Table 3.

Bleeding outcomes in the overall population and in participants < 2 years old receiving treatment with emicizumab

	Overall	Moderate HA	Severe HA
Overall population	N = 106	n = 15	n = 91
Annualised bleeding rate, median (IQR)	0.91 (0.00–2.46)	0.00 (0.00–1.23)	1.10 (0.00–2.55)
Model-based annualised bleeding rate*, mean (95% CI)	2.3 (1.6–3.3)	NA	NA
Participants with zero bleeds, n (%)	57 (53.8)	11 (73.3)	46 (50.5)
Participants < 2 years old	N = 15	n = 1	n = 14
Annualised bleeding rate, median (IQR)	1.06 (0.00–1.46)	1.46 (1.46–1.46)	1.04 (0.00–1.35)
Participants with zero bleeds, n (%)	9 (60.0)	0 (0.0)	9 (64.3)

^*Model-based annualised bleeding rates are generated using negative binomial regression

CI confidence interval, HA haemophilia A, IQR interquartile range, NA not available