Reconstitution of normal megakaryocytopoiesis and immunologic functions in
Wiskott-Aldrich syndrome by marrow transplantation following myeloablation
and immunosuppression with busulfan and cyclophosphamide
N Kapoor, D Kirkpatrick, RM Blaese, J Oleske, MH Hilgartner, RS Chaganti, RA Good and RJ O'Reilly
Three patients with Wiskott-Aldrich syndrome received transplants of marrow
from their HLA-A, B, C, D identical siblings after myeloablation with
busulfan, 2 mg/kg/day x 4 days, followed by immunosuppression with
cyclophosphamide, 50 mg/kg/day x 4. Sustained engraftment of lymphoid and
hematopoietic elements was documented in each case. Platelet counts in
excess of 100,000/cu mm were restored 20--50 days posttransplant and remain
in the normal range 6--12 mo later. Platelets exhibit normal size and in
vitro aggregation. The patients produce isoagglutinins and antibodies to
other polysaccharides. The use of busulfan in moderate dosages as a
myeloablative agent, coupled with cyclophosphamide, may offer an improved
alternative to the use of lethal total body irradiation as a preparative
regimen for complete correction of Wiskott- Aldrich syndrome by marrow
transplantation.
Volume 57,
Issue 4,
pp. 692-696,
04/01/1981
Copyright © 1981 by The American Society of Hematology
