title: Unrelated donor bone marrow transplantation to treat severe aplastic anaemia in children and young adults.
abstract: Alternative donor bone marrow transplantation (BMT) to treat severe aplastic anaemia (SAA) in children and young adults has been complicated by high rates of graft rejection and severe graft-versus-host disease (GVHD). We hypothesized that increased immunosuppression combined with T-cell depletion of the marrow graft would enable successful use of unrelated donor BMT in this disease. Preconditioning consisted of cytosine arabinoside, cyclophosphamide, and total body irradiation (TBI). T-cell depletion was with the anti-CD3 antibody T10B9. GVHD prophylaxis consisted of cyclosporine A. 28 previously transfused patients were transplanted. Nine donor/recipient pairs were HLA matched. As of 1 January 1996, 15/28 (54%) patients are alive, transfusion independent and well with a range of follow-up of 13 months to 8 years (median 2.75 years). Fatalities include all three patients with nonengraftment and all three patients with grade III/IV GVHD. Other fatalities were due to infections or therapy-related toxicity. The incidence >or= grade II acute GVHD was 28%. These data show that in children with SAA who have failed immunosuppression, unrelated donor BMT offers a reasonable hope of long-term survival.
authors: Department of Pediatrics, Medical College of Wisconsin, Milwaukee, USA.
authors: D
authors: D
authors: Margolis
authors: B
authors: B
authors: Camitta
authors: D
authors: D
authors: Pietryga
authors: C
authors: C
authors: Keever-Taylor
authors: L A
authors: LA
authors: Baxter-Lowe
authors: K
authors: K
authors: Pierce
authors: M J
authors: MJ
authors: Kupst
authors: J
authors: J
authors: French
authors: R
authors: R
authors: Truitt
authors: C
authors: C
authors: Lawton
authors: K
authors: K
authors: Murray
authors: F
authors: F
authors: Garbrecht
authors: N
authors: N
authors: Flomenberg
authors: J
authors: J
authors: Casper
mesh_terms: D000293:Adolescent; D000328:Adult; D000741:Anemia, Aplastic / Q000628:therapy*; D016026:Bone Marrow Transplantation / Q000009:adverse effects / Q000379:methods*; D002648:Child; D002675:Child, Preschool; D005260:Female; D005500:Follow-Up Studies; D006085:Graft Survival; D006086:Graft vs Host Disease / Q000209:etiology; D006801:Humans; D007223:Infant; D008297:Male; D016019:Survival Analysis; D016896:Treatment Outcome