec><jats:title>Conclusion<\/jats:title><jats:p>This study demonstrated that early HSCT is vital in patients with cALD. The early\u2010stage disease had a significant survival advantage and free from disease progression after HSCT.<\/jats:p><\/jats:sec>","DOI":"10.1111\/petr.14015","type":"journal-article","created":{"date-parts":[[2021,3,29]],"date-time":"2021-03-29T15:39:04Z","timestamp":1617032344000},"update-policy":"https:\/\/doi.org\/10.1002\/crossmark_policy","source":"Crossref","is-referenced-by-count":7,"title":["Allogeneic hematopoietic stem cell transplantation in patients with childhood cerebral adrenoleukodystrophy: A single\u2010center experience \u201cBetter prognosis in earlier stage\u201d"],"prefix":"10.1111","volume":"25","author":[{"ORCID":"https:\/\/orcid.org\/0000-0003-3454-9870","authenticated-orcid":false,"given":"Koray","family":"Yalcin","sequence":"first","affiliation":[{"name":"Medicalpark Goztepe Hospital Pediatric Stem Cell Transplantation Unit Istanbul Turkey"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Suna S.","family":"\u00c7elen","sequence":"additional","affiliation":[{"name":"Medicalpark Goztepe Hospital Pediatric Stem Cell Transplantation Unit Istanbul Turkey"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Hayriye","family":"Daloglu","sequence":"additional","affiliation":[{"name":"Medicalpark Antalya Hospital Pediatric Stem Cell Transplantation Unit Antalya Turkey"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Mustafa Kemal","family":"Demir","sequence":"additional","affiliation":[{"name":"Department of Radiology G\u00f6ztepe Medical Park Training and Education Hospital Bah\u00e7e\u015fehir University School of Medicine Istanbul Turkey"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Seda","family":"\u00d6zt\u00fcrkmen","sequence":"additional","affiliation":[{"name":"Medicalpark Antalya Hospital Pediatric Stem Cell Transplantation Unit Antalya 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