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Therapy of severe aplastic anemia in young adults and children with
allogeneic bone marrow transplantation
PB McGlave, R Haake, W Miller, T Kim, J Kersey and NK Ramsay
Department of Medicine, University of Minnesota Hospital, Minneapolis
55455.
During an 8-year period, 28 young adults (median age 27 years) and 30
children (median age 10 years) with severe aplastic anemia have received
allogeneic bone marrow transplantation (BMT) from major histocompatibility
locimatched sibling donors after preparation with cyclophosphamide and
total lymphoid irradiation (TLI). All recipients were previously
transfused. Comparison of post-bone marrow transplantation events in adults
and children reveals equivalent median time to engraftment, median duration
of hospitalization, median Karnofsky assessment of activity, and equivalent
low rejection rate. Although the incidence of moderate and severe acute
graft-v-host disease (GVHD) and of extensive chronic GVHD was greater in
adults than in children, the projected survival at 4 years of adults (67%;
95% confidence interval [CI] 49% to 85%) and of children (73%; 95% CI 57%
to 89%) was equivalent. All survivors are transfusion-free and have normal
peripheral blood counts. One of 28 adults and 2 of 30 children have
experienced rejection, and 1 of these patients survives after a second
transplant. No malignancies have been identified following transplantation.
An unexpectedly high incidence of hypothyroidism has been detected and may
be attributable to preparation of recipients with TLI. Therapy of severe
aplastic anemia with allogeneic BMT after preparation with cyclophosphamide
and TLI offers a high rate of transfusion-free survival and a low rejection
rate in previously transfused young adults and children.
Volume 70,
Issue 5,
pp. 1325-1330,
11/01/1987
Copyright © 1987 by The American Society of Hematology
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