LONG-TERM OUTCOME AFTER MARROW TRANSPLANTATION FOR SEVERE APLASTIC-ANEMIA

We reviewed the records and reevaluated 212 patients with aplastic ane mia transplanted at the Fred Hutchinson Cancer Research Center (FHCRC) between 1970 and 1993 who survived greater than or equal to 2 years a nd who have been followed for up to 26 years. Parameters analyzed incl uded hematopoietic function, chronic graft-versus-host disease (GVHD), skin disease, cataracts, lung disease, skeletal problems, posttranspl ant malignancy, depression, pregnancy/fatherhood, and the return to wo rk or school, as well as patient self-assessment of physical and psych osocial health, social interactions, memory and concentration, and ove rall 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 patie nts had normal hematopoietic parameters. Skin problems occurred in 14% , cataracts in 12%, lung disease in 24%, and bone and joint problems i n 18% of patients. Eleven patients (12%) developed a solid tumor malig nancy 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 sch ool or work; the proportion increased to 90% by 20 years. At least hal f 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 transplantatio n in patients with aplastic anemia established long-term normal hemato poiesis. No new hematologic disorders occurred. The major cause of mor bidity and mortality was chronic GVHD. However, the majority of patien ts who survived beyond 2 years returned to a fully functional life. (C ) 1998 by The American Society of Hematology.