Monoclonal antibody T-cell-depleted HLA-haploidentical bone marrow
transplantation for Wiskott-Aldrich syndrome
SL Rumelhart, ME Trigg, SD Horowitz and R Hong
Department of Pediatrics, University of Iowa, Iowa City.
Four patients with Wiskott-Aldrich syndrome received bone marrow
transplants (BMT) using monoclonal antibody T cell-depleted HLA-
haploidentical marrow from a family member donor. The patients did not
receive a significantly larger inoculum of mature T cells than other
recipients of T cell-depleted marrow transplants. All four patients
achieved quick engraftment, and three of the four patients are alive and
well today. The three living patients have all had a complete return of
normal T-cell and B-cell function. Infectious complications in the
surviving patients were minimal; however, all three experienced some degree
of graft-versus-host disease (GVHD). Two of these three patients received
GVHD prophylaxis. The patient not receiving GVHD prophylaxis experienced
severe GVHD and had a difficult posttransplant course. The patient who did
not survive was chronically ill before BMT, whereas the other patients were
in relatively good health at the time of BMT. Since the majority of
individuals with this disease lack a matched bone marrow donor, our results
using partially matched donors suggest that a greater number of patients
can be successfully treated for Wiskott-Aldrich syndrome and that outcome
is related to control of GVHD and state of health before BMT. Marrow
transplantation should be offered earlier in the disease course before the
onset of major infectious problems.
Volume 75,
Issue 4,
pp. 1031-1035,
02/15/1990
Copyright © 1990 by The American Society of Hematology
