by Lori Solomon
Patients with hemophilia A or B with inhibitors have a lower annualized bleeding rate with concizumab than with no prophylaxis, according to a phase 3 study published online Aug. 31 in the New England Journal of Medicine.
Tadashi Matsushita, M.D., Ph.D., from Nagoya University Hospital in Japan, and colleagues evaluated the safety and efficacy of concizumab in patients with hemophilia A or B with inhibitors. As part of the explorer7 phase 3 trial, participants were randomly assigned to receive no prophylaxis for at least 24 weeks (group 1; 19 patients) or to receive concizumab prophylaxis for at least 32 weeks (group 2; 33 patients), or they were nonrandomly assigned to receive concizumab prophylaxis for at least 24 weeks (groups 3 and 4; 81 patients).
The researchers found that the estimated mean annualized bleeding rate in group 1 was 11.8 episodes versus 1.7 episodes in group 2 (rate ratio, 0.14; P < 0.001). For patients receiving concizumab (groups 2, 3, and 4), the overall median annualized bleeding rate was zero episodes. After concizumab therapy was restarted, no thromboembolic events were reported. Plasma concentrations of concizumab were stable over time.
“Concizumab represents a novel, subcutaneous treatment option in patients with hemophilia A or B with inhibitors that can potentially improve long-term outcomes,” the authors write.
More information: Tadashi Matsushita et al, Phase 3 Trial of Concizumab in Hemophilia with Inhibitors, New England Journal of Medicine (2023). DOI: 10.1056/NEJMoa2216455 H. Marijke van den Berg et al, Hemostasis—A Balancing Act, New England Journal of Medicine (2023). DOI: 10.1056/NEJMe2304535 Journal information: New England Journal of Medicine
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