Table 2.

Efficacy of lonoctocog alfa in the treatment of bleeding episodes in the two pivotal multicentre trials in patients with severe haemophilia A

	In adults and adolescents aged ≥12 years [4, 12, 16]			In children aged 0 to <12 years [8, 16]
Pt population	All pts	On-demand	Prophylaxis	All pts	On-demand	Prophylaxis
No. of evaluable pts	173	27	146	83	3	80a
No. of treated bleeding eps (IA/all)	835/848	577/590	258/258	347/347	132/132	215/215
Haemostatic efficacy rating (% bleeds)
Excellent or good (all bleeding eps)b,c	92.3	92.4	92.2	96.3	100.0d	94.0
Moderate (all bleeding eps)e	6.1	5.4	7.8	3.5	0	5.6
Excellent or good (IA bleeding eps)c	93.8	94.5	92.2	96.3	100.0d	94.0
Moderate (IA bleeding eps)e	6.2	3.8	7.8	3.5	0	5.6
Bleeding eps (%) controlled withf
1 inj	80.9	82.7	76.7	85.9	99.2	77.7
2 injs	12.6	12.0	14.0	9.8	0	15.8
≥3 injs	6.5	5.3	9.3	4.3	0.8	6.5
Overall med dose/inj/bleeding ep (IU/kg)	31.7	30.0	31.0	27.3	25.9	30.0
Overall med cumul dose/bleeding ep (IU/kg)	34.7	–	–	27.6	25.9	37.0g

Cumul cumulative, ep(s) episode(s), IA investigator-assessed, inj(s) injection(s), LON lonoctocog alfa, med median, pt(s) patient(s)

^aProphylaxis regimens were: every second day (n = 3), three times weekly (n = 24), twice weekly (n = 43), and other regimens (n = 10)

^bPrimary endpoint; missing data counted as treatment failures

^cExcellent = definite pain relief ± improvement in signs of bleeding within ≈8 h post 1st inj of LON; good = definite pain relief ± improvement in signs of bleeding at ≈8 h post 1st inj, but required 2 injs for complete resolution

^dHaemostatic efficacy was assessed as excellent in all (100%) treatment procedures

^eProbable/slight beneficial effect within ≈8 h post 1st inj of LON, requires >2 injs for complete resolution

^fSecondary endpoint. Based on all treated bleeding episodes; missing ratings counted as treatment failures

^gAcross all prophylaxis regimens, the median prophylaxis dose/subject was 342 IU/kg/month and 4109 IU/kg/year