Summary

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• This summary is based on the review of one systematic review/meta-analysis. ^[1]
• In males aged 12–75 years with congenital hemophilia A or B, fitusiran likely reduced annualized bleeding rates (ABR) for all bleeds (mean difference [MD] -28.80, 95% confidence interval [CI] -40.07 to -17.53), treated bleeds (MD -16.80, 95% CI -25.80 to -7.80), joint bleeds (MD -12.50, 95% CI -19.91 to -5.09), and spontaneous bleeds (MD -14.80, 95% CI -24.90 to -4.71); emicizumab likely reduced ABR for all bleeds (MD -22.80, 95% CI -37.39 to -8.21), treated bleeds (MD -20.40, 95% CI -35.19 to -5.61), and spontaneous bleeds (MD -15.50, 95% CI -24.06 to -6.94); concizumab may reduce ABR for all bleeds (MD -12.31, 95% CI -19.17 to -5.45), treated bleeds (MD -10.10, 95% CI -17.74 to -2.46), joint bleeds (MD -9.55, 95% CI -13.55 to -5.55), and spontaneous bleeds (MD -11.96, 95% CI -19.89 to -4.03).
• In the subgroup without inhibitors, fitusiran, emicizumab 1.5 mg/kg/week, and emicizumab 3.0 mg/kg bi-weekly likely reduced ABR for all bleeds, treated bleeds, and joint bleeds; fitusiran (MD -20.21, 95% CI -32.12 to -8.30) and emicizumab 3.0 mg/kg bi-weekly (MD -15.30, 95% CI -30.46 to -0.14) likely reduced spontaneous bleeds, while emicizumab 1.5 mg/kg/week did not (MD -14.60, 95% CI -29.78 to 0.58).
• The percentage of individuals with zero bleeds increased by 12.5-fold with fitusiran, 11.31-fold with emicizumab, and 1.59-fold with concizumab.
• Fitusiran, emicizumab, and concizumab were associated with higher non-serious adverse events compared to on-demand therapy; injection site reactions were the most frequently reported, and transient antidrug antibodies were noted for fitusiran and concizumab.
• No significant difference in the risk of serious adverse events was observed for fitusiran and emicizumab compared to on-demand therapy in participants without inhibitors; no treatment-related cancer or mortality was reported in any study group.
• In male participants aged 12 to 75 years, fitusiran, emicizumab, and concizumab likely reduced ABR and improved health-related quality of life (HRQoL) in those with inhibitors; in those without inhibitors, fitusiran and emicizumab similarly reduced ABR and improved HRQoL, though emicizumab 1.5 mg/kg/week did not improve some HRQoL scores.