Table 3.

Efficacy of lonoctocog alfa in the prevention or reduction of bleeding episodes in patients with severe haemophilia A: results from the two pivotal trials

	In adults and adolescents aged ≥12 years [4, 12, 16]		In children aged 0 to <12 years [8, 12, 16]
Pt population	On-demand	Prophylaxis	On-demanda	Prophylaxis
No. of evaluable pts	27	146	3	80
Pts (%) with no treated bleeding episodes	3.7	43.2	0	26.3
Med AsBRb	11.73c	0.00*c	31.76	0
Med (overall) ABR	19.64d	1.14*d	78.56d	3.69d
Med ABR for traumatic bleeds	3.12d	0d	35.12	1.97

ABR annualized bleeding rate, AsBR annualized spontaneous bleeding rate, med median, pt(s) patient(s)

* p < 0.0001 vs. pts in the on-demand treatment group

^aAll on-demand recipients were aged ≥6 to <12 years; no formal comparisons of ABR between groups were performed

^bABR [4, 8] and AsBR [4] = no. of treated events × 365.25/efficacy evaluation period (excluded data from the pharmacokinetics and the surgical parts [4])

^cCo-primary efficacy endpoint

^dSecondary endpoint