6/28/23, 11:03 PM HERCULES Pathway Feedback Search Clinical Topics Home Studies HERCULES HERCULES Disease Thrombotic thrombocytopenic p Trial question What is the effect of caplacizumab in patients with acquired TTP? Study design Multi-center Double blinded RCT Population Characteristics of study participants 69.0% female N = 145 31.0% male 145 patients (100 female, 45 male) Inclusion criteria: patients with acquired TTP Key exclusion criteria: severe ADAMTS13 deficiency, thrombotic microangiopathies unassociated with TTP, such as HUS, or congenital TTP Interventions N=72 caplacizumab (10 mg IV bolus, then 10 mg SC daily during PLEX and for 30 days thereafter) N=73 placebo (standard of care, including PLEX, with matching placebo) Primary outcome Platelet count normalization 90.3 % 90.3 87.7 67.7 % https://web.pathway.md/studies/rec6gG92da5Vxed2B 1/2 6/28/23, 11:03 PM HERCULES Pathway 45.1 % Significant increase 22.6 % NNH = 38 0.0 % Caplacizumab Placebo Significant increase in platelet count normalization (90.3% vs. 87.7%; RR 1.55, 95% CI 1.09 to 2.19) Secondary outcomes Significant decrease in death due to TTP, recurrence, or thromboembolism (12% vs. 49%; RR 0.24, 95% CI 0.1 to 0.38) Significant decrease in recurrence of TTP during trial (12% vs. 38%; RR 0.32, 95% CI 0.13 to 0.51) Safety outcomes No significant differences in bleeding-related adverse events (65% vs. 48%), death during the trial (1 vs. 3 patients). Conclusion In patients with acquired TTP, caplacizumab was superior to placebo with respect to platelet count normalization. Reference Scully M, Cataland SR, Peyvandi F et al. Caplacizumab Treatment for Acquired Thrombotic Thrombocytopenic Purpura. N Engl J Med. 2019 Jan 24;380(4):335-346. Open reference URL https://web.pathway.md/studies/rec6gG92da5Vxed2B 2/2