Medical College of Wisconsin
CTSICores SearchResearch InformaticsREDCap

Bone marrow transplantation for children with severe aplastic anemia: use of donors other than HLA-identical siblings. Blood 1989 Oct;74(5):1852-7 PMID: 2529004

Pubmed ID



Eighty-five percent of untransfused and 70% of transfused patients with severe aplastic anemia (SAA) are cured with bone marrow transplants from histocompatible sibling donors. Use of partially matched family donors or unrelated donors has been relatively unsuccessful because of high incidences of graft rejection and graft-versus-host disease (GVHD). Thirteen children with SAA received marrow grafts from alternative donors (sibling 4, parent 5, unrelated 4). The first three patients were pretreated with cyclophosphamide (CYCLO) +/- irradiation and received methotrexate for GVHD prophylaxis. Subsequent children were pretreated with CYCLO + high-dose cytosine arabinoside + methylprednisolone + total body irradiation, had monoclonal antibody T-cell depletion of the donor marrow, and received cyclosporine for GVHD prophylaxis. Three heavily transfused patients with haploidentical-related donors failed to engraft and died. All 10 patients with more closely matched donors engrafted. Acute GVHD was grade II in only one patient (non-T-depleted); this patient is the only one with severe chronic GVHD. Three engrafted patients died (Pneumocystis pneumonia, systemic parainfluenza, venocclusive disease). Seven children are alive 33+ to 2,692+ days. Donors for the survivors were siblings 3, parent 1, unrelated 3. These data suggest that bone marrow transplantation from closely matched donors other than histocompatible siblings can be effective therapy for SAA if an intensive conditioning regimen is used. These results must be confirmed with larger numbers and longer follow-up.

Author List

Camitta B, Ash R, Menitove J, Murray K, Lawton C, Hunter J, Casper J


Bruce M. Camitta MD Clinical Professor in the Medicine department at Medical College of Wisconsin


2-s2.0-0024430673   45 Citations

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

Anemia, Aplastic
Bone Marrow Transplantation
Child, Preschool
HLA Antigens
Histocompatibility Testing
Lymphocyte Culture Test, Mixed
Tissue Donors
jenkins-FCD Prod-353 9ccd8489072cb19f5b9f808bb23ed672c582f41e