Table 3.

Prophylactic efficacy of nonacog beta pegol in previously treated patients with haemophilia B in phase 3 trials

Treatment	No. of ptsa	No bleeds (% pts)	Annualized bleeding rates
			Statistic	Spontaneous	Traumatic	Joint	Total
Paradigm 2 [8]
10 IU/kg qw	30	16.7	Median (IQR)	0.97 (0.00–4.01)	0.98 (0.00–1.93)	NA	2.93 (0.99–6.02)
			Meanb (95% CI)	3.14 (1.78–5.56)	1.35 (0.81–2.24)	NA	4.56 (3.01–6.90)
40 IU/kg qw	29	44.8	Median (IQR)	0.00 (0.00–0.98)	0.00 (0.00–2.04)	0.97 (0.00–2.07)c	1.04 (0.00–4.00)
			Meanb (95% CI)	1.22 (0.48–3.10)	1.29 (0.76–2.19)	NA	2.51* (1.42–4.43)d
Paradigm 4 [15]
10 IU/kg qw	20	30.0	Median (IQR)	1.05 (0.00–2.16)	0.00 (0.00–1.01)	0.97 (0.00–1.41)	1.36 (0.00–2.23)
			Meanb (95% CI)	1.31 (0.63–2.73)	0.53 (0.20–1.39)	1.36 (0.56–3.33)	1.84 (1.00–3.38)
40 IU/kg qw	49	40.8	Median (IQR)	0.00 (0.00–1.00)	0.00 (0.00–1.10)	0.00 (0.00–1.97)	1.00 (0.00–2.03)
			Meanb (95% CI)	0.71 (0.38–1.33)	1.01 (0.64–1.57)	1.49 (0.87–2.54)	1.84 (1.26–2.70)
Total	66	34.8	Median (IQR)	0.00 (0.00–1.29)	0.00 (0.00–1.06)	0.59 (0.00–1.89)	1.05 (0.00–2.20)
			Meanb (95% CI)	0.89 (0.55–1.42)	0.87 (0.57–1.32)	1.46 (0.91–2.34)	1.84 (1.33–2.56)

See text for trial design details

IQR interquartile range, NA not available, qw once weekly, pt(s) patient(s)

*p = 0.01 based on 1-sided test of the null hypothesis that the estimated rate is at least 4.8, evaluated at the 2.5% level

^aRandomized pts (Paradigm 2) or pts who were on same treatment regimen for ≥ 3 months (Paradigm 4)

^bEstimate of mean based on a Poisson regression model

^cData from the EU summary of product characteristics [12]

^dProphylactic effect was confirmed based on a predefined criteria (i.e. the upper limit of the 95% CI was below 4.8, which corresponds to a > 60% reduction in a literature-based value of 12 bleeds/pt/year in pts receiving on-demand treatment)