Table I.

Bleeding rates observed with licensed extended plasma half-life (EHLs) factor VIII (FVIII) products and non-replacement factor therapy.

Product and clinical trial	Regimen	Overall ABR (median)	Zero bleeding ratesa
rFVIII-Fc (Eloctate®)
A-LONG29	Individualised prophylaxis (25–65 IU/kg every 3–5 days) (n=117)	1.6	45.3%
	QW prophylaxis (65 IU/kg) (n=23)	3.6	17.4%
	Episodic treatment (10–50 IU/kg) (n=23)	33.6	0
Kids A-LONG30	Twice-weekly prophylaxis (25 IU/kg and 50 IU/kg on days 1 and 4) (n=69)	1.96	NR
ASPIRE (extension study)b,31	A-LONG patients:
	Individualised prophylaxis (n=108)	0.66	38.9%
	QW prophylaxis (n=27)	2.03	22.2%
	Modified prophylaxis (n=17)	1.97	23.5%
	Episodic treatment (n=14)	18.36	-
	Kids A-LONG patients:
	Individualised prophylaxis <6 years (n=29)	0	NR
	Individualised prophylaxis 6–12 years (n=30)	1.54	NR
	Individualised prophylaxis <6–12 years (n=59)	NR	59.3%
	Modified prophylaxis <6 years (n=1)	6.55	NR
	Modified prophylaxis 6–12 years (n=1)	0	NR
	Modified prophylaxis <6–12 years (n=2)	NR	50%
BAX 855 (Adynovate®)
PROLONG-ATE23	Twice-weekly prophylaxis (45±5 IU/kg for 6 months [≥50 EDs]) (n=120)	1.9	39.6%
	On-demand treatment (10–60±5 IU/kg) (n=17)	41.5	-
Pediatric trial (NCT02210091)37	Twice-weekly prophylaxis (50±10 IU/kg for 6 months [≥50 EDs])	2.0	38%
BAY 94-9027 (Jivi®)
PROTECT VIII44	Twice-weekly prophylaxis (30–40 IU/kg), not eligible for randomisation (n=13)	4.1	15.4%
	Twice-weekly prophylaxis (30–40 IU/kg), eligible for randomisation (n=11)	1.9	45.5%
	Every 5 days prophylaxis (45–60 IU/kg) (n=43)	1.9	44.2%
	QW prophylaxis enroled (60 IU/kg) (n=43)	3.9	37.2%
	QW prophylaxis completed (60 IU/kg) (n=32)	1.0	50%
	On-demand treatment (n=20)	23.4	-
PROTECT VIII extension46	Any PROTECT VIII regimen (n=36)	1.2c	NR
	Twice-weekly prophylaxis (n=4)	1.5
	Every 5 days prophylaxis (n=10)	1.1
	QW prophylaxis (n=8)	0.4
	Variable frequency (n=14)	2.0
N8-GP (Esperoct®)
Pathfinder 249	Every 4 days prophylaxis (50 IU/kg) (n=175)	1.33	40%
	On-demand treatment (n=12)	30.87	0
Pathfinder 2 extension50	Every 4 days prophylaxis (50 IU/kg) (n=17)	0.0	52.9%
	QW prophylaxis (75 IU/kg) (n=38)	0.0	57.9%
Pathfinder 552	Twice-weekly prophylaxis (50–75 IU/kg) (n=68)	1.95	42.6%
Emicizumab (Hemlibra®)
HAVEN 357	QW prophylaxis (1.5 mg/kg) (n=36)	2.5d	50%
	Q2W prophylaxis (3.0 mg/kg) (n=35)	2.6d	40%
	No prophylaxis (n=18)	47.6d	0
HAVEN 458,59	Q4W prophylaxis (6 mg/kg) (n=41; 5 with inhibitors)	4.5d,e	29%

Data cannot be directly compared between studies due to differences in trial design and patient populations.

^aPatients with no bleeding episodes during the trial.

^bInterim data.

^cMedian annualised bleeding rate (ABR) during the full 5-year period.

^dAll bleeding events, regardless of treatment with FVIII.

^eMedian ABR during the expansion phase.

NR: not reported; QW: once weekly; Q2W: every 2 weeks; Q4W: every 4 weeks.