{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,9,8]],"date-time":"2026-09-08T16:57:40Z","timestamp":1788886660737,"version":"build-2803163510"},"reference-count":33,"publisher":"American Society of Hematology","issue":"5","content-domain":{"domain":["ashpublications.org"],"crossmark-restriction":true},"short-container-title":[],"published-print":{"date-parts":[[2022,3,8]]},"abstract":"<jats:title>Abstract<\/jats:title><jats:p>Allogeneic hematopoietic stem cell transplantation (allo-HSCT) in early cerebral adrenoleukodystrophy can stabilize neurologic function and improve survival but has associated risks including transplant-related mortality (TRM), graft failure, and graft-versus-host disease (GVHD). An observational study of 59 patients with median age at allo-HSCT of 8 years addressed impact of donor source, donor match, conditioning regimen, and cerebral disease stage on first allo-HSCT outcomes. Efficacy analyses included 53 patients stratified by disease category: advanced disease (AD; n = 16) with Loes score &amp;gt;9 or neurological function score (NFS) &amp;gt;1 and 2 early disease (ED) cohorts (ED1 [Loes \u22644 and NFS \u22641; n = 24] and ED2 [Loes &amp;gt;4-9 and NFS \u22641; n = 13]). Survival free of major functional disabilities and without second allo-HSCT at 4 years was significantly higher in the ED (66%) vs AD (41%) cohort (P = .015) and comparable between ED1 and ED2 cohorts (P = .991). The stabilization of neurologic function posttransplant was greater in the ED vs AD cohort, with a median change from baseline at 24 months after allo-HSCT in NFS and Loes score, respectively, of 0 and 0.5 in ED1 (n = 13), 0.5 and 0 in ED2 (n = 6), and 2.5 and 3.0 (n = 4) in AD cohort. TRM was lower in the ED (7%) compared with the AD (22%) cohort; however, the difference was not significant (P = .094). Transplant-related safety outcomes were also affected by transplant-related characteristics: graft failure incidence was significantly higher with unrelated umbilical cord grafts vs matched related donors (P = .039), and acute GVHD and graft failure incidences varied by conditioning regimen. This study was registered at www:\/\/clinicaltrials.gov as #NCT02204904.<\/jats:p>","DOI":"10.1182\/bloodadvances.2021005294","type":"journal-article","created":{"date-parts":[[2021,11,15]],"date-time":"2021-11-15T23:01:31Z","timestamp":1637017291000},"page":"1512-1524","update-policy":"https:\/\/doi.org\/10.1182\/blood.2019cm0000","source":"Crossref","is-referenced-by-count":40,"title":["Variables affecting outcomes after allogeneic hematopoietic stem cell transplant for cerebral adrenoleukodystrophy"],"prefix":"10.1182","volume":"6","author":[{"given":"Robert","family":"Chiesa","sequence":"first","affiliation":[{"name":"Bone Marrow Transplant Department, Great Ormond Street Hospital for Children, London, UK;"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0003-2232-6952","authenticated-orcid":false,"given":"Jaap Jan","family":"Boelens","sequence":"additional","affiliation":[{"name":"Stem Cell Transplantation andCellular Therapies, Department of Pediatrics, Memorial Sloan-Kettering Cancer Center, New York, NY;"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Christine N.","family":"Duncan","sequence":"additional","affiliation":[{"name":"Department of Pediatric Oncology, Boston Children's Hospital and Dana-Farber Cancer Institute, Boston, MA;"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"J\u00f6rn-Sven","family":"K\u00fchl","sequence":"additional","affiliation":[{"name":"Stem Cell Transplantation, Department of Pediatric Oncology, Hematology and Hemostasis, University Hospital Leipzig, Leipzig, Germany;"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Caroline","family":"Sevin","sequence":"additional","affiliation":[{"name":"Pediatric Neurology Department, Leukodystrophies Reference Center, H\u00f4pital Universitaire H\u00f4pital Bic\u00eatre-H\u00f4pitaux Universitaires Paris Sud, Paris, France;"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Neena","family":"Kapoor","sequence":"additional","affiliation":[{"name":"Hematology, Oncology and Blood & Marrow Transplantation, Department of Pediatrics, Children's Hospital Los Angeles, Los Angeles, CA;"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0001-8151-8212","authenticated-orcid":false,"given":"Vinod K.","family":"Prasad","sequence":"additional","affiliation":[{"name":"Division of Pediatric Transplant and Cellular Therapy, Department of Pediatrics, Duke University Medical Center, Durham, NC;"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Caroline A.","family":"Lindemans","sequence":"additional","affiliation":[{"name":"Pediatric Blood and Bone Marrow Transplantation Program, Princess M\u00e1xima Center for Pediatric Oncology, Utrecht, the Netherlands;"},{"name":"Department of Pediatrics, University Medical Center Utrecht, UU, Utrecht, the Netherlands;"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Simon A.","family":"Jones","sequence":"additional","affiliation":[{"name":"Paediatric Inborn Errors of Metabolism, Manchester University NHS Foundation Trust, Manchester, UK;"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Hernan M.","family":"Amartino","sequence":"additional","affiliation":[{"name":"Child Neurology Service, Hospital Universitario Austral, Buenos Aires, Argentina;"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Mattia","family":"Algeri","sequence":"additional","affiliation":[{"name":"Department ofPediatric Hematology and Oncology, Cell and Gene Therapy,Bambino Ges\u00f9Children's Hospital,IRRCS, Rome, Italy;"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Nancy","family":"Bunin","sequence":"additional","affiliation":[{"name":"Cellular Therapy and Transplant Section, Division of Oncology, Children's Hospital of Philadelphia, Philadelphia, PA;"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0001-8086-296X","authenticated-orcid":false,"given":"Cristina","family":"Diaz-de-Heredia","sequence":"additional","affiliation":[{"name":"Paediatric Oncology and Haematology Department, Hospital Universitari Vall d'Hebron and Vall d'Hebron Institut de Recerca, Barcelona, Spain;"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Daniel J.","family":"Loes","sequence":"additional","affiliation":[{"name":"Midwest Radiology, Minneapolis, MN;"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Esther","family":"Shamir","sequence":"additional","affiliation":[{"name":"bluebird bio, Inc., Cambridge, MA; 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