Pharmacokinetics and pharmacodynamics
Plasma caplacizumab concentrations remained consistent throughout the treatment period (Supplementary Fig. S1). During the post-daily TPE period, mean plasma concentrations ranged from 611 ng/mL to 785 ng/mL.Mean levels of VWF:Ag (Supplementary Fig. S2) and FVIII:C (Supplementary Fig. S3) and VWF:RCo (Fig. Mean plasma levels of von Willebrand factor ristocetin cofactor (VWF:RCo) in the safety population. Error bars represent standard error of the mean; D2–3 is the daily TPE period, W1–W10 is the post-daily TPE period; VWF:RCo values of < 20% represent the threshold for pharmacologic activity of caplacizumab. The mean VWF:RCo of 29.2% at Week 4 (slightly above the 20% threshold) was caused by an aberrant data point of 279.6% from one participant who stopped caplacizumab due to physician decision, 