Long-term outcomes after allogeneic stem cell transplantation for children with hematological malignancies

We analyzed long-term outcomes and psycho-social aspects in 112 children with malignancies surviving 1 year after hematopoietic stem cell transplantation. At 10 years, overall survival was 75 +/- 5%, TRM 18 +/- 4% and relapse 14 +/- 3%; 10-year cumulative incidence of infections was 31 +/- 4%, cataract 44 +/- 4%, pulmonary dysfunction 20 +/- 4%, bone and joint complications 29 +/- 5%, hypothyroidism 36 +/- 4%, cardiac complications 11 +/- 3% and secondary malignancies 7 +/- 3%. Total body irradiation (TBI) was the most significant risk factor associated with cataract, pulmonary impairment, osteoarticular complications and hypothyroidism. Chronic graft-versus-host disease was associated with higher incidence of pulmonary dysfunction. The number of complications per patient increased with time. Half of the patients had psychological disturbance, 13 signs of depression and 16 a history of eating behavior disorders; 54% of patients with one or more long-term complications had psychological problems. Sixty-nine patients had learning difficulties and 36 achieved normal scholarship. With increased follow-up, development of late effects and of psycho-social disturbance are of major concern. While the use of single-dose TBI has now been abandoned, other risk factors are still of concern in the early 2000s.