TITAN (caplacizumab)
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
41.0% male
N = 75
75 patients (44 female, 31 male).
Inclusion criteria: patients with an acute episode of acquired TTP with a platelet count of < 100,000/mcL, without active bleeding, and who required plasma exchange.
Key exclusion criteria: platelet count ≥ 100,000/mcL; severe active infection; antiphospholipid syndrome; DIC; hematopoietic stem cell or bone marrow transplantation-associated thrombotic microangiopathy; known congenital TTP; active bleeding or high risk of bleeding; uncontrolled arterial hypertension; pregnancy or lactation.
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).
Primary outcome
Median time to response
3 days
4.9 days
4.9 days
3.7 days
2.5 days
1.2 days
0.0 days
Caplacizumab
Placebo
Significant
decrease ▼
Significantly shorter 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/mcL, 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.
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