HAVEN 3 demonstrated significantly fewer treated bleeds with HEMLIBRA prophylaxis vs. episodic FVIII2
HAVEN 3 was a randomised, multicentre, open-label, phase III study in 152 adult and adolescent males with severe congenital haemophilia A (endogenous FVIII activity, <1%) without inhibitors, ≥12 years; treated with episodic FVIII or FVIII prophylaxis in the prior 24 weeks.2,3
In the HAVEN 3 study, treated annualised bleed rate (ABR; primary efficacy endpoint) was 38.2 with episodic FVIII (95% CI, 22.9–63.8; Arm C, n=18) compared with:2
- 1.5 with HEMLIBRA 1.5 mg/kg/QW, a 96% reduction (95% CI, 0.9–2.5; Arm A, n=36; p<0.001)
- 1.3 with HEMLIBRA 3.0 mg/kg/Q2W, a 97% reduction (95% CI, 0.8–2.3; Arm B, n=35; p<0.001)
56% and 60% of patients had zero treated bleeds with HEMLIBRA QW (95% CI, 0–18; Arm A, n=20/36) and HEMLIBRA Q2W (95% CI, 42–76; Arm B, n=21/35) vs. 0% with episodic FVIII (95% CI, 0–18; Arm C, n=0/18).2
Significantly fewer treated bleeds with HEMLIBRA prophylaxis vs. prior FVIII prophylaxis (intra-individual comparison of patients from non-interventional study, Arm D)2
Treated ABR was 4.8 with prior FVIII prophylaxis (95% CI, 3.2–7.1) compared with:2
- 1.5 with HEMLIBRA 1.5 mg/kg/QW, a 68% reduction (95% CI, 1.0–2.3; Arm D, n=48; risk ratio 0.32; 95% CI, 0.20–0.51; p<0.001)
54% of patients had zero treated bleeds with HEMLIBRA QW (95% CI, 39–69; n=26/48) vs. 40% with prior FVIII prophylaxis (95% CI, 26–55; Arm D, n=19/48).2
For further details of the intra-individual study please see Study designs
For full study details please refer to the HAVEN 3 publication
HAVEN 1 demonstrated significantly fewer treated bleeds with HEMLIBRA prophylaxis vs. episodic bypassing agents (BPAs)4
HAVEN 1 was a randomised, multicentre, open-label, phase III study in 109 adult and adolescent males with congenital haemophilia A with FVIII inhibitors ≥12 years, ≥40 kg; treated with episodic BPAs or BPA prophylaxis in the prior 24 weeks.4,5
In HAVEN 1, treated ABR (primary efficacy endpoint) was 23.3 with episodic BPAs (95% CI, 12.3–43.9, Arm B, n=18) compared with:4
- 2.9 with HEMLIBRA 1.5 mg/kg/QW, an 87% reduction (95% CI, 1.7–5.0; Arm A, n=35; p<0.001)
62.9% of patients had zero treated bleeds with HEMLIBRA QW (95% CI, 44.9–78.5; Arm A, n=22/35) vs. 5.6% with episodic BPAs (95% CI, 0.1–27.3; Arm B, n=1/18).4,5
For details of the HAVEN 2 and HAVEN 4 non-randomised trials please see Efficacy (non-randomised trials)
For details on safety please see Safety
For full study details please refer to the HAVEN 1 publication
ABR, annualised bleed rate; BPA, bypassing agent; CI, confidence interval; FVIII, Factor VIII; QW, every week; Q2W, every 2 weeks.
References:
- HEMLIBRA Summary of Product Characteristics.
- Mahlangu J et al. N Eng J Med 2018;379:811–22.
- Mahlangu J et al. N Eng J Med 2018;379:811–22 [Supplementary Appendix].
- Oldenburg J et al. N Eng J Med 2017;377:809–18.
- Oldenburg J et al. N Eng J Med 2017;377:809–18 [Supplementary Appendix].
RCUKEMIC00223a
Date of preparation: November 2019
