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Long-Term Outcome After Marrow Transplantation for Severe Aplastic
Anemia
H. Joachim Deeg,
Wendy Leisenring,
Rainer Storb,
Janet Nims,
Mary E.D. Flowers,
Robert P. Witherspoon,
Jean Sanders, and
Keith M. Sullivan
From the Fred Hutchinson Cancer Research Center and the University of
Washington School of Medicine, Seattle, WA.
We reviewed the records and reevaluated 212 patients with aplastic
anemia transplanted at the Fred Hutchinson Cancer Research Center
(FHCRC) between 1970 and 1993 who survived  2 years and who have been
followed for up to 26 years. Parameters analyzed included hematopoietic
function, chronic graft-versus-host disease (GVHD), skin disease,
cataracts, lung disease, skeletal problems, posttransplant malignancy,
depression, pregnancy/fatherhood, and the return to work or school, as
well as patient self-assessment of physical and psychosocial health,
social interactions, memory and concentration, and overall severity of
symptoms. Survival probabilities at 20 years were 89% for patients
without (n = 125) and 69% for patients with chronic GVHD (n = 86)
(the status was uncertain in 1 surviving patient). All patients had
normal hematopoietic parameters. Skin problems occurred in 14%,
cataracts in 12%, lung disease in 24%, and bone and joint problems in
18% of patients. Eleven patients (12%) developed a solid tumor
malignancy and 19% of patients experienced depression. Chronic GVHD
was the dominant risk factor for late complications. Seventeen patients
died at 2.5 to 20.4 years posttransplant; 13 of these had chronic GVHD and related complications. At 2 years, 83% of patients had returned to
school or work; the proportion increased to 90% by 20 years. At least
half of the patients preserved or regained the ability to become
pregnant or father children. Patients rated their quality of life as
excellent and symptoms as minimal or mild. In conclusion, marrow
transplantation in patients with aplastic anemia established long-term
normal hematopoiesis. No new hematologic disorders occurred. The major
cause of morbidity and mortality was chronic GVHD. However, the
majority of patients who survived beyond 2 years returned to a fully
functional life.
Blood, Vol. 91 No. 10 (May 15), 1998:
pp. 3637-3645
© 1998 by The American Society of Hematology.
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