{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2025,10,31]],"date-time":"2025-10-31T13:52:23Z","timestamp":1761918743879},"reference-count":38,"publisher":"Wiley","issue":"5","license":[{"start":{"date-parts":[[2005,8,2]],"date-time":"2005-08-02T00:00:00Z","timestamp":1122940800000},"content-version":"vor","delay-in-days":0,"URL":"http:\/\/onlinelibrary.wiley.com\/termsAndConditions#vor"}],"content-domain":{"domain":[],"crossmark-restriction":false},"short-container-title":["Pediatric Blood &amp; Cancer"],"published-print":{"date-parts":[[2006,5]]},"abstract":"<jats:title>Abstract<\/jats:title><jats:sec><jats:title>Background<\/jats:title><jats:p>To potentially reduce late effects of malignancy, chronic graft\u2010versus\u2010host disease (GVHD), endocrinopathy, and infertility in patients with Fanconi anemia (FA) undergoing HLA\u2010matched related donor hematopoietic cell transplantation (HCT), we developed a regimen using fludarabine (FLU), cyclophosphamide (CY), and anti\u2010thymocyte globulin (ATG) followed by infusion of T\u2010cell depleted (TCD) bone marrow (BM) or unmanipulated umbilical cord blood (UCB). GVHD prophylaxis consisted of cyclosporine and short course methylprednisolone.<\/jats:p><\/jats:sec><jats:sec><jats:title>Procedure<\/jats:title><jats:p>Between April 2000 and June 2003, 11 patients (10 aplastic anemia (AA), 1 myelodysplastic syndrome (MDS)) underwent HCT using this regimen. Stem cell sources were BM and UCB in eight and three patients, respectively.<\/jats:p><\/jats:sec><jats:sec><jats:title>Results<\/jats:title><jats:p>All patients demonstrated primary engraftment. Median days to neutrophil and platelet engraftment were 11 days (range 9\u201321) and 38 days (range 19\u2013381), respectively. No patient developed GVHD after primary HCT. The patient with MDS relapsed with AML and a maternal donor recipient experienced secondary graft failure. For the nine FA patients with AA who underwent HLA\u2010identical sibling donor HCT, the Kaplan\u2013Meier estimates of overall survival and event\u2010free survival (EFS) at 2 years are 100% and 82%, respectively, at a median follow\u2010up of 2.9 years (range 1.9\u20134.8).<\/jats:p><\/jats:sec><jats:sec><jats:title>Conclusions<\/jats:title><jats:p>In summary, a FLU\u2010based, non\u2010irradiation approach is effective for FA patients with AA undergoing HLA\u2010identical sibling donor HCT. \u00a9 2005 Wiley\u2010Liss, Inc.<\/jats:p><\/jats:sec>","DOI":"10.1002\/pbc.20538","type":"journal-article","created":{"date-parts":[[2005,8,3]],"date-time":"2005-08-03T23:16:50Z","timestamp":1123111010000},"page":"630-636","source":"Crossref","is-referenced-by-count":75,"title":["Successful engraftment without radiation after fludarabine\u2010based regimen in Fanconi anemia patients undergoing genotypically identical donor hematopoietic cell transplantation"],"prefix":"10.1002","volume":"46","author":[{"given":"Poh\u2010Lin","family":"Tan","sequence":"first","affiliation":[],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"John E.","family":"Wagner","sequence":"additional","affiliation":[],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Arleen D.","family":"Auerbach","sequence":"additional","affiliation":[],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Todd E.","family":"DeFor","sequence":"additional","affiliation":[],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Arne","family":"Slungaard","sequence":"additional","affiliation":[],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Margaret L.","family":"MacMillan","sequence":"additional","affiliation":[],"role":[{"role":"author","vocabulary":"crossref"}]}],"member":"311","published-online":{"date-parts":[[2005,8,2]]},"reference":[{"key":"e_1_2_5_2_2","doi-asserted-by":"publisher","DOI":"10.1182\/blood.V73.2.391.391"},{"key":"e_1_2_5_3_2","doi-asserted-by":"publisher","DOI":"10.1182\/blood.V84.5.1650.1650"},{"key":"e_1_2_5_4_2","first-page":"53","article-title":"Allogeneic bone marrow transplantation for Fanconi anemia","volume":"10","author":"Di Bartolomeo P","year":"1992","journal-title":"Bone Marrow Transplant"},{"key":"e_1_2_5_5_2","first-page":"167","article-title":"Marrow transplantation for Fanconi anemia with or without leukemic transformation: An update of the Seattle experience","volume":"9","author":"Flowers ME","year":"1992","journal-title":"Bone Marrow Transplant"},{"key":"e_1_2_5_6_2","doi-asserted-by":"publisher","DOI":"10.1182\/blood.V86.7.2856.2856"},{"key":"e_1_2_5_7_2","first-page":"S24","article-title":"Allogeneic stem cell transplantation for Fanconi anaemia. 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