Table 2.

Efficacy of damoctocog alfa pegol in the PROTECT VIII phase II/III study in previously treated male patients aged 12–65 years with severe haemophilia A

Treatment	Dosing interval	No. of mITT pts	Run-in ABRa	Median ABR			Average doseb
			(median)	Totalc	Joint	Spontaneous	(IU/kg per infusion)
Main studyd [26, 29]
On-demand		20		23.4	16.3	14.3	35.5
Prophylaxis	Twice weekly, non-eligible for randomization	13	17.4	4.1	4.0	3.9	38.9
	Twice weekly, eligible but not randomized	11	0	1.9	1.9	0	31.5
	Every 5 days	43	0	1.9	1.9	0	45.3
	Every 7 dayse	43	0	3.9	1.9	1.9	56.8
	Total prophylaxis	110		2.1	1.9	0
Extension studyf [29]
On-demand		14		33.7	20.5	20.7	32.8
Prophylaxis	Twice weekly	23		1.8	0.8	0.8	36.7
	Every 5 days	34		1.3	1.0	0.7	45.1
	Every 7 days	23		0.7	0.3	0	59.3
	Variable frequencyg	27		3.2	2.4	1.9	52.1
	Total prophylaxis	107		1.6	0.9	0.8	46.6

ABR annualized bleeding rate, mITT modified intention-to-treat, pts patient

^aWeeks 1–10; fixed twice-weekly dose of 25 IU/kg per infusion

^bMean dose reported in main phase of the main study; median dose reported in extension phase

^cPrimary endpoint in main efficacy phase; weeks 0–36 in on-demand treatment arm, weeks 11–36 for prophylaxis arms

^dSome data for spontaneous and joint ABRs and average dose are reported from other sources [13, 17, 31]

^eIncludes data from pts that remained in (n = 32) and pts that left (n = 11) the treatment arm

^fData assessed over median duration of 166 weeks in extension study (data cut-off August 2017)

^gIncludes pts who switched dosing regimens during the extension phase