{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,9,10]],"date-time":"2026-09-10T04:06:09Z","timestamp":1789013169742,"version":"build-2803163510"},"reference-count":50,"publisher":"Wiley","issue":"1","license":[{"start":{"date-parts":[[2022,11,23]],"date-time":"2022-11-23T00:00:00Z","timestamp":1669161600000},"content-version":"vor","delay-in-days":0,"URL":"http:\/\/creativecommons.org\/licenses\/by-nc-nd\/4.0\/"}],"content-domain":{"domain":["onlinelibrary.wiley.com"],"crossmark-restriction":true},"short-container-title":["Clinical Transplantation"],"published-print":{"date-parts":[[2023,1]]},"abstract":"<jats:title>Abstract<\/jats:title><jats:p>Allogeneic hematopoietic stem cell transplantation (HSCT) is currently the only curative treatment for the hematological complications of patients with Fanconi anemia (FA). Over the last two decades, HSCT outcomes have improved dramatically following the development of regimens tailored for FA patients. In this study, we analyzed genetic, clinical, and transplant data of 41 patients with FA who underwent HSCT at Hadassah Medical Center between November 1996 and September 2020. Overall survival (OS) was 82.9% with a median follow\u2010up time of 2.11\u2010years (95% CI, .48\u201316.56). Thirteen patients (31.7%) developed acute graft\u2010versus\u2010host disease (GVHD), three of them with grades 3\u20104. Nine patients developed chronic GVHD, five had extensive disease. Twelve patients (29.3%) developed stable mixed\u2010chimerism with complete resolution of bone marrow failure (BMF); none of them had acute nor chronic GVHD. Significantly higher GVHD rates were observed in transplants from peripheral blood stem cell grafts as compared to other stem cell sources (<jats:italic>p<\/jats:italic>\u00a0=\u00a0.002 for acute and <jats:italic>p<\/jats:italic>\u00a0=\u00a0.004 for chronic GVHD). Outcome parameters were comparable between HSCT from matched\u2010sibling (<jats:italic>n<\/jats:italic>\u00a0=\u00a020) to other donors (<jats:italic>n<\/jats:italic>\u00a0=\u00a021), including survival rates (<jats:italic>p<\/jats:italic>\u00a0=\u00a0.1), time to engraftment (<jats:italic>p<\/jats:italic>\u00a0=\u00a0.69 and <jats:italic>p<\/jats:italic>\u00a0=\u00a0.14 for neutrophil and platelet engraftment time, respectively), chimerism status (<jats:italic>p<\/jats:italic>\u00a0=\u00a0.36 and <jats:italic>p<\/jats:italic>\u00a0=\u00a0.83 for full\u2010donor and mixed chimerism, respectively), and GVHD prevalence (<jats:italic>p<\/jats:italic>\u00a0=\u00a01). Our results demonstrate the vast improvements in HSCT outcomes of patients with FA, narrowing the gap between matched\u2010sibling versus alternative donor transplantations. Our data identifies factors that may significantly affect transplant outcomes such as graft source and chimerism status.<\/jats:p>","DOI":"10.1111\/ctr.14835","type":"journal-article","created":{"date-parts":[[2022,10,19]],"date-time":"2022-10-19T07:11:28Z","timestamp":1666163488000},"update-policy":"https:\/\/doi.org\/10.1002\/crossmark_policy","source":"Crossref","is-referenced-by-count":11,"title":["Two decades of stem cell transplantation in patients with Fanconi anemia: Analysis of factors affecting transplant outcomes"],"prefix":"10.1111","volume":"37","author":[{"given":"Orly","family":"Fink","sequence":"first","affiliation":[{"name":"Faculty of Medicine Hebrew University of Jerusalem  Jerusalem Israel"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Ehud","family":"Even\u2010Or","sequence":"additional","affiliation":[{"name":"Faculty of Medicine Hebrew University of Jerusalem  Jerusalem Israel"},{"name":"Department of Bone Marrow Transplantation and Cancer Immunotherapy Hadassah Medical Center  Jerusalem Israel"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Batia","family":"Avni","sequence":"additional","affiliation":[{"name":"Faculty of Medicine Hebrew University of Jerusalem  Jerusalem Israel"},{"name":"Department of Bone Marrow Transplantation and Cancer Immunotherapy Hadassah Medical Center  Jerusalem Israel"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Sigal","family":"Grisariu","sequence":"additional","affiliation":[{"name":"Faculty of Medicine Hebrew University of Jerusalem  Jerusalem Israel"},{"name":"Department of Bone Marrow Transplantation and Cancer Immunotherapy Hadassah Medical Center  Jerusalem Israel"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Irina","family":"Zaidman","sequence":"additional","affiliation":[{"name":"Faculty of Medicine Hebrew University of Jerusalem  Jerusalem Israel"},{"name":"Department of Bone Marrow Transplantation and Cancer Immunotherapy Hadassah Medical Center  Jerusalem Israel"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Yael Dinur","family":"Schejter","sequence":"additional","affiliation":[{"name":"Faculty of Medicine Hebrew University of Jerusalem  Jerusalem Israel"},{"name":"Department of Bone Marrow Transplantation and Cancer Immunotherapy Hadassah Medical Center  Jerusalem Israel"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Adeeb","family":"NaserEddin","sequence":"additional","affiliation":[{"name":"Faculty of Medicine Hebrew University of Jerusalem  Jerusalem Israel"},{"name":"Department of Bone Marrow Transplantation and Cancer Immunotherapy Hadassah Medical Center  Jerusalem Israel"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Mohammad","family":"Najajreh","sequence":"additional","affiliation":[{"name":"The Huda Al Masri Pediatric Cancer Department Beit Jala Hospital  Beit Jala Palestine"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Polina","family":"Stepensky","sequence":"additional","affiliation":[{"name":"Faculty of Medicine Hebrew University of Jerusalem  Jerusalem Israel"},{"name":"Department of Bone Marrow Transplantation and Cancer Immunotherapy Hadassah Medical Center  Jerusalem Israel"}],"role":[{"vocabulary":"crossref","role":"author"}]}],"member":"311","published-online":{"date-parts":[[2022,11,23]]},"reference":[{"key":"e_1_2_9_2_1","volume-title":"Jahrbuch f\u00fcr Kinderheilkunde und physische Erziehung","author":"Fanconi","year":"1927"},{"key":"e_1_2_9_3_1","doi-asserted-by":"publisher","DOI":"10.1182\/blood\u20102004\u201007\u20102588"},{"key":"e_1_2_9_4_1","doi-asserted-by":"publisher","DOI":"10.1182\/blood\u20102004\u201010\u20103968"},{"key":"e_1_2_9_5_1","doi-asserted-by":"publisher","DOI":"10.1007\/s10689-004-9565-8"},{"key":"e_1_2_9_6_1","doi-asserted-by":"crossref","unstructured":"RosenbergPS TamaryH AlterBP.How high are carrier frequencies of rare recessive syndromes? 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