Figure 1.
Patient disposition. *Primary reason: platelet count <100 000/mL at screening (n = 1), scheduled for surgical intervention (n = 1), history or presence of a VWF inhibitor at screening (n = 1), patient not willing or able to comply with protocol requirements (n = 2), VWD inclusion criteria not met (n = 1). †Patients who were treated on-demand with any VWF during the 12-month period before enrolling in this study. ‡Patients who were treated prophylactically with a pdVWF for ≥12 months before enrolling in this study. §Nonserious headache (moderate intensity), which was considered by the investigator to be possibly related to rVWF and began during an rVWF infusion. ‖One patient in each group withdrew consent for reasons unrelated to efficacy/bleeding, and 1 patient in the prior on-demand group was lost to follow-up. ¶Scheduled for extended treatment with hydrocortisone >10 mg per day (not permitted during the study). **Required treatment with high corticosteroid doses for rheumatoid arthritis (not permitted during the study). ††All patients who were enrolled and received any amount of rVWF. ‡‡All patients who received rVWF prophylaxis. §§All patients who received ≥1 rVWF infusion and provided ≥1 quantifiable PK/PD postdose measurement. FAS, full analysis set; PKFAS, pharmacokinetic full analysis set; SAS, safety analysis set.