title: Long-term follow-up of allogeneic stem cell transplantation in patients with severe aplastic anemia after conditioning with cyclophosphamide plus antithymocyte globulin.
abstract: We investigated the efficacy of an antithymocyte globulin/cyclophosphamide preparative regimen prior to allogeneic stem cell transplantation from HLA-identical siblings in patients with severe aplastic anemia. Since 1990, 21 patients, 6 males and 15 females, with a median age of 25 years (range: 7-43) have been enrolled in the protocol consisting of 200 mg/kg cyclophosphamide and 90-120 mg/kg antithymocyte globulin (ATG, rabbit, Fresenius, Bad Homburg, Germany). For further graft-versus-host disease (GVHD) prophylaxis all patients received cyclosporin A and a short course of methotrexate (MTX). Only one patient had a primary graft failure (5%). All other patients engrafted with a leukocyte count >1.0 x 10(9)/l and a platelet count >20 x 10(9)/l after a median of 19 (range: 11-28) and 26 days (range: 13-67), respectively. No late graft failure or relapse was observed. Two patients experienced mild acute GVHD grade I (10%), and one patient developed grade II GVHD (5%). No severe grade III/IV GVHD was observed; 17% of the patients developed limited chronic GVHD. The treatment-related mortality was 14% and mainly due to fungal infection. After a median follow-up of 70 months (range: 2-139), the estimated overall and event-free survival at 10 years for all patients is 86% (95% confidence interval: 70-100%). We conclude that ATG plus cyclophosphamide is an effective conditioning regimen in patients with aplastic anemia undergoing stem cell transplantation with a low treatment-related mortality, resulting in an excellent outcome.
authors.affiliation: Department of Bone Marrow Transplantation, University Hospital Hamburg-Eppendorf, Martinistrasse 52, 20246 Hamburg, Germany. nkroeger@uke.uni-hamburg.de
authors.forename: N
authors.initials: N
authors.lastname: Kröger
authors.forename: T
authors.initials: T
authors.lastname: Zabelina
authors.forename: H
authors.initials: H
authors.lastname: Renges
authors.forename: W
authors.initials: W
authors.lastname: Krüger
authors.forename: U
authors.initials: U
authors.lastname: Kordes
authors.forename: J
authors.initials: J
authors.lastname: Rischewski
authors.forename: J
authors.initials: J
authors.lastname: Schrum
authors.forename: M
authors.initials: M
authors.lastname: Horstmann
authors.forename: F
authors.initials: F
authors.lastname: Ayuk
authors.forename: R
authors.initials: R
authors.lastname: Erttmann
authors.forename: H
authors.initials: H
authors.lastname: Kabisch
authors.forename: A R
authors.initials: AR
authors.lastname: Zander
mesh_terms: D000293:Adolescent; D000328:Adult; D000741:Anemia, Aplastic / Q000150:complications / Q000401:mortality / Q000628:therapy*; D000961:Antilymphocyte Serum / Q000008:administration & dosage; D000971:Antineoplastic Combined Chemotherapy Protocols / Q000008:administration & dosage* / Q000633:toxicity; D016026:Bone Marrow Transplantation / Q000379:methods / Q000401:mortality; D002648:Child; D003520:Cyclophosphamide / Q000008:administration & dosage; D016572:Cyclosporine / Q000008:administration & dosage; D005260:Female; D006086:Graft vs Host Disease / Q000188:drug therapy / Q000473:pathology / Q000517:prevention & control; D018380:Hematopoietic Stem Cell Transplantation / Q000379:methods* / Q000401:mortality; D006801:Humans; D008137:Longitudinal Studies; D008297:Male; D008727:Methotrexate / Q000008:administration & dosage; D009181:Mycoses / Q000139:chemically induced; D016019:Survival Analysis; D019172:Transplantation Conditioning / Q000379:methods* / Q000401:mortality; D014184:Transplantation, Homologous / Q000379:methods / Q000401:mortality
chemical_list: D000961:Antilymphocyte Serum; D016572:Cyclosporine; D003520:Cyclophosphamide; D008727:Methotrexate