Summary: Transplantation of hematopoietic stem cells is a therapy of choice for some highrisk malignancies for 25 years. As it is combined with highly toxic chemotherapy and/or irradiation its use is associated with both early and late morbidity and mortality. Long-term follow up of all patients is therefore essential in our continuing efforts to improve this therapeutic modality. In our institution we have transplanted a total of 33 children (21 boys and 12 girls, mean age 12.6 years, range 1.3-20.3 y) in 1992-1994. The average follow-up period of the patients (including pre-transplant) is currently 67.7 months (13.1-126.9, median = 72). The group is subdivided also according the diagnosis and the pre-transplantation therapeutic protocol used. In 31 pts autologous grafts (either BM or PBSC) were used; 2 pts received allogeneic sibling grafts. Fractionated total body irradiation was used in 9 pts and as a single dose in pts. Eighteen pts (54.5 %) are alive; 14 (42.4 %) in CR, 1 (3 %) in PR and 3 (9.1 %) with disease progression. Fifteen pts died; 3 in the peritransplant period {(30d)-2 of mycotic sepsis and 1 of multiple organ dysfunction). No mortality occured in the early post-transplant period (30-100 d). Symptoms of organ toxicity were recorded as per the CCG 3891 protocol; the following side-effects were noted according the WHO criteria: Hepatopathias 1st degree in 2 pts (6.7 %), 2nd degree in 1 pts (3.3 %); nephropathias 1st degree in pts (10 %), 2nd degree in 1 pts (3.3 %); pneumopathias 1st degree in 4 pts (13.3 %), 2nd degree in 2 pts (6.7 %), 3rd degree in 3 pts (10 %); cardiomyopathies in 1 pt requiring medication; slightly compromised hearing in 4 pts (13.3 %), partial hearing loss requiring aid in 1 pt (3.3 %); sicca syndr. in 1 pt (no eye cataracts were observed). Growth retardation (>2 SD) was observed in 2 pts (6.7 %); amenorrhea occured in 7 girls (70 %). So far the limited number of pts in the follow-up does not allow for statistical correlations with the diagnosis and the protocols used; a higher incidence of pneumopathias and growth retardation seems to occur in patients with TBI. The above outcomes'seem and peritransplant mortality to be in line with reports from other paediatic BMT centres.
Long-term adverse effect of hematopoietic stem cell transplantation in children with cancer
