{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,8,3]],"date-time":"2026-08-03T17:17:52Z","timestamp":1785777472045,"version":"3.56.0"},"reference-count":23,"publisher":"Wiley","issue":"6","license":[{"start":{"date-parts":[[2013,6,27]],"date-time":"2013-06-27T00:00:00Z","timestamp":1372291200000},"content-version":"vor","delay-in-days":0,"URL":"http:\/\/onlinelibrary.wiley.com\/termsAndConditions#vor"}],"content-domain":{"domain":[],"crossmark-restriction":false},"short-container-title":["Pediatric Transplantation"],"published-print":{"date-parts":[[2013,9]]},"abstract":"<jats:title>Abstract<\/jats:title><jats:p>We report a retrospective analysis of 53 haematopoietic stem cell transplants for inherited metabolic disorders performed at <jats:styled-content style=\"fixed-case\">ANZCHOG<\/jats:styled-content> transplant centres between 1992 and 2008. Indications for transplant included Hurler syndrome, ALD, and MLD. The majority of transplants utilized unrelated donor stem cells (66%) with 65% of those being unrelated cord blood. Conditioning therapy was largely myeloablative, with Bu plus another cytotoxic agent used in 89% of recipients. Primary graft failure was rare, occurring in three patients, all of whom remain long\u2010term survivors following the second transplant. The CI of grade <jats:styled-content style=\"fixed-case\">II<\/jats:styled-content>\u2010<jats:styled-content style=\"fixed-case\">IV<\/jats:styled-content> and grade <jats:styled-content style=\"fixed-case\">III<\/jats:styled-content>\u2010<jats:styled-content style=\"fixed-case\">IV<\/jats:styled-content> acute GVHD at day +100 was 39% and 14%, respectively. Chronic <jats:styled-content style=\"fixed-case\">GVHD<\/jats:styled-content> occurred in 17% of recipients. TRM was 12% at day +100 and 19% at one\u00a0yr post\u2010transplant. OS at five\u00a0yr was 78% for the cohort, 73% for patients with ALD and 83% for patients with Hurler syndrome. There was no statistically significant difference in overall survival between unrelated marrow and unrelated cord blood donor groups. The development of interstitial pneumonitis was an independent variable shown to significantly impact on <jats:styled-content style=\"fixed-case\">TRM<\/jats:styled-content> and <jats:styled-content style=\"fixed-case\">OS<\/jats:styled-content>. In summary, we report a large cohort of patients with inherited metabolic disorders with excellent survival post\u2010allogeneic transplant.<\/jats:p>","DOI":"10.1111\/petr.12109","type":"journal-article","created":{"date-parts":[[2013,6,27]],"date-time":"2013-06-27T07:36:58Z","timestamp":1372318618000},"page":"582-588","source":"Crossref","is-referenced-by-count":29,"title":["Outcomes of haematopoietic stem cell transplantation for inherited metabolic disorders: A report from the <scp>A<\/scp>ustralian and <scp>N<\/scp>ew <scp>Z<\/scp>ealand <scp>C<\/scp>hildren's <scp>H<\/scp>aematology <scp>O<\/scp>ncology <scp>G<\/scp>roup and the <scp>A<\/scp>ustralasian <scp>B<\/scp>one <scp>M<\/scp>arrow <scp>T<\/scp>ransplant <scp>R<\/scp>ecipient <scp>R<\/scp>egistry"],"prefix":"10.1111","volume":"17","author":[{"given":"R.","family":"Mitchell","sequence":"first","affiliation":[{"name":"Centre for Children's Cancer and Blood Disorders Sydney Children's Hospital Randwick NSW Australia"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"I.","family":"Nivison\u2010Smith","sequence":"additional","affiliation":[{"name":"Australasian Bone Marrow Transplant Recipient Registry Darlinghurst NSW Australia"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"A.","family":"Anazodo","sequence":"additional","affiliation":[{"name":"Centre for Children's Cancer and Blood Disorders Sydney Children's Hospital Randwick NSW Australia"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"K.","family":"Tiedemann","sequence":"additional","affiliation":[{"name":"Children's Cancer Centre Royal Children's Hospital Melbourne Vic. Australia"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"P. J.","family":"Shaw","sequence":"additional","affiliation":[{"name":"Oncology Unit Children's Hospital Westmead Westmead NSW Australia"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"L.","family":"Teague","sequence":"additional","affiliation":[{"name":"Haematology\/Oncology Service Starship Children's Hospital Auckland New Zealand"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"C. J.","family":"Fraser","sequence":"additional","affiliation":[{"name":"Queensland Children's Cancer Centre Royal Children's Hospital Herston Qld Australia"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"T. L.","family":"Carter","sequence":"additional","affiliation":[{"name":"Department of Paediatric Haematology and Oncology Princess Margaret Hospital for Children Perth WA Australia"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"H.","family":"Tapp","sequence":"additional","affiliation":[{"name":"Haematology\/Oncology Unit Women's and Children's Hospital Adelaide SA Australia"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"F.","family":"Alvaro","sequence":"additional","affiliation":[{"name":"Children's Cancer and Haematology Service John Hunter Children's Hospital Newcastle NSW Australia"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"T. A.","family":"O'Brien","sequence":"additional","affiliation":[{"name":"Centre for Children's Cancer and Blood Disorders Sydney Children's Hospital Randwick NSW Australia"}],"role":[{"vocabulary":"crossref","role":"author"}]}],"member":"311","published-online":{"date-parts":[[2013,6,27]]},"reference":[{"key":"e_1_2_9_2_1","doi-asserted-by":"publisher","DOI":"10.1182\/blood-2003-10-3402"},{"key":"e_1_2_9_3_1","doi-asserted-by":"publisher","DOI":"10.1182\/blood.V91.7.2601"},{"key":"e_1_2_9_4_1","doi-asserted-by":"publisher","DOI":"10.1001\/archneur.55.1.98"},{"key":"e_1_2_9_5_1","doi-asserted-by":"publisher","DOI":"10.1016\/S0140-6736(81)91046-1"},{"key":"e_1_2_9_6_1","doi-asserted-by":"publisher","DOI":"10.1126\/science.162.3853.570"},{"key":"e_1_2_9_7_1","doi-asserted-by":"publisher","DOI":"10.1007\/s00431-011-1401-1"},{"key":"e_1_2_9_8_1","doi-asserted-by":"publisher","DOI":"10.1056\/NEJMoa042604"},{"key":"e_1_2_9_9_1","doi-asserted-by":"publisher","DOI":"10.1182\/blood-2008-03-140830"},{"key":"e_1_2_9_10_1","doi-asserted-by":"publisher","DOI":"10.1016\/j.bbmt.2005.09.016"},{"key":"e_1_2_9_11_1","doi-asserted-by":"publisher","DOI":"10.1182\/blood-2011-01-329235"},{"key":"e_1_2_9_12_1","doi-asserted-by":"publisher","DOI":"10.1038\/sj.bmt.1705718"},{"key":"e_1_2_9_13_1","doi-asserted-by":"publisher","DOI":"10.1038\/bmt.2009.319"},{"key":"e_1_2_9_14_1","doi-asserted-by":"publisher","DOI":"10.1016\/j.bbmt.2007.04.003"},{"key":"e_1_2_9_15_1","doi-asserted-by":"publisher","DOI":"10.1097\/00007890-197410000-00001"},{"key":"e_1_2_9_16_1","doi-asserted-by":"publisher","DOI":"10.1016\/0002-9343(80)90380-0"},{"key":"e_1_2_9_17_1","doi-asserted-by":"publisher","DOI":"10.1038\/sj.bmt.1704560"},{"key":"e_1_2_9_18_1","doi-asserted-by":"publisher","DOI":"10.1016\/j.bbmt.2007.01.082"},{"key":"e_1_2_9_19_1","doi-asserted-by":"publisher","DOI":"10.1016\/j.bbmt.2009.01.020"},{"key":"e_1_2_9_20_1","doi-asserted-by":"publisher","DOI":"10.1517\/14712598.8.7.1003"},{"key":"e_1_2_9_21_1","doi-asserted-by":"publisher","DOI":"10.1038\/sj.bmt.1705401"},{"key":"e_1_2_9_22_1","doi-asserted-by":"publisher","DOI":"10.1038\/sj.bmt.1705934"},{"key":"e_1_2_9_23_1","doi-asserted-by":"publisher","DOI":"10.1182\/blood-2010-04-278234"},{"key":"e_1_2_9_24_1","doi-asserted-by":"publisher","DOI":"10.1126\/science.1171242"}],"container-title":["Pediatric Transplantation"],"original-title":[],"language":"en","link":[{"URL":"https:\/\/api.wiley.com\/onlinelibrary\/tdm\/v1\/articles\/10.1111%2Fpetr.12109","content-type":"unspecified","content-version":"vor","intended-application":"text-mining"},{"URL":"https:\/\/onlinelibrary.wiley.com\/doi\/pdf\/10.1111\/petr.12109","content-type":"unspecified","content-version":"vor","intended-application":"similarity-checking"}],"deposited":{"date-parts":[[2023,9,29]],"date-time":"2023-09-29T15:34:49Z","timestamp":1696001689000},"score":1,"resource":{"primary":{"URL":"https:\/\/onlinelibrary.wiley.com\/doi\/10.1111\/petr.12109"}},"subtitle":[],"short-title":[],"issued":{"date-parts":[[2013,6,27]]},"references-count":23,"journal-issue":{"issue":"6","published-print":{"date-parts":[[2013,9]]}},"alternative-id":["10.1111\/petr.12109"],"URL":"https:\/\/doi.org\/10.1111\/petr.12109","archive":["Portico"],"relation":{},"ISSN":["1397-3142","1399-3046"],"issn-type":[{"value":"1397-3142","type":"print"},{"value":"1399-3046","type":"electronic"}],"subject":[],"published":{"date-parts":[[2013,6,27]]}}}