Statistical analysis
SE
ITTP, DISEASE, RECURRENCE
The planned sample size was 15 participants, based on feasibility considerations; no sample size calculation was performed. The success criterion for the study was ≤ 20% of evaluable participants (i.e., PP population) with a recurrence of iTTP during the overall study period. The PP population (the primary analysis population) included all participants in the mITT who completed treatment and follow-up, or had an iTTP recurrence, and no major protocol deviations. Any individual who was found to have a disease other than iTTP after enrollment was excluded from the PP population. The mITT and safety populations were both defined as all participants who received at least one dose of caplacizumab.In general, all endpoints were summarized descriptively using total numbers, means, standard errors (SE), medians, confidence intervals (CIs), and ranges (minimum–maximum) for continuous variables, and frequencies and percentages for categorical variables. Time-to-event data were summarized using Kaplan–Meier estimates.