title: Cyclophosphamide and antithymocyte globulin to condition patients with aplastic anemia for allogeneic marrow transplantations: the experience in four centers.
abstract: This report summarizes the experience with a conditioning regimen of cyclophosphamide and antithymocyte globulin in patients with severe aplastic anemia given HLA-matched related marrow grafts at 4 transplantation centers. Enrolled were 94 consecutive patients, of whom 87 had received multiple transfusions and 38 had failed immunosuppressive therapy. Their ages ranged from 2 to 59 years. After transplantation, 89 patients received a methotrexate/cyclosporine regimen for graft-versus-host disease (GVHD) prevention. Cyclosporine with or without prednisone was given in 4 patients, and no immunosuppression was given in 1 patient. Ninety-six percent of patients had sustained grafts, whereas 4% rejected grafts between 2 and 7 months after transplantation. Of the 4 rejecting patients, 3 are alive with successful second engraftments. Acute grade II GVHD was seen in 21% of patients, grade III in 7%, and grade IV in 1% of patients. Chronic GVHD was seen in 32% of patients, most of whom responded completely to immunosuppressive therapy. With a median follow-up of 6.0 years (range, 0.5-11.6 years), the survival rate was 88%. No unusual long-term side effects have been seen with the regimen. We conclude that the cyclophosphamide/antithymocyte globulin regimen combined with methotrexate/cyclosporine after transplantation is well tolerated and effective in heavily pretreated patients with aplastic anemia.
authors.affiliation: Clinical Research Division, Fred Hutchinson Cancer Research Center, University of Washington School of Medicine, Seattle 98109, USA. rstorb@fhcrc.org
authors.forename: R
authors.initials: R
authors.lastname: Storb
authors.forename: K G
authors.initials: KG
authors.lastname: Blume
authors.forename: M R
authors.initials: MR
authors.lastname: O'Donnell
authors.forename: T
authors.initials: T
authors.lastname: Chauncey
authors.forename: S J
authors.initials: SJ
authors.lastname: Forman
authors.forename: H J
authors.initials: HJ
authors.lastname: Deeg
authors.forename: W W
authors.initials: WW
authors.lastname: Hu
authors.forename: F R
authors.initials: FR
authors.lastname: Appelbaum
authors.forename: K
authors.initials: K
authors.lastname: Doney
authors.forename: M E
authors.initials: ME
authors.lastname: Flowers
authors.forename: J
authors.initials: J
authors.lastname: Sanders
authors.forename: W
authors.initials: W
authors.lastname: Leisenring
mesh_terms: D000293:Adolescent; D000328:Adult; D000741:Anemia, Aplastic / Q000628:therapy*; D000961:Antilymphocyte Serum / Q000008:administration & dosage*; D016026:Bone Marrow Transplantation; D002648:Child; D002675:Child, Preschool; D003520:Cyclophosphamide / Q000008:administration & dosage*; D005260:Female; D006084:Graft Rejection / Q000517:prevention & control; D006801:Humans; D007166:Immunosuppressive Agents / Q000008:administration & dosage*; D008297:Male; D008875:Middle Aged; D014184:Transplantation, Homologous; D016896:Treatment Outcome
chemical_list: D000961:Antilymphocyte Serum; D007166:Immunosuppressive Agents; D003520:Cyclophosphamide