{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2025,7,30]],"date-time":"2025-07-30T14:12:38Z","timestamp":1753884758163,"version":"3.41.2"},"reference-count":22,"publisher":"Wiley","issue":"1","license":[{"start":{"date-parts":[[2021,10,18]],"date-time":"2021-10-18T00:00:00Z","timestamp":1634515200000},"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":[[2022,2]]},"abstract":"<jats:title>Abstract<\/jats:title><jats:sec><jats:title>Background<\/jats:title><jats:p>Adrenoleukodystrophy (ALD) is an X\u2010linked recessive disorder and 30\u201340% of patients develop progressive cerebral neurodegeneration. For symptomatic ALD patients, allogeneic stem cell transplantation (SCT) is considered the standard treatment modality to stabilize or prevent the progression of neurological symptoms.<\/jats:p><\/jats:sec><jats:sec><jats:title>Methods<\/jats:title><jats:p>We retrospectively analyzed the transplant outcomes of 99 pediatric patients with cerebral ALD in Japan. The conditioning regimens included Regimen A: fludarabine\/melphalan\/low\u2010dose total body irradiation (TBI) with brain sparing (<jats:italic>n<\/jats:italic>\u00a0=\u00a039), Regimen B; busulfan\/cyclophosphamide\u00a0\u00b1\u00a0others (<jats:italic>n<\/jats:italic>\u00a0=\u00a023), Regimen C: melphalan\/total lymphoid irradiation\/thoracoabdominal irradiation\u00a0\u00b1\u00a0anti\u2010T lymphocyte globulin\u00a0\u00b1\u00a0fludarabine (<jats:italic>n<\/jats:italic>\u00a0=\u00a027), and Regimen D: others (<jats:italic>n<\/jats:italic>\u00a0=\u00a010).<\/jats:p><\/jats:sec><jats:sec><jats:title>Results<\/jats:title><jats:p>The 5\u2010year overall survival (OS) and event\u2010free survival (EFS) of all patients were 90.0% and 72.9%, respectively. The 5\u2010year OS was 100.0% for Regimen A, 91.1% for Regimen B, 84.4% for Regimen C, and 67.5% for Regimen D (<jats:italic>p<\/jats:italic>\u00a0=\u00a00.028). The 5\u2010year EFS was 78.3% for Regimen A, 78.0% for Regimen B, 70.4% for Regimen C, and 48.0% for Regimen D (<jats:italic>p<\/jats:italic>\u00a0=\u00a00.304). The OS marginally improved after 2007 compared with before 2006 (95.3% vs. 85.2%, <jats:italic>p<\/jats:italic>\u00a0=\u00a00.066), due to the improvement of cord blood transplantation (CBT) outcomes after 2007 compared with before 2006 (96.6% vs. 68.4%, <jats:italic>p<\/jats:italic>\u00a0=\u00a00.005). On magnetic resonance imaging of the brain, a reduced Loes score after SCT was only observed in one of the 15 bone marrow transplantation (BMT) patients, but in 5 of the 15 CBT patients (<jats:italic>p<\/jats:italic>\u00a0=\u00a00.173).<\/jats:p><\/jats:sec><jats:sec><jats:title>Conclusions<\/jats:title><jats:p>Our study revealed that a reduced conditioning regimen with fludarabine\/melphalan\/low\u2010dose TBI provides better outcomes, and the results of CBT significantly improved after 2007.<\/jats:p><\/jats:sec>","DOI":"10.1111\/petr.14125","type":"journal-article","created":{"date-parts":[[2021,10,21]],"date-time":"2021-10-21T07:37:11Z","timestamp":1634801831000},"update-policy":"https:\/\/doi.org\/10.1002\/crossmark_policy","source":"Crossref","is-referenced-by-count":4,"title":["Stem cell transplantation for pediatric patients with adrenoleukodystrophy: A nationwide retrospective analysis in Japan"],"prefix":"10.1111","volume":"26","author":[{"ORCID":"https:\/\/orcid.org\/0000-0002-7397-1575","authenticated-orcid":false,"given":"Koji","family":"Kato","sequence":"first","affiliation":[{"name":"Department of Hematology and Oncology Children's Medical Center Japanese Red Cross Nagoya First Hospital Nagoya Japan"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Hiromasa","family":"Yabe","sequence":"additional","affiliation":[{"name":"Division of Stem Cell Transplantation Department of Innovative Medicine Tokai University School of Medicine Isehara Japan"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Nobuyuki","family":"Shimozawa","sequence":"additional","affiliation":[{"name":"Division of Genomics Research Life Science Research Center Gifu University Gifu Japan"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Souichi","family":"Adachi","sequence":"additional","affiliation":[{"name":"Human Health Sciences Kyoto University Kyoto Japan"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Mineo","family":"Kurokawa","sequence":"additional","affiliation":[{"name":"Department of Cell Therapy and Transplantation Medicine The University of Tokyo Hospital Tokyo Japan"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Yoshiko","family":"Hashii","sequence":"additional","affiliation":[{"name":"Department of Pediatrics Osaka International Cancer Institute Osaka Japan"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Atsushi","family":"Sato","sequence":"additional","affiliation":[{"name":"Department of Hematology and Oncology Miyagi Children\u2019s Hospital Sendai Japan"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Nao","family":"Yoshida","sequence":"additional","affiliation":[{"name":"Department of Hematology and Oncology Children's Medical Center Japanese Red Cross Nagoya First Hospital Nagoya Japan"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Makiko","family":"Kaga","sequence":"additional","affiliation":[{"name":"Department of Developmental Disorders National Institute of Mental Health National Center of Neurology and Psychiatry Tokyo Japan"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Osamu","family":"Onodera","sequence":"additional","affiliation":[{"name":"Department of Neurology Clinical Neuroscience Branch Brain Research Institute Niigata University Niigata Japan"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Shunichi","family":"Kato","sequence":"additional","affiliation":[{"name":"Division of Stem Cell Transplantation Department of Innovative Medicine Tokai University School of Medicine Isehara Japan"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Yoshiko","family":"Atsuta","sequence":"additional","affiliation":[{"name":"Japanese Data Center for Hematopoietic Cell Transplantation Nagoya Japan"},{"name":"Department of Registry Science for Transplant and Cellular Therapy Aichi Medical University School of Medicine Nagakute Japan"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Tomohiro","family":"Morio","sequence":"additional","affiliation":[{"name":"Department of Pediatrics and Developmental Biology Tokyo Medical and Dental University Tokyo Japan"}],"role":[{"role":"author","vocabulary":"crossref"}]}],"member":"311","published-online":{"date-parts":[[2021,10,18]]},"reference":[{"key":"e_1_2_10_2_1","doi-asserted-by":"publisher","DOI":"10.1093\/brain\/120.8.1485"},{"key":"e_1_2_10_3_1","doi-asserted-by":"publisher","DOI":"10.1016\/S1474-4422(07)70177-1"},{"key":"e_1_2_10_4_1","first-page":"1761","article-title":"Adrenoleukodystrophy: a scoring method for brain MR observations","volume":"15","author":"Loes DJ","year":"1994","journal-title":"Am J 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