Introduction: Pediatric allogeneic hematopoietic stem cell
transplantation (HSCT) is associated with severe treatment-related
complications, including mortality. Therefore, HSCT remains a therapeutic option
for patients with life-threatening medical conditions. After the introduction of
reduced toxicity conditioning regimen, we aimed to identify the interrelation
and the time to development of early transplant-related complications, as
graft-versus-host disease (GVHD), viral reactivations and
transplantation-associated thrombotic microangiopathy (TA-TMA). At the same time
we also aimed to analyze the role of different conditioning therapies in the
development of these complications. Patients and methods:
Hundred pediatric (age 8.5 ± 4.9 yrs) patients, treated with HSCT for malignant
(N = 54) and non-malignant (N = 46)
indications, have been enrolled in this prospective study. From the start of
conditioning therapy, early transplant-related comlications, as acute GVHD,
viral reactivations, and TA-TMA and the occurrence of relapse were monitored.
Results: Two third of the patients (67%) developed at least
one of the monitored early complications. The most frequent HSCT complication
was viral reactivation (50%). Acute GVHD occurred in 26% of patients, on median
day 31 (10–174). 27% of the acute GVHD cases (7/26) were graded as severe (Grade
III–IV). In the majority of patients, TA-TMA remained mild, self-limiting,
without any sign of organ damage, and occurred only during ciclosporin
immunosuppression. TA-TMA was preceded by acute GVHD in 5 in 20, by viral
reactivation in 5 in 20, or by both in 4 in 20 cases. In contrast to the low
incidence of early HSCT-related complications, relapse rate (22/54; 41%) was
high. Overall survival after a median 3.8 (1.3–6.5) year follow-up time was 74%.
Overall survival was excellent (43/46; 93.5%) in non-malignant patients. On the
contrary, in patients with malignant diseases, mortality rate (17/54, 31.5%) was
high due to high relapse rate. Conclusions: Since the
introduction of reduced toxicity conditioning regimen, the rate of severe
HSCT-related complications is low. TA-TMA occurred typically in a mild form
after GVHD and/or viral infection or reactivation. In patients with malignant
conditions, mortality was driven by high relapse rate.
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