{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,3,26]],"date-time":"2026-03-26T04:05:07Z","timestamp":1774497907062,"version":"3.50.1"},"reference-count":43,"publisher":"Wiley","issue":"3","license":[{"start":{"date-parts":[[2006,3,24]],"date-time":"2006-03-24T00:00:00Z","timestamp":1143158400000},"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":["Br J Haematol"],"published-print":{"date-parts":[[2006,5]]},"abstract":"<jats:title>Summary<\/jats:title><jats:p>Allogeneic haematopoietic cell transplantation (HCT) can cure a variety of non\u2010malignant haematological disorders. Although transplant outcomes for selected patients with severe aplastic anaemia (SAA) and paroxysmal nocturnal haemoglobinuria (PNH) have improved, older age, allo\u2010immunisation from transfusions, prior immunosuppressive therapy and a prolonged time from diagnosis to transplantation are associated with worse outcome. Because of its potent immunosuppressive effects, we investigated a fludarabine\u2010based non\u2010myeloablative conditioning regimen in patients with transfusion\u2010dependent non\u2010malignant haematological disorders at increased risk for graft rejection with conventional transplant conditioning. Twenty\u2010six patients with transfusion dependent\/anti\u2010thymocyte globulin (ATG)\u2010refractory SAA, PNH or pure red cell aplasia underwent HCT from a human leucocyte antigen (HLA)\u2010compatible relative. Transplant conditioning consisted of cyclophosphamide (120\u2003mg\/kg) and fludarabine (125\u2003mg\/m<jats:sup>2<\/jats:sup>) with or without ATG. Ciclosporine, alone or combined with mycophenolate mofetil or methotrexate, was used as graft\u2010<jats:italic>versus<\/jats:italic>\u2010host disease (GVHD) prophylaxis. All patients achieved durable engraftment and transfusion\u2010independence. Twenty\u2010four of 26 patients are alive at a median of 21\u2003months following transplantation. Although a high cumulative incidence of acute (65% grades II\u2013IV, 54% grades III\u2013IV) and chronic GVHD (56%) was observed, only one patient died from transplant\u2010related causes (cumulative incidence 7%). These data show that HCT following fludarabine\u2010based non\u2010myeloablative conditioning results in durable engraftment and excellent survival in SAA and PNH patients at high risk for graft rejection.<\/jats:p>","DOI":"10.1111\/j.1365-2141.2006.06019.x","type":"journal-article","created":{"date-parts":[[2006,3,24]],"date-time":"2006-03-24T14:09:24Z","timestamp":1143209364000},"page":"305-314","update-policy":"https:\/\/doi.org\/10.1002\/crossmark_policy","source":"Crossref","is-referenced-by-count":94,"title":["Overcoming graft rejection in heavily transfused and allo\u2010immunised patients with bone marrow failure syndromes using fludarabine\u2010based haematopoietic cell transplantation"],"prefix":"10.1111","volume":"133","author":[{"given":"Ramaprasad","family":"Srinivasan","sequence":"first","affiliation":[],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Yoshiyuki","family":"Takahashi","sequence":"additional","affiliation":[],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"J.","family":"Philip McCoy","sequence":"additional","affiliation":[],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Igor","family":"Espinoza\u2010Delgado","sequence":"additional","affiliation":[],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Colleen","family":"Dorrance","sequence":"additional","affiliation":[],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Takehito","family":"Igarashi","sequence":"additional","affiliation":[],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Andreas","family":"Lundqvist","sequence":"additional","affiliation":[],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"A.","family":"John Barrett","sequence":"additional","affiliation":[],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Neal S","family":"Young","sequence":"additional","affiliation":[],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Nancy","family":"Geller","sequence":"additional","affiliation":[],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Richard W.","family":"Childs","sequence":"additional","affiliation":[],"role":[{"role":"author","vocabulary":"crossref"}]}],"member":"311","published-online":{"date-parts":[[2006,3,31]]},"reference":[{"key":"e_1_2_6_2_1","doi-asserted-by":"publisher","DOI":"10.1182\/blood-2003-07-2546"},{"key":"e_1_2_6_3_1","doi-asserted-by":"crossref","first-page":"69","DOI":"10.1016\/S0037-1963(00)90031-3","article-title":"Treatment of acquired severe aplastic anemia: bone marrow transplantation compared with immunosuppressive therapy \u2013 The European Group for Blood and Marrow Transplantation experience","volume":"37","author":"Bacigalupo A.","year":"2000","journal-title":"Seminars in Hematology"},{"key":"e_1_2_6_4_1","doi-asserted-by":"publisher","DOI":"10.1111\/j.1365-2141.1999.01374.x"},{"key":"e_1_2_6_5_1","first-page":"59","article-title":"[Results and current indications of bone marrow allograft in sickle cell disease]","volume":"47","author":"Bernaudin F.","year":"1999","journal-title":"Pathologie-Biologie"},{"key":"e_1_2_6_6_1","doi-asserted-by":"publisher","DOI":"10.1182\/blood.V98.6.1687"},{"key":"e_1_2_6_7_1","doi-asserted-by":"publisher","DOI":"10.1182\/blood-2003-04-1170"},{"key":"e_1_2_6_8_1","first-page":"94","article-title":"Nonmyeloablative preparative regimens for allogeneic hematopoietic transplantation. 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