Background: Fifty-three children (39 male, 14 female, median age 9 years 3 months) with different forms of leukemia underwent bone marrow transplantation (BMT) at our center. Various conditioning regimens were used according to the disease and time of BMT. In this paper we evaluate the impact of the experience of a pediatric hematology center on BMT-related problems in children.
Methods: We analyzed disease-free survival (DFS), early BMT-related effects, hepatic, cardiac and respiratory function and late endocrine effects as shown by standard instrumental and laboratory tests.
Results and conclusions: Outcome (overall median follow-up 34 months) was satisfactory. Three years DFS was 50.1% in all patients, 58.8% in lymphoblastic leukemia in 2nd complete remission (CR), and 50.0% in acute myeloid leukemia (some in 2nd or subsequent CR). Three of four patients with chronic myeloid leukemia were alive at 38 months. Management of the problems causing early post-BMT toxicity contributed to a progressive fall in early morbidity and mortality (14.3% in the last 3 years). Pre-BMT hepatitis in most patients was not associated with increased post-BMT hepatotoxicity. Cardiac function, even in patients given aggressive anthracycline treatment before BMT, remained normal 3 years after transplantation, as did respiratory function, although 8 cases presented subclinical restrictive and/or obstructive alterations. Compensated hypothyroidism was observed in 9 patients. Six boys received replacement treatment for hypogonadism. Severe height impairment was seen in 2 patients. Post-BMT endocrine and growth effects require a longer follow-up for definitive conclusions to be drawn.(ABSTRACT TRUNCATED AT 250 WORDS)