explorer7
Trial question
What is the role of concizumab prophylaxis in patients with hemophilia A or B with inhibitors?
Study design
Multi-center
Open label
RCT
Population
52 male patients.
Inclusion criteria: patients aged ≥ 12 years with hemophilia A or B with inhibitors.
Key exclusion criteria: hypersensitivity to the trial drug; known inherited or acquired coagulation disorder other than congenital hemophilia; ongoing/planned immune tolerance induction treatment; history of thromboembolic disease.
Interventions
N=33 concizumab prophylaxis (loading dose of 1.0 mg/kg of body weight, followed by 0.2 mg/kg/day for at least 32 weeks).
N=19 no prophylaxis (no injection for at least 24 weeks).
Primary outcome
Treated spontaneous and traumatic bleeding episodes
1.7
11.8
11.8 episodes
8.9 episodes
5.9 episodes
3.0 episodes
0.0 episodes
Concizumab
prophylaxis
No
prophylaxis
Significant
decrease ▼
Significant decrease in treated spontaneous and traumatic bleeding episodes (1.7 episodes vs. 11.8 episodes; RR 0.14, 95% CI 0.07 to 0.29).
Secondary outcomes
Significant decrease in spontaneous bleeding episodes (1.3 episodes vs. 9.4 episodes; RR 0.14, 95% CI 0.06 to 0.3).
Significant decrease in joint bleeding episodes (1.4 episodes vs. 9.1 episodes; RR 0.15, 95% CI 0.07 to 0.32).
Significant decrease in all treated and untreated bleeding episodes (4.4 episodes vs. 13.3 episodes; RR 0.33, 95% CI 0.17 to 0.64).
Safety outcomes
No significant difference in serious adverse events.
Conclusion
In patients aged ≥ 12 years with hemophilia A or B with inhibitors, concizumab prophylaxis was superior to no prophylaxis with respect to treated spontaneous and traumatic bleeding episodes.
Reference
Tadashi Matsushita, Amy Shapiro, Aby Abraham et al. Phase 3 Trial of Concizumab in Hemophilia with Inhibitors. N Engl J Med. 2023 Aug 31;389(9):783-794.
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