Summary

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• This summary is based on the review of one systematic review. ^[1]
• In individuals with inhibitors, emicizumab reduced Annualized Bleed Rate (ABR) for all bleeds, treated bleeds, and spontaneous bleeds but did not reduce Annualized Joint Bleed Rate (AjBR) or Annualized Target Joint Bleed Rate (AtjBR). Fitusiran reduced ABR for all bleeds, treated bleeds, joint bleeds, and spontaneous bleeds, with no evidence for AtjBR reduction. Concizumab may reduce ABR for all bleeds, treated bleeds, joint bleeds, and spontaneous bleeds but did not affect target joint bleeds. The occurrence of zero bleeds increased 11.31-fold with emicizumab, 12.5-fold with fitusiran, and 1.59-fold with concizumab. Health-Related Quality of Life (HRQoL) improved with all three therapies.
• In individuals without inhibitors, emicizumab (1.5 mg/kg weekly and 3.0 mg/kg bi-weekly) reduced ABR for all bleeds, treated bleeds, and joint bleeds, with the higher dose likely reducing spontaneous bleeds. Fitusiran reduced ABR for all bleeds, treated bleeds, joint bleeds, and spontaneous bleeds. Zero bleeds occurred in 50% with emicizumab (1.5 mg/kg weekly), 40% with emicizumab (3.0 mg/kg bi-weekly), and 40% with fitusiran compared to 0% or 5% in on-demand groups. HRQoL improved with fitusiran and emicizumab (3.0 mg/kg bi-weekly) but not with emicizumab (1.5 mg/kg weekly).
• Non-serious adverse events were higher with non-clotting factor therapies compared to on-demand therapy; injection site reactions were the most frequent events, and transient antidrug antibodies (ADA) were reported for fitusiran and concizumab.
• No significant differences in serious adverse events were observed between prophylaxis and on-demand therapy for emicizumab and fitusiran in participants without inhibitors; no treatment-related cancer or mortality was reported.
• The review included males aged 12–75 years with congenital hemophilia A or B, showing that prophylaxis with emicizumab, fitusiran, and concizumab reduced bleeding rates and improved HRQoL in individuals with inhibitors (though concizumab had very low-certainty evidence). Prophylaxis with emicizumab or fitusiran reduced bleeding rates and improved HRQoL in individuals without inhibitors (noting that some doses of emicizumab did not improve HRQoL).