Anouk Donners

133 Entire-vial dosing of emicizumab Table 2. Bleeds before and during emicizumab therapy (n = 112) according to age subgroups. All (n = 112) Adults/adolescents (n = 80) Children (n = 32) Before During Before During Before During Total follow-up in person-years 131 96 81 67 37 29 Follow-up in median (IQR) weeks 56 (52–68) 39 (19–58) 53 (52–64) 37 (17–56) 67 (53–77) 50 (29–63) Total number of bleeds 442 74 360 55 82 19 Mean ABR (95% CI) 3.6 (2.9–4.4) 0.8 (0.6–1.1) 4.0 (3.2–5.3) 0.8 (0.6–1.2) 2.3 (1.5–3.5) 0.7 (0.4–1.2) Total number of joint bleeds 271 34 257 29 14 5 Mean AJBR (95% CI) 2.2 (1.8–2.8) 0.4 (0.2–0.6) 2.9 (2.3–3.9) 0.4 (0.3–0.7) 0.4 (0.2–0.7) 0.2 (0.1–0.5) Total number of muscle bleeds 72 13 57 11 15 2 Notes: Only treated bleeds were considered. Abbreviations: ABR: annualized bleed rate; AJBR: annualized joint bleed rate; CI: confidence interval; IQR: interquartile range. 7

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