Late effects of chemotherapy compared to bone marrow transplantation in the treatment of pediatric acute myeloid leukemia and myelodysplasia

Background. As more pediatric patients with acute myeloid leukemia (AML) an d myelodysplastic syndrome (MDS) survive, comparison of the late effects of various therapies becomes increasingly important. This study of survivors of AML is the largest to date comparing the late effects of patients treate d with chemotherapy (CT) with or without irradiation (RT) or CT followed by bone marrow transplantation (BMT). Procedure. In a retrospective review of 228 patients with AML or MDS from 1970 to 1995, 62 survived and had follow -up data available more than 1 year following completion of therapy. Ten pa tients with Down syndrome were excluded. Twenty-six received CT and 26 unde rwent BMT. Weight and height Z scores, endocrine, ophthalmologic, renal, an d cardiac function following CT +/- RT or BMT +/- total body irradiation (T BI) were compared at a mean follow-up of 7.4 and 5.6 years, respectively. R esults. Both groups experienced a dec rement in height and increase in weig ht. The mean height Z score in the CT group fell from -0.29 to -0.72 (P = 0 .02) and mean weight Z score rose from -0.06 at diagnosis (T0) to 0.51 at l ast follow-up (T2) (P = 0.02), a finding no longer significant when patient s who received RT were excluded. The mean height Z score in the BMT group f ell from -0.17 at TO to -0.65 at T2 (P = 0.02), while the mean weight rose from 0.29 at TO to 0.84 at T2, (P = 0.07). Six of 9 BMT adolescent girls ex perienced ovarian failure Versus 0 of 11 girls treated with CT (P = 0.002). Seven adolescent CT males and seven BMT males showed normal pubertal progr ession. Two BMT patients require thyroid hormone supplementation, and one r eceives growth hormone. Six BMT patients and one CT patient developed catar acts, all of whom received irradiation (P = 0.10). Serum creatinine level, hypertension, or left ventricular shortening fraction were not different in the two groups. One BMT patient has chronic graft versus host disease. Con clusions. Growth, renal, and cardiac functions were similar in the two grou ps. The need for estrogen supplementation was more frequent following BMT. Recommendations concerning therapy for AML should depend on the probability of cure. (C) 1999 Wiley-Liss, Inc.