{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,10,19]],"date-time":"2026-10-19T01:09:14Z","timestamp":1792372154701,"version":"4.4.0"},"reference-count":59,"publisher":"Wiley","issue":"6","license":[{"start":{"date-parts":[[2024,10,27]],"date-time":"2024-10-27T00:00:00Z","timestamp":1729987200000},"content-version":"vor","delay-in-days":0,"URL":"http:\/\/onlinelibrary.wiley.com\/termsAndConditions#vor"},{"start":{"date-parts":[[2024,10,27]],"date-time":"2024-10-27T00:00:00Z","timestamp":1729987200000},"content-version":"tdm","delay-in-days":0,"URL":"http:\/\/doi.wiley.com\/10.1002\/tdm_license_1.1"}],"content-domain":{"domain":["onlinelibrary.wiley.com"],"crossmark-restriction":true},"short-container-title":["Br J Haematol"],"published-print":{"date-parts":[[2024,12]]},"abstract":"<jats:title>Summary<\/jats:title>\n                  <jats:p>Aplastic anaemia in infants and young children presents unique challenges due to high prevalence of inherited bone marrow failure syndromes (IBMFS) in this age group. The objective of this study is assessing clinical characteristics and outcomes of haematopoietic cell transplantation in children \u22645\u2009years with bone marrow failure syndromes. We analysied 106 patients (66% males), median age 4.6\u2009years, including 40 with Fanconi anaemia (FA), 32 with Acquired Severe Aplastic anaemia (aSAA), 15 with Diamond\u2013Blackfan Anaemia, 11 with Amegakaryocytic Purpura and 8 with other IBMFS. Molecular testing was limited (39%), with 25.4% confirmed genetically. Retrospective longitudinal study across three paediatric transplantation centres (1982\u20132020). Overall survival (OS) was 76.4% over a median 10\u2010year follow\u2010up. OS rates were similar between aSAA and IBMFS (FA 77.5%, other IBMFS 76.5%). Transplant\u2010related mortality (TRM) was lower in aSAA (9.4%) compared with IBMFS (16.2%). Recent years showed improved outcomes, with TRM declining post\u20102010. Choice of stem cell source impacted OS, favouring bone marrow over umbilical cord, but showing encouraging results with haploidentical. Late complications were common, including endocrine\u2010metabolic issues and delayed neuropsychomotor development. Diagnosing and managing bone marrow failures in young children pose significant challenges. Despite advancements in transplant practices, ongoing vigilance and comprehensive care are necessary to improve long\u2010term survival rates.<\/jats:p>","DOI":"10.1111\/bjh.19831","type":"journal-article","created":{"date-parts":[[2024,10,28]],"date-time":"2024-10-28T00:06:28Z","timestamp":1730073988000},"page":"2387-2402","update-policy":"https:\/\/doi.org\/10.1002\/crossmark_policy","source":"Crossref","is-referenced-by-count":4,"title":["Haematopoietic cell transplantation for 106 infants and preschoolers with acquired and inherited bone marrow failures"],"prefix":"10.1111","volume":"205","author":[{"ORCID":"https:\/\/orcid.org\/0000-0003-2378-5026","authenticated-orcid":false,"given":"Gisele","family":"Loth","sequence":"first","affiliation":[{"name":"Faculdades Pequeno Pr\u00edncipe  Curitiba Paran\u00e1 Brazil"},{"name":"Bone Marrow Transplantation Unit Hospital de Cl\u00ednicas, Universidade Federal do Paran\u00e1  Curitiba Paran\u00e1 Brazil"},{"name":"Bone Marrow Transplantation Unit Hospital Pequeno Pr\u00edncipe  Curitiba Paran\u00e1 Brazil"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Cilmara C. 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