Statistical analysis of clinical data
ADVERSE REACTIONS, REGRESSION
Analyses of clinical outcomes were performed using SASIn relation to WBM frequency, we considered two sub-cohorts as presenting with clinically relevant bowel movement patterns at baseline, defined as children with <4 WBMs and children with <5 WBMs. This second cohort is a superset of the first. In other words, the population of children with baseline WBMs up to 4 WBMs (e.g., including 0, 1, 2, or 3) is referred to as “<4 WBMs” in accordance with the protocol primary outcome definition. The population of children with baseline WBMs up to 5 WBMs (e.g., including 0, 1, 2, 3, or 4) is referred to as “<5 WBMs.”Endpoints assessed were (i) increase of ≥1 WBM from baseline to day 84, (ii) increase of ≥2 WBMs from baseline to day 84, (iii) increase of ≥3 WBMs from baseline to day 84, and (iv) KINDLE quality of life (QOL questionnaire) as a standardized tool to assess adverse reactions and tolerability. We additionally measured if children with >4 WBMs experienced a change in WBM frequency as a function of treatment.Binary endpoints (1, 2, and 3) were analyzed by logistic regression, with adjustment for age and baseline number of WBM. The continuous endpoint 4 was analyzed with covariance (ANCOVA), with adjustment for baseline and age. Cross-tabulations of the three responder endpoints with the treatment provided counts and percentages. Summary statistics were provided for a change in number of WBMs by subject groupings.