Statistical analysis plan
cGVHD, aGVHD
DISEASE, EVENT, ACUTE GVHD, CHRONIC GVHD, SECONDARY
The trial was not powered for confirmatory statistical testing of any pre-specified hypotheses. Following the approved PIP, at least 100 evaluable children had to be enrolled. Descriptive statistics including 95% confidence intervals (CI) was applied to summarize all endpoints, including baseline characteristics and covariates used in multivariate analyses. Three (2.9%) umbilical cord blood transplanted patients were included in the matched unrelated donor (2) and matched family donor (1) subgroup (Supplementary Table All time-to-event endpoints were measured from the day of HSCT (except for chronic GVHD [cGVHD] from 100 days after HSCT) to the event or competing event (if applicable). The probability of event over time for freedom from TRM, TRM, OS, and GVHD-free survival was estimated by Kaplan-Meier estimator, and for engraftment, primary and secondary graft failure until 12 months after HSCT, incidence of acute GVHD (aGVHD) and cGVHD was estimated by cumulative incidence functions due to competing risks. For comparisons, Pepe-Mori tests for engraftment were performed. Cox models for freedom from TRM, TRM, OS, and GVHD-free survival, and Fine and Gray models for engraftment, graft failure, and incidence of aGVHD and cGVHD were applied to adjust for covariates in multivariate analyses. The following covariates are additionally considered to examine efficacy and safety in prespecified subgroups or in multivariate analyses (disease groups, age group, donor type, thiotepa, and serotherapy). All analyses were predefined and SAS software (Version 9.4) was used.