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

Abstract

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 7575%, TRM 1874% and relapse 1473%; 10-year cumulative incidence of infections was 3174%, cataract 4474%, pulmonary dysfunction 2074%, bone and joint complications 2975%, hypothyroidism 3674%, cardiac complications 1173% and secondary malignancies 773%. 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 singledose TBI has now been abandoned, other risk factors are still of concern in the early 2000s.

Use of intrathecal chemoprophylaxis in children after SCT and the risk of central nervous system relapse

“…These are not specific parameters to detect neurological or cognitive sequelae but they do provide valuable and comparable information on the level of neurological function of the patient. 16,17 The observation period extends to only 192 months, and these complications and sequelae can occur and/or develop later post-HSCT. With the tools available, we could not identify any difference in the cognitive outcome between the patients with and without i.t.…”

Adverse health events and late mortality after pediatric allogeneic hematopoietic SCT—two decades of longitudinal follow-up

“…1 The cumulative burden of treatment-related morbidity after childhood HSCT is considerable [2][3][4][5] and seems to increase over time. 6 Conditioning with TBI is a risk factor for late effects when compared with chemotherapy-based conditioning. [2][3][4][5]7,8 There is a relatively limited literature evaluating very long-term effects of pediatric HSCT.…”

Examination of late pulmonary toxicity in children treated for malignancies

“…Sok esetben kifejezett volt a dinamikus paraméterek kóros változása (37%). Ez az eredmény jó-val magasabb a nemzetközi irodalomban megjelenő 8-10%-hoz képest [10].…”

“…A nemzetközi irodalomban 8-14%-ban obstruktív elváltozás tapasztalható [10], míg saját eseteinkben 37% volt az obstruktív elvál-tozás aránya. A vizsgálatunkban szereplő gyermekek át-lagéletkora megegyezett a nemzetközi tanulmányokban vizsgált gyermekek életkorával (9-32 év) [11].…”