{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2025,2,21]],"date-time":"2025-02-21T08:24:03Z","timestamp":1740126243833,"version":"3.37.3"},"reference-count":17,"publisher":"Wiley","issue":"10","license":[{"start":{"date-parts":[[2017,7,28]],"date-time":"2017-07-28T00:00:00Z","timestamp":1501200000000},"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":["J Paediatrics Child Health"],"published-print":{"date-parts":[[2017,10]]},"abstract":"<jats:sec><jats:title>Aim<\/jats:title><jats:p>Haematopoietic stem cell transplantation (<jats:styled-content style=\"fixed-case\">HSCT<\/jats:styled-content>) is a central therapy in the treatment of primary immunodeficiency diseases (<jats:styled-content style=\"fixed-case\">PIDs<\/jats:styled-content>). Over the past 5 years, outcomes have been greatly improved due to earlier diagnosis, improved donor availability, advancements in graft manipulation and the use of less toxic preparative regimens. We present a 5\u2010year audit of <jats:styled-content style=\"fixed-case\">HSCT<\/jats:styled-content> for <jats:styled-content style=\"fixed-case\">PID<\/jats:styled-content> at a single Australian tertiary hospital.<\/jats:p><\/jats:sec><jats:sec><jats:title>Methods<\/jats:title><jats:p>Retrospective case note review identified diagnosis, pre\u2010transplant medical morbidity, transplant protocol, engraftment, adverse events, post\u2010transplant immune reconstitution and general health.<\/jats:p><\/jats:sec><jats:sec><jats:title>Results<\/jats:title><jats:p>A total of 22 patients with <jats:styled-content style=\"fixed-case\">PID<\/jats:styled-content> underwent 24 <jats:styled-content style=\"fixed-case\">HSCTs<\/jats:styled-content> at our institution between 2012 and 2016. The most common indications were severe combined immunodeficiency, chronic granulomatous disease and familial haemophagocytic lymphohistiocytosis, with a genetic diagnosis in all but two patients. Reduced intensity or reduced toxicity conditioning was used in 91% of cases, and 75% of the donors were unrelated. Transplant\u2010related mortality at day +100 was 9.5%, and cumulative overall survival was 86%. There were three mortalities, all secondary to viral infection, one of which occurred in the context of graft failure. Two patients remained on immune support, with the remainder achieving adequate immune reconstitution.<\/jats:p><\/jats:sec><jats:sec><jats:title>Conclusions<\/jats:title><jats:p>The outcomes for <jats:styled-content style=\"fixed-case\">HSCT<\/jats:styled-content> for <jats:styled-content style=\"fixed-case\">PIDs<\/jats:styled-content> performed at Sydney Children's Hospital were in line with the world's best practice. <jats:styled-content style=\"fixed-case\">HSCT<\/jats:styled-content> should be considered a potential therapeutic option for all Australian <jats:styled-content style=\"fixed-case\">PID<\/jats:styled-content> patients with a valid disease indication.<\/jats:p><\/jats:sec>","DOI":"10.1111\/jpc.13643","type":"journal-article","created":{"date-parts":[[2017,7,28]],"date-time":"2017-07-28T08:43:30Z","timestamp":1501231410000},"page":"988-994","update-policy":"https:\/\/doi.org\/10.1002\/crossmark_policy","source":"Crossref","is-referenced-by-count":6,"title":["Haematopoietic stem cell transplantation for primary immunodeficiency syndromes: A 5\u2010year single\u2010centre experience"],"prefix":"10.1111","volume":"53","author":[{"ORCID":"https:\/\/orcid.org\/0000-0002-9648-9861","authenticated-orcid":false,"given":"Melissa","family":"Norman","sequence":"first","affiliation":[{"name":"Department of Immunology Sydney Children's Hospital  Sydney New South Wales Australia"},{"name":"School of Women's and Children's Health University of New South Wales  Sydney New South Wales 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