6/30/23, 2:17 AM TITAN Pathway Feedback Search Clinical Topics Home Studies TITAN TITAN Disease Thrombotic thrombocytopenic p Trial question What is the effect of caplacizumab in patients with acquired TTP? Study design Multi-center Single blinded RCT Population Characteristics of study participants 59.0% female N = 75 41.0% male 75 patients (44 female, 31 male) Inclusion criteria: patients with an acute episode of acquired TTP with a platelet count of < 100,000/ L, without active bleeding, and who required plasma exchange Key exclusion criteria: platelet count 100,000/ L, severe active infection, antiphospholipid syndrome, DIC, pregnant or breastfeeding, hematopoietic stem cell or BMT-associated thrombotic microangiopathy, known congenital TTP, active bleeding or high risk of bleeding, uncontrolled arterial hypertension. Interventions N=36 caplacizumab (10 mg SC once daily during plasma exchange and for 30 days afterward) N=39 placebo (SC daily during plasma exchange daily and for 30 days afterward) https://web.pathway.md/studies/rec8rjup5T3QnSLAz 1/2 6/30/23, 2:17 AM TITAN Pathway Primary outcome Median time to response 4.9 days 4.9 3.7 days 3 2.5 days 1.2 days Significant decrease 0.0 days Caplacizumab Placebo Significant decrease in median time to response (3 days vs. 4.9 days; RR 0.61, 95% CI 0.18 to 1.04) Safety outcomes No significant difference in total adverse events. Significant difference in mild-to-moderate bleeding-related adverse events (54% vs. 38%). Conclusion In patients with an acute episode of acquired TTP with a platelet count of < 100,000/ L, without active bleeding, and who required plasma exchange, caplacizumab was superior to placebo with respect to median time to response. Reference Peyvandi F, Scully M, Kremer Hovinga JA et al. Caplacizumab for Acquired Thrombotic Thrombocytopenic Purpura. N Engl J Med. 2016 Feb 11;374(6):511-22. Open reference URL https://web.pathway.md/studies/rec8rjup5T3QnSLAz 2/2