{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,7,27]],"date-time":"2026-07-27T12:08:55Z","timestamp":1785154135440,"version":"3.55.0"},"reference-count":0,"publisher":"Hematology Section, Dept. of Radiological Science and Hematology, Catholic University, Rome, Italy","issue":"1","license":[{"start":{"date-parts":[[2023,6,29]],"date-time":"2023-06-29T00:00:00Z","timestamp":1687996800000},"content-version":"unspecified","delay-in-days":0,"URL":"http:\/\/creativecommons.org\/licenses\/by-nc\/4.0"}],"content-domain":{"domain":[],"crossmark-restriction":false},"short-container-title":["Mediterr J Hematol Infect Dis"],"abstract":"<jats:p>Background: Hematopoietic stem cell transplantation [HSCT] is the only curative option for Fanconi Anemia (FA) patients with hematological abnormalities.\r\nMaterials and Methods: This is a retrospective analysis of patients with FA who underwent a matched-related donor HSCT.\r\nResults: Sixty patients underwent 65 transplants between 1999-2021 using a fludarabine-based low-intensity conditioning regimen. The median age at transplant was 11 years (range: 3-37). Aplastic anemia (AA) was the underlying diagnosis in 55 (84.6%) while 8 (12.4%) had myelodysplastic syndrome (MDS) and 2 (3%) had acute myeloid leukemia (AML). The conditioning regimen used was fludarabine with low-dose cyclophosphamide for aplastic anemia and Fludarabine with low-dose busulfan for MDS\/AML. Graft versus host disease (GVHD)prophylaxis consisted of cyclosporine and methotrexate. Peripheral blood stem cells was the predominant graft source (86.2%). Engraftment occurred in all but 1 patient. The median time to neutrophil and platelet engraftment was 13 days (range: 9-29) &amp; 13 days (range: 5-31), respectively. Day 28 chimerism analysis showed complete chimerism in 75.4 % and mixed chimerism in 18.5%. Secondary graft failure was encountered in 7.7%. Grade II\u2013IV acute GVHD occurred in 29.2% while Grade III-IV acute GVHD occurred in 9.2%. Chronic GVHD was seen in 58.5% and was limited in most patients. The median follow-up is 55 months [range: 2-144] &amp; the 5-year estimated overall survival (OS) is 80.2 \u00b1 5.1%. Secondary malignancies were noted in 4 patients. The 5-year OS was significantly higher in patients undergoing HSCT for AA (86.6 + 4.7%) as compared to MDS\/AML (45.7+16.6%) (p= 0.001).\r\nConclusion: SCT using a fully matched donor provides good outcomes with low-intensity conditioning regimens in patients with FA who have an aplastic marrow.<\/jats:p>","DOI":"10.4084\/mjhid.2023.039","type":"journal-article","created":{"date-parts":[[2023,7,21]],"date-time":"2023-07-21T13:46:29Z","timestamp":1689947189000},"page":"e2023039","source":"Crossref","is-referenced-by-count":6,"title":["FLUDARABINE-BASED LOW-INTENSITY CONDITIONING FOR FANCONI ANEMIA IS ASSOCIATED WITH GOOD OUTCOMES IN APLASTIC ANEMIA BUT NOT IN MDS - A SINGLE-CENTER EXPERIENCE"],"prefix":"10.4084","volume":"15","author":[{"given":"Biju","family":"George","sequence":"first","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Sohini","family":"Chattopadhyay","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Sharon","family":"Lionel","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Sushil","family":"Selvarajan","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Anup","family":"Devasia","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Anu","family":"Korula","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Uday","family":"Kulkarni","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Fouzia","family":"Aboobacker","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Eunice","family":"Sindhuvi","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Kavitha","family":"Lakshmi","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Alok","family":"Srivastava","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Aby","family":"Abraham","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Vikram","family":"Mathews","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]}],"member":"2554","published-online":{"date-parts":[[2023,6,29]]},"container-title":["Mediterranean Journal of Hematology and Infectious Diseases"],"original-title":[],"link":[{"URL":"https:\/\/www.mjhid.org\/index.php\/mjhid\/article\/download\/5297\/4373","content-type":"application\/pdf","content-version":"vor","intended-application":"text-mining"},{"URL":"https:\/\/www.mjhid.org\/index.php\/mjhid\/article\/download\/5297\/4374","content-type":"text\/html","content-version":"vor","intended-application":"text-mining"},{"URL":"https:\/\/www.mjhid.org\/index.php\/mjhid\/article\/download\/5297\/4373","content-type":"unspecified","content-version":"vor","intended-application":"similarity-checking"}],"deposited":{"date-parts":[[2023,7,21]],"date-time":"2023-07-21T13:46:29Z","timestamp":1689947189000},"score":1,"resource":{"primary":{"URL":"https:\/\/www.mjhid.org\/index.php\/mjhid\/article\/view\/5297"}},"subtitle":[],"short-title":[],"issued":{"date-parts":[[2023,6,29]]},"references-count":0,"journal-issue":{"issue":"1","published-online":{"date-parts":[[2023,1,1]]}},"URL":"https:\/\/doi.org\/10.4084\/mjhid.2023.039","relation":{},"ISSN":["2035-3006"],"issn-type":[{"value":"2035-3006","type":"electronic"}],"subject":[],"published":{"date-parts":[[2023,6,29]]}}}