{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,4,22]],"date-time":"2026-04-22T09:46:32Z","timestamp":1776851192488,"version":"3.51.2"},"reference-count":31,"publisher":"American Society of Hematology","issue":"1","content-domain":{"domain":["ashpublications.org"],"crossmark-restriction":true},"short-container-title":[],"published-print":{"date-parts":[[2018,1,9]]},"abstract":"<jats:title>Key Points<\/jats:title>\n               <jats:p>A 6-year overall survival of 61% was observed in leukodystrophy patients after cord blood transplantation. Mismatched cord blood donors, symptomatic disease, and lower PS before cord blood transplantation were predictors of lower survival.<\/jats:p>","DOI":"10.1182\/bloodadvances.2017010645","type":"journal-article","created":{"date-parts":[[2018,1,4]],"date-time":"2018-01-04T20:31:03Z","timestamp":1515097863000},"page":"49-60","update-policy":"https:\/\/doi.org\/10.1182\/blood.2019cm0000","source":"Crossref","is-referenced-by-count":51,"title":["Early and late outcomes after cord blood transplantation for pediatric patients with inherited leukodystrophies"],"prefix":"10.1182","volume":"2","author":[{"ORCID":"https:\/\/orcid.org\/0000-0003-3208-0685","authenticated-orcid":false,"given":"Brigitte T. A.","family":"van den Broek","sequence":"first","affiliation":[{"name":"Blood and Marrow Transplantation Program,"},{"name":"Laboratory for Translational Immunology, and"},{"name":"Sylvia Toth Center for Multidisciplinary Follow Up After Hematopoietic Cell Transplantation, UMC Utrecht, Utrecht, The Netherlands;"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Kristin","family":"Page","sequence":"additional","affiliation":[{"name":"Pediatric Blood and Marrow Transplantation Program, Duke University Medical Center, Durham, NC;"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Annalisa","family":"Paviglianiti","sequence":"additional","affiliation":[{"name":"Eurocord, Paris, France;"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Janna","family":"Hol","sequence":"additional","affiliation":[{"name":"Eurocord, Paris, France;"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Heather","family":"Allewelt","sequence":"additional","affiliation":[{"name":"Pediatric Blood and Marrow Transplantation Program, Duke University Medical Center, Durham, NC;"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Fernanda","family":"Volt","sequence":"additional","affiliation":[{"name":"Eurocord, Paris, France;"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Gerard","family":"Michel","sequence":"additional","affiliation":[{"name":"AP-HM H\u00f4pital de la Timone, Marseille, France;"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Miguel Angel","family":"Diaz","sequence":"additional","affiliation":[{"name":"Hospital Infantil Universitario Ni\u00f1o Jes\u00fas, Madrid, Spain;"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Victoria","family":"Bordon","sequence":"additional","affiliation":[{"name":"Blood and Marrow Transplantation Program, Universiteits Ziekenhuis Gent, Gent, Belgium;"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Tracey","family":"O'Brien","sequence":"additional","affiliation":[{"name":"Sydney Children's Hospital, Sydney, Australia;"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Peter J.","family":"Shaw","sequence":"additional","affiliation":[{"name":"Children's Hospital at Westmead, Sydney, Australia;"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Chantal","family":"Kenzey","sequence":"additional","affiliation":[{"name":"Eurocord, Paris, France;"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Amal","family":"Al-Seraihy","sequence":"additional","affiliation":[{"name":"King Faisal Specialist Hospital and Research Center, Riyadh, Saudi Arabia;"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"ORCID":"https:\/\/orcid.org\/0000-0001-9821-8398","authenticated-orcid":false,"given":"Peter M.","family":"van Hasselt","sequence":"additional","affiliation":[{"name":"Sylvia Toth Center for Multidisciplinary Follow Up After Hematopoietic Cell Transplantation, UMC Utrecht, Utrecht, The Netherlands;"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"ORCID":"https:\/\/orcid.org\/0000-0002-6218-1324","authenticated-orcid":false,"given":"Andrew R.","family":"Gennery","sequence":"additional","affiliation":[{"name":"Institute of Cellular Medicine, Newcastle University, Newcastle upon Tyne, United Kingdom; and"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"ORCID":"https:\/\/orcid.org\/0000-0001-7893-2199","authenticated-orcid":false,"given":"Eliane","family":"Gluckman","sequence":"additional","affiliation":[{"name":"Eurocord, Paris, France;"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Vanderson","family":"Rocha","sequence":"additional","affiliation":[{"name":"Eurocord, Paris, France;"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"ORCID":"https:\/\/orcid.org\/0000-0002-7261-2765","authenticated-orcid":false,"given":"Annalisa","family":"Ruggeri","sequence":"additional","affiliation":[{"name":"Eurocord, Paris, France;"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"ORCID":"https:\/\/orcid.org\/0000-0002-3370-0703","authenticated-orcid":false,"given":"Joanne","family":"Kurtzberg","sequence":"additional","affiliation":[{"name":"Duke University Medical Center, Durham, NC"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"ORCID":"https:\/\/orcid.org\/0000-0003-2232-6952","authenticated-orcid":false,"given":"Jaap Jan","family":"Boelens","sequence":"additional","affiliation":[{"name":"Blood and Marrow Transplantation Program,"},{"name":"Laboratory for Translational Immunology, and"},{"name":"Sylvia Toth Center for Multidisciplinary Follow Up After Hematopoietic Cell Transplantation, UMC Utrecht, Utrecht, The Netherlands;"}],"role":[{"role":"author","vocabulary":"crossref"}]}],"member":"234","published-online":{"date-parts":[[2018,1,4]]},"reference":[{"issue":"5","key":"2019111606431845300_B1","doi-asserted-by":"crossref","first-page":"285","DOI":"10.1055\/s-0036-1584602","article-title":"Neurodevelopmental outcome after hematopoietic cell transplantation in inborn errors of metabolism: current considerations and future perspectives","volume":"47","author":"Boelens","year":"2016","journal-title":"Neuropediatrics"},{"issue":"6","key":"2019111606431845300_B2","doi-asserted-by":"crossref","first-page":"765","DOI":"10.1016\/j.jcyt.2015.03.609","article-title":"Cord blood is the optimal graft source for the treatment of pediatric patients with lysosomal storage diseases: clinical outcomes and future directions","volume":"17","author":"Aldenhoven","year":"2015","journal-title":"Cytotherapy"},{"key":"2019111606431845300_B3","doi-asserted-by":"crossref","first-page":"285","DOI":"10.1182\/asheducation-2011.1.285","article-title":"Stem cell transplantation in inherited metabolic disorders","volume":"2011","author":"Wynn","year":"2011","journal-title":"Hematology Am Soc Hematol Educ Program"},{"issue":"2","key":"2019111606431845300_B4","doi-asserted-by":"crossref","first-page":"184","DOI":"10.1016\/j.bbmt.2005.09.016","article-title":"Results of the cord blood transplantation study (COBLT): outcomes of unrelated donor umbilical cord blood transplantation in pediatric patients with lysosomal and peroxisomal storage diseases","volume":"12","author":"Martin","year":"2006","journal-title":"Biol Blood Marrow Transplant"},{"issue":"7","key":"2019111606431845300_B5","doi-asserted-by":"crossref","first-page":"2979","DOI":"10.1182\/blood-2008-03-140830","article-title":"Unrelated donor umbilical cord blood transplantation for inherited metabolic disorders in 159 pediatric patients from a single center: influence of cellular composition of the graft on transplantation outcomes","volume":"112","author":"Prasad","year":"2008","journal-title":"Blood"},{"issue":"20","key":"2019111606431845300_B6","doi-asserted-by":"crossref","first-page":"2069","DOI":"10.1056\/NEJMoa042604","article-title":"Transplantation of umbilical-cord blood in babies with infantile Krabbe\u2019s disease","volume":"352","author":"Escolar","year":"2005","journal-title":"N Engl J Med"},{"issue":"6","key":"2019111606431845300_B7","doi-asserted-by":"crossref","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":"4","key":"2019111606431845300_B8","doi-asserted-by":"crossref","first-page":"616","DOI":"10.1016\/j.bbmt.2013.01.010","article-title":"Neurodevelopmental outcomes of umbilical cord blood transplantation in metachromatic leukodystrophy","volume":"19","author":"Martin","year":"2013","journal-title":"Biol Blood Marrow Transplant"},{"issue":"9","key":"2019111606431845300_B9","doi-asserted-by":"crossref","first-page":"1133","DOI":"10.1001\/jamaneurol.2016.2067","article-title":"Long-term outcome of allogeneic hematopoietic stem cell transplantation in patients with juvenile metachromatic leukodystrophy compared with nontransplanted control patients","volume":"73","author":"Groeschel","year":"2016","journal-title":"JAMA Neurol"},{"issue":"3","key":"2019111606431845300_B10","doi-asserted-by":"crossref","first-page":"e879","DOI":"10.1542\/peds.2006-0747","article-title":"A staging system for infantile Krabbe disease to predict outcome after unrelated umbilical cord blood transplantation","volume":"118","author":"Escolar","year":"2006","journal-title":"Pediatrics"},{"issue":"19","key":"2019111606431845300_B11","doi-asserted-by":"crossref","first-page":"3981","DOI":"10.1182\/blood-2012-09-455238","article-title":"Outcomes of transplantation using various hematopoietic cell sources in children with Hurler syndrome after myeloablative conditioning","volume":"121","author":"Boelens","year":"2013","journal-title":"Blood"},{"issue":"3","key":"2019111606431845300_B12","doi-asserted-by":"crossref","first-page":"S102","DOI":"10.1016\/j.bbmt.2015.11.405","article-title":"Long-term functional outcomes following hematopoietic stem cell transplantation for Krabbe disease","volume":"22","author":"Allewelt","year":"2016","journal-title":"Biol Blood Marrow Transplant"},{"issue":"9","key":"2019111606431845300_B13","doi-asserted-by":"crossref","first-page":"1362","DOI":"10.1016\/j.bbmt.2011.01.011","article-title":"Total colony-forming units are a strong, independent predictor of neutrophil and platelet engraftment after unrelated umbilical cord blood transplantation: a single-center analysis of 435 cord blood transplants","volume":"17","author":"Page","year":"2011","journal-title":"Biol Blood Marrow Transplant"},{"issue":"4","key":"2019111606431845300_B14","doi-asserted-by":"crossref","first-page":"295","DOI":"10.1097\/00007890-197410000-00001","article-title":"Clinical manifestations of graft-versus-host disease in human recipients of marrow from HL-A-matched sibling donors","volume":"18","author":"Glucksberg","year":"1974","journal-title":"Transplantation"},{"issue":"2","key":"2019111606431845300_B15","doi-asserted-by":"crossref","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":"1","key":"2019111606431845300_B16","doi-asserted-by":"crossref","first-page":"103","DOI":"10.1212\/WNL.0000000000002811","article-title":"Gallbladder and the risk of polyps and carcinoma in metachromatic leukodystrophy","volume":"87","author":"van Rappard","year":"2016","journal-title":"Neurology"},{"key":"2019111606431845300_B17","doi-asserted-by":"crossref","first-page":"187","DOI":"10.1016\/j.jss.2016.08.081","article-title":"Gallbladder abnormalities in children with metachromatic leukodystrophy","volume":"208","author":"Kim","year":"2017","journal-title":"J Surg Res"},{"issue":"7","key":"2019111606431845300_B18","doi-asserted-by":"crossref","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":"24","key":"2019111606431845300_B19","doi-asserted-by":"crossref","first-page":"3098","DOI":"10.1182\/blood-2016-03-708479","article-title":"Efficacy of hematopoietic cell transplantation in metachromatic leukodystrophy: the Dutch experience","volume":"127","author":"van Rappard","year":"2016","journal-title":"Blood"},{"issue":"5","key":"2019111606431845300_B20","doi-asserted-by":"crossref","first-page":"618","DOI":"10.1016\/j.bbmt.2009.01.020","article-title":"Risk factor analysis of outcomes after unrelated cord blood transplantation in patients with hurler syndrome","volume":"15","author":"Boelens","year":"2009","journal-title":"Biol Blood Marrow Transplant"},{"issue":"6","key":"2019111606431845300_B21","doi-asserted-by":"crossref","first-page":"1106","DOI":"10.1016\/j.bbmt.2015.02.011","article-title":"Hematopoietic cell transplantation for mucopolysaccharidosis patients is safe and effective: results after implementation of international guidelines","volume":"21","author":"Aldenhoven","year":"2015","journal-title":"Biol Blood Marrow Transplant"},{"issue":"6","key":"2019111606431845300_B22","doi-asserted-by":"crossref","first-page":"846","DOI":"10.1038\/bmt.2017.5","article-title":"Changes in the incidence, patterns and outcomes of graft failure following hematopoietic stem cell transplantation for Hurler syndrome","volume":"52","author":"Lum","year":"2017","journal-title":"Bone Marrow Transplant"},{"issue":"3","key":"2019111606431845300_B23","doi-asserted-by":"crossref","first-page":"169","DOI":"10.1055\/s-0033-1364179","article-title":"Hematopoietic stem cell transplantation in the leukodystrophies: a systematic review of the literature","volume":"45","author":"Musolino","year":"2014","journal-title":"Neuropediatrics"},{"issue":"1","key":"2019111606431845300_B24","doi-asserted-by":"crossref","first-page":"94","DOI":"10.1186\/s13023-015-0313-y","article-title":"Long-term outcomes after allogeneic hematopoietic stem cell transplantation for metachromatic leukodystrophy: the largest single-institution cohort report","volume":"10","author":"Boucher","year":"2015","journal-title":"Orphanet J Rare Dis"},{"issue":"13","key":"2019111606431845300_B25","doi-asserted-by":"crossref","first-page":"2164","DOI":"10.1182\/blood-2014-11-608075","article-title":"Long-term outcome of Hurler syndrome patients after hematopoietic cell transplantation: an international multicenter study","volume":"125","author":"Aldenhoven","year":"2015","journal-title":"Blood"},{"issue":"1","key":"2019111606431845300_B26","doi-asserted-by":"crossref","first-page":"S87","DOI":"10.1016\/j.ymgme.2016.11.214","article-title":"Long term survival and cardiopulmonary outcome in children with Hurler syndrome after haematopoietic stem cell transplantation in Manchester","volume":"120","author":"Lum","year":"2017","journal-title":"Mol Genet Metab"},{"issue":"2","key":"2019111606431845300_B27","doi-asserted-by":"crossref","first-page":"S32","DOI":"10.1016\/j.ymgme.2008.11.094","article-title":"Health, developmental, and functional outcome surveillance in preschool children with lysosomal storage diseases (LSD)","volume":"96","author":"Msall","year":"2009","journal-title":"Mol Genet Metab"},{"issue":"12","key":"2019111606431845300_B28","doi-asserted-by":"crossref","first-page":"1235","DOI":"10.1038\/gim.2016.35","article-title":"Clinical outcomes of children with abnormal newborn screening results for Krabbe disease in New York State","volume":"18","author":"Wasserstein","year":"2016","journal-title":"Genet Med"},{"issue":"3","key":"2019111606431845300_B29","doi-asserted-by":"crossref","first-page":"369","DOI":"10.1038\/bmt.2012.155","article-title":"Juvenile metachromatic leukodystrophy 10 years post transplant compared with a non-transplanted cohort","volume":"48","author":"Kr\u00e4geloh-Mann","year":"2013","journal-title":"Bone Marrow Transplant"},{"issue":"8","key":"2019111606431845300_B30","doi-asserted-by":"crossref","first-page":"718","DOI":"10.1212\/WNL.0b013e3181eee46b","article-title":"The burden of inherited leukodystrophies in children","volume":"75","author":"Bonkowsky","year":"2010","journal-title":"Neurology"},{"issue":"10043","key":"2019111606431845300_B31","doi-asserted-by":"crossref","first-page":"476","DOI":"10.1016\/S0140-6736(16)30374-9","article-title":"Lentiviral haemopoietic stem-cell gene therapy in early-onset metachromatic leukodystrophy: an ad-hoc analysis of a non-randomised, open-label, phase 1\/2 trial","volume":"388","author":"Sessa","year":"2016","journal-title":"Lancet"}],"container-title":["Blood Advances"],"original-title":[],"language":"en","link":[{"URL":"http:\/\/ashpublications.org\/bloodadvances\/article-pdf\/2\/1\/49\/879739\/advances010645.pdf","content-type":"application\/pdf","content-version":"vor","intended-application":"syndication"},{"URL":"http:\/\/ashpublications.org\/bloodadvances\/article-pdf\/2\/1\/49\/879739\/advances010645.pdf","content-type":"unspecified","content-version":"vor","intended-application":"similarity-checking"}],"deposited":{"date-parts":[[2019,11,16]],"date-time":"2019-11-16T06:43:31Z","timestamp":1573886611000},"score":1,"resource":{"primary":{"URL":"https:\/\/ashpublications.org\/bloodadvances\/article\/2\/1\/49\/15708\/Early-and-late-outcomes-after-cord-blood"}},"subtitle":[],"short-title":[],"issued":{"date-parts":[[2018,1,4]]},"references-count":31,"journal-issue":{"issue":"1","published-print":{"date-parts":[[2018,1,9]]}},"URL":"https:\/\/doi.org\/10.1182\/bloodadvances.2017010645","relation":{},"ISSN":["2473-9529","2473-9537"],"issn-type":[{"value":"2473-9529","type":"print"},{"value":"2473-9537","type":"electronic"}],"subject":[],"published":{"date-parts":[[2018,1,4]]}}}