{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,9,29]],"date-time":"2026-09-29T06:27:18Z","timestamp":1790663238863,"version":"4.1.0"},"reference-count":31,"publisher":"American Medical Association (AMA)","issue":"3","content-domain":{"domain":[],"crossmark-restriction":false},"short-container-title":["JAMA Netw Open"],"DOI":"10.1001\/jamanetworkopen.2018.0769","type":"journal-article","created":{"date-parts":[[2018,7,20]],"date-time":"2018-07-20T12:31:57Z","timestamp":1532089917000},"page":"e180769","source":"Crossref","is-referenced-by-count":43,"title":["Potential Risks to Stable Long-term Outcome of Allogeneic Hematopoietic Stem Cell Transplantation for Children With Cerebral X-linked Adrenoleukodystrophy"],"prefix":"10.1001","volume":"1","author":[{"given":"J\u00f6rn-Sven","family":"K\u00fchl","sequence":"first","affiliation":[{"name":"Department of Pediatric Hematology\/Oncology\/Hemostaseology, University Hospital Leipzig, Leipzig, Germany"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Jana","family":"Kupper","sequence":"additional","affiliation":[{"name":"Department of Pediatric Oncology\/Hematology\/SCT, Charit\u00e9 Campus Virchow-Klinikum, Berlin, Germany"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Hermann","family":"Baqu\u00e9","sequence":"additional","affiliation":[{"name":"Department of Pediatric Neurology, Charit\u00e9 Campus Virchow-Klinikum, Berlin, Germany"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Wolfram","family":"Ebell","sequence":"additional","affiliation":[{"name":"Department of Pediatric Oncology\/Hematology\/SCT, Charit\u00e9 Campus Virchow-Klinikum, Berlin, Germany"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Jutta","family":"G\u00e4rtner","sequence":"additional","affiliation":[{"name":"Department of Pediatric Neurology, University Medical Center G\u00f6ttingen, G\u00f6ttingen, Germany"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Christoph","family":"Korenke","sequence":"additional","affiliation":[{"name":"Department of Pediatric Neurology, Klinikum Oldenburg, Oldenburg, Germany"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Birgit","family":"Spors","sequence":"additional","affiliation":[{"name":"Department of Pediatric Radiology, Charit\u00e9 Campus Virchow-Klinikum, Berlin, Germany"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Ingo G.","family":"Steffen","sequence":"additional","affiliation":[{"name":"Department of Pediatric Oncology\/Hematology\/SCT, Charit\u00e9 Campus Virchow-Klinikum, Berlin, Germany"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Gabriele","family":"Strauss","sequence":"additional","affiliation":[{"name":"Department of Pediatrics, Helios-Klinikum Berlin-Buch, Berlin, Germany"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Sebastian","family":"Voigt","sequence":"additional","affiliation":[{"name":"Department of Pediatric Oncology\/Hematology\/SCT, Charit\u00e9 Campus Virchow-Klinikum, Berlin, Germany"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Bernhard","family":"Weschke","sequence":"additional","affiliation":[{"name":"Department of Pediatric Neurology, Charit\u00e9 Campus Virchow-Klinikum, Berlin, Germany"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Almuth","family":"Weddige","sequence":"additional","affiliation":[{"name":"Department of Pediatric Neurology, University Medical Center G\u00f6ttingen, G\u00f6ttingen, Germany"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Wolfgang","family":"K\u00f6hler","sequence":"additional","affiliation":[{"name":"Department of Neurology, University Hospital Leipzig, Leipzig, Germany"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Robert","family":"Steinfeld","sequence":"additional","affiliation":[{"name":"Department of Pediatric Neurology, University Medical Center G\u00f6ttingen, G\u00f6ttingen, Germany"}],"role":[{"vocabulary":"crossref","role":"author"}]}],"member":"10","published-online":{"date-parts":[[2018,7,20]]},"reference":[{"issue":"pt 8","key":"zoi180058r1","doi-asserted-by":"publisher","first-page":"1485","DOI":"10.1093\/brain\/120.8.1485","article-title":"Adrenoleukodystrophy: phenotype, genetics, pathogenesis and therapy.","volume":"120","author":"Moser","year":"1997","journal-title":"Brain"},{"key":"zoi180058r2","doi-asserted-by":"publisher","first-page":"51","DOI":"10.1186\/1750-1172-7-51","article-title":"X-linked adrenoleukodystrophy (X-ALD): clinical presentation and guidelines for diagnosis, follow-up and management.","volume":"7","author":"Engelen","year":"2012","journal-title":"Orphanet J Rare Dis"},{"key":"zoi180058r3","first-page":"109","article-title":"The genetic landscape of X-linked adrenoleukodystrophy: inheritance, mutations, modifier genes, and diagnosis.","volume":"8","author":"Wiesinger","year":"2015","journal-title":"Appl Clin Genet"},{"issue":"9-10","key":"zoi180058r4","doi-asserted-by":"publisher","first-page":"219","DOI":"10.1007\/s10354-016-0534-2","article-title":"Therapeutic strategies in adrenoleukodystrophy.","volume":"167","author":"Turk","year":"2017","journal-title":"Wien Med Wochenschr"},{"issue":"3","key":"zoi180058r5","doi-asserted-by":"publisher","first-page":"881","DOI":"10.1182\/blood-2003-10-3402","article-title":"Cerebral X-linked adrenoleukodystrophy: the international hematopoietic cell transplantation experience from 1982 to 1999.","volume":"104","author":"Peters","year":"2004","journal-title":"Blood"},{"issue":"9231","key":"zoi180058r6","doi-asserted-by":"publisher","first-page":"713","DOI":"10.1016\/S0140-6736(00)02629-5","article-title":"Long-term effect of bone-marrow transplantation for childhood-onset cerebral X-linked adrenoleukodystrophy.","volume":"356","author":"Shapiro","year":"2000","journal-title":"Lancet"},{"issue":"7","key":"zoi180058r7","doi-asserted-by":"publisher","first-page":"1971","DOI":"10.1182\/blood-2011-01-329235","article-title":"Outcomes after allogeneic hematopoietic cell transplantation for childhood cerebral adrenoleukodystrophy: the largest single-institution cohort report.","volume":"118","author":"Miller","year":"2011","journal-title":"Blood"},{"issue":"1","key":"zoi180058r8","doi-asserted-by":"publisher","first-page":"6","DOI":"10.1007\/s00431-002-1097-3","article-title":"Haematopoietic stem cell transplantation in 12 patients with cerebral X-linked adrenoleukodystrophy.","volume":"162","author":"Baumann","year":"2003","journal-title":"Eur J Pediatr"},{"issue":"6","key":"zoi180058r9","doi-asserted-by":"publisher","first-page":"665","DOI":"10.1016\/j.bbmt.2007.01.082","article-title":"Outcomes of unrelated umbilical cord blood transplantation for X-linked adrenoleukodystrophy.","volume":"13","author":"Beam","year":"2007","journal-title":"Biol Blood Marrow Transplant"},{"issue":"26","key":"zoi180058r10","doi-asserted-by":"publisher","first-page":"1860","DOI":"10.1056\/NEJM199006283222607","article-title":"Reversal of early neurologic and neuroradiologic manifestations of X-linked adrenoleukodystrophy by bone marrow transplantation.","volume":"322","author":"Aubourg","year":"1990","journal-title":"N Engl J Med"},{"issue":"8","key":"zoi180058r11","doi-asserted-by":"publisher","first-page":"687","DOI":"10.1016\/S1474-4422(07)70177-1","article-title":"Survival analysis of haematopoietic cell transplantation for childhood cerebral X-linked adrenoleukodystrophy: a comparison study.","volume":"6","author":"Mahmood","year":"2007","journal-title":"Lancet Neurol"},{"issue":"2","key":"zoi180058r12","doi-asserted-by":"publisher","first-page":"203","DOI":"10.1111\/petr.2016.20.issue-2","article-title":"Hematopoietic stem cell transplantation for inborn errors of metabolism: a report from the Research Committee on Transplantation for Inborn Errors of Metabolism of the Japanese Ministry of Health, Labour and Welfare and the Working Group of the Japan Society for Hematopoietic Cell Transplantation.","volume":"20","author":"Kato","year":"2016","journal-title":"Pediatr Transplant"},{"issue":"1","key":"zoi180058r13","doi-asserted-by":"publisher","first-page":"49","DOI":"10.1182\/bloodadvances.2017010645","article-title":"Early and late outcomes after cord blood transplantation for pediatric patients with inherited leukodystrophies.","volume":"2","author":"van den Broek","year":"2018","journal-title":"Blood Adv"},{"issue":"3","key":"zoi180058r14","doi-asserted-by":"publisher","first-page":"363","DOI":"10.1007\/s00401-014-1330-y","article-title":"The role of microglia in human disease: therapeutic tool or target?","volume":"128","author":"Cartier","year":"2014","journal-title":"Acta Neuropathol"},{"issue":"5","key":"zoi180058r15","doi-asserted-by":"publisher","first-page":"631","DOI":"10.1001\/archneur.64.5.631","article-title":"New insights about hematopoietic stem cell transplantation in adrenoleukodystrophy.","volume":"64","author":"Moser","year":"2007","journal-title":"Arch Neurol"},{"issue":"5","key":"zoi180058r16","doi-asserted-by":"publisher","first-page":"651","DOI":"10.1001\/archneur.64.5.noc60105","article-title":"Genotype and protein expression after bone marrow transplantation for adrenoleukodystrophy.","volume":"64","author":"Sch\u00f6nberger","year":"2007","journal-title":"Arch Neurol"},{"issue":"37","key":"zoi180058r17","doi-asserted-by":"publisher","first-page":"15018","DOI":"10.1073\/pnas.1205858109","article-title":"Brain conditioning is instrumental for successful microglia reconstitution following hematopoietic stem cell transplantation.","volume":"109","author":"Capotondo","year":"2012","journal-title":"Proc Natl Acad Sci U S A"},{"issue":"5954","key":"zoi180058r18","doi-asserted-by":"publisher","first-page":"818","DOI":"10.1126\/science.1171242","article-title":"Hematopoietic stem cell gene therapy with a lentiviral vector in X-linked adrenoleukodystrophy.","volume":"326","author":"Cartier","year":"2009","journal-title":"Science"},{"issue":"17","key":"zoi180058r19","doi-asserted-by":"publisher","first-page":"1630","DOI":"10.1056\/NEJMoa1700554","article-title":"Hematopoietic stem-cell gene therapy for cerebral adrenoleukodystrophy.","volume":"377","author":"Eichler","year":"2017","journal-title":"N Engl J Med"},{"issue":"1","key":"zoi180058r20","doi-asserted-by":"publisher","first-page":"121","DOI":"10.1038\/gim.2016.68","article-title":"Newborn screening for X-linked adrenoleukodystrophy: evidence summary and advisory committee recommendation.","volume":"19","author":"Kemper","year":"2017","journal-title":"Genet Med"},{"issue":"20","key":"zoi180058r21","doi-asserted-by":"publisher","first-page":"2191","DOI":"10.1001\/jama.2013.281053","article-title":"World Medical Association Declaration of Helsinki: ethical principles for medical research involving human subjects.","volume":"310","author":"World Medical Association","year":"2013","journal-title":"JAMA"},{"issue":"4","key":"zoi180058r22","doi-asserted-by":"publisher","first-page":"229","DOI":"10.1038\/sj.bmt.1703839","article-title":"Hematopoietic cell transplantation for inherited metabolic diseases: an overview of outcomes and practice guidelines.","volume":"31","author":"Peters","year":"2003","journal-title":"Bone Marrow Transplant"},{"issue":"6","key":"zoi180058r23","first-page":"825","article-title":"1994 Consensus conference on acute GVHD grading.","volume":"15","author":"Przepiorka","year":"1995","journal-title":"Bone Marrow Transplant"},{"issue":"2","key":"zoi180058r24","doi-asserted-by":"publisher","first-page":"204","DOI":"10.1016\/0002-9343(80)90380-0","article-title":"Chronic graft-versus-host syndrome in man: a long-term clinicopathologic study of 20 Seattle patients.","volume":"69","author":"Shulman","year":"1980","journal-title":"Am J Med"},{"issue":"9","key":"zoi180058r25","first-page":"1761","article-title":"Adrenoleukodystrophy: a scoring method for brain MR observations.","volume":"15","author":"Loes","year":"1994","journal-title":"AJNR Am J Neuroradiol"},{"issue":"3","key":"zoi180058r26","doi-asserted-by":"publisher","first-page":"369","DOI":"10.1212\/01.WNL.0000079050.91337.83","article-title":"Analysis of MRI patterns aids prediction of progression in X-linked adrenoleukodystrophy.","volume":"61","author":"Loes","year":"2003","journal-title":"Neurology"},{"issue":"4","key":"zoi180058r27","doi-asserted-by":"publisher","first-page":"600","DOI":"10.1016\/j.ejpn.2017.02.006","article-title":"Long-term outcome of patients with X-linked adrenoleukodystrophy: a retrospective cohort study.","volume":"21","author":"Tran","year":"2017","journal-title":"Eur J Paediatr Neurol"},{"issue":"3","key":"zoi180058r28","doi-asserted-by":"publisher","first-page":"338","DOI":"10.1016\/j.bbmt.2012.11.619","article-title":"Outcomes of hematopoietic stem cell transplantation in primary immunodeficiency: a report from the Australian and New Zealand Children\u2019s Haematology Oncology Group and the Australasian Bone Marrow Transplant Recipient Registry.","volume":"19","author":"Mitchell","year":"2013","journal-title":"Biol Blood Marrow Transplant"},{"issue":"4","key":"zoi180058r29","doi-asserted-by":"publisher","first-page":"392","DOI":"10.1038\/s41409-017-0015-2","article-title":"Haploidentical bone marrow transplantation with post transplant cyclophosphamide for patients with X-linked adrenoleukodystrophy: a suitable choice in an urgent situation.","volume":"53","author":"Fernandes","year":"2018","journal-title":"Bone Marrow Transplant"},{"issue":"6","key":"zoi180058r30","doi-asserted-by":"publisher","first-page":"710","DOI":"10.1001\/jamaneurol.2017.0013","article-title":"Neurocognitive trajectory of boys who received a hematopoietic stem cell transplant at an early stage of childhood cerebral adrenoleukodystrophy.","volume":"74","author":"Pierpont","year":"2017","journal-title":"JAMA Neurol"},{"key":"zoi180058r31","doi-asserted-by":"publisher","first-page":"187","DOI":"10.1016\/B978-0-12-386509-0.00010-7","article-title":"Lentiviral hematopoietic cell gene therapy for X-linked adrenoleukodystrophy.","volume":"507","author":"Cartier","year":"2012","journal-title":"Methods Enzymol"}],"container-title":["JAMA Network Open"],"original-title":[],"language":"en","link":[{"URL":"http:\/\/\/jamanetworkopen\/article-pdf\/1\/3\/e180769\/17326180\/khl_2018_oi_180058.pdf","content-type":"unspecified","content-version":"vor","intended-application":"similarity-checking"}],"deposited":{"date-parts":[[2019,2,26]],"date-time":"2019-02-26T15:19:05Z","timestamp":1551194345000},"score":1,"resource":{"primary":{"URL":"http:\/\/jamanetworkopen.jamanetwork.com\/article.aspx?doi=10.1001\/jamanetworkopen.2018.0769"}},"subtitle":[],"short-title":[],"issued":{"date-parts":[[2018,7,20]]},"references-count":31,"journal-issue":{"issue":"3","published-print":{"date-parts":[[2018,7,6]]}},"URL":"https:\/\/doi.org\/10.1001\/jamanetworkopen.2018.0769","relation":{},"ISSN":["2574-3805"],"issn-type":[{"value":"2574-3805","type":"electronic"}],"subject":[],"published":{"date-parts":[[2018,7,20]]}}}