Faculty Collaboration Database

Unrelated donor bone marrow transplantation to treat severe aplastic anaemia in children and young adults. Br J Haematol 1996 Jul;94(1):65-72

Date

07/01/1996

Pubmed ID

8757510

DOI

10.1046/j.1365-2141.1996.d01-1772.x

Scopus ID

2-s2.0-8944254249 (requires institutional sign-in at Scopus site)   88 Citations

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.

Author List

Margolis D, Camitta B, Pietryga D, Keever-Taylor C, Baxter-Lowe LA, Pierce K, Kupst MJ, French J 3rd, Truitt R, Lawton C, Murray K, Garbrecht F, Flomenberg N, Casper J

Author

David A. Margolis MD Chair, Professor in the Pediatrics department at Medical College of Wisconsin

MESH terms used to index this publication - Major topics in bold

Adolescent
Adult
Anemia, Aplastic
Bone Marrow Transplantation
Child
Child, Preschool
Female
Follow-Up Studies
Graft Survival
Graft vs Host Disease
Humans
Infant
Male
Survival Analysis
Treatment Outcome