HAVEN 2 was a non-randomised, multicentre, open-label, phase III study in 88 paediatric males with congenital haemophilia A with inhibitors, <12 years, or aged 12–17 and <40 kg; previously treated with episodic bypassing agents (BPAs) or BPA prophylaxis.4
In the HAVEN 2 study, mean treated ABR (efficacy endpoint) was:4
- 0.3 with HEMLIBRA 1.5 mg/kg/QW (95% CI, 0.2–0.5; Arm A, n=65)
- 0.2 with HEMLIBRA 3.0 mg/kg/Q2W (95% CI, 0.0–1.7; Arm B, n=10)
- 2.2 with HEMLIBRA 6.0 mg/kg/Q4W (95% CI, 0.7–6.8; Arm C, n=10)
HAVEN 2 was designed as a descriptive study with no defined primary or secondary endpoints.4
Median (range) observation time: QW: 57.6 weeks (17.9–92.6); Q2W: 21.3 weeks (18.6–24.1); Q4W: 19.9 weeks (8.9–24.1).4
Efficacy assessment was conducted only in patients aged <12 years; analysis excludes three patients aged ≥12 years.4
For full study details please refer to the HAVEN 2 publication
76.9%, 90.0% and 60.0% of patients had zero treated bleeds with HEMLIBRA QW (95% CI, 64.8–86.5; n=50/65), Q2W (95% CI, 55.5–99.7, n=9/10) and Q4W (95% CI, 26.2–87.8, n=6/10).4
HAVEN 4 was a non-randomised, multicentre, open-label, phase III study in 48 adult and adolescent males with congenital haemophilia A, classified as severe or with FVIII inhibitors, ≥12 years; previously treated with BPAs or FVIII in the prior 24 weeks. Seven patients were part of an initial run-in cohort and were not included in the efficacy analysis.5
In the HAVEN 4 study, treated ABR (efficacy endpoint) was 2.4 with HEMLIBRA 6.0 mg/kg/Q4W (95% CI, 1.4–4.3; n=41).5
Median (range) efficacy period time: 25.6 weeks (24.1–29.4). Majority of treated bleeds were traumatic (75%; 38/51).5
Five patients had FVIII inhibitors at study entry.5
For full study details please refer to the HAVEN 4 publication
56.1% of patients had zero treated bleeds (95% CI, 39.7–71.5; n=23/41).5
ABR, annualised bleed rate; BPA, bypassing agent; CI, confidence interval; FVIII, Factor VIII; QW, every week; Q2W, every 2 weeks; Q4W, every 4 weeks; RCT, randomised clinical trial.
- HEMLIBRA Summary of Product Characteristics.
- Mahlangu J et al. N Eng J Med 2018;379:811–22.
- Oldenburg J et al. N Eng J Med 2017;377:809–18.
- Young G et al. Blood 2019;134:2127–38
- Pipe S et al. Lancet Haematol 2019;6:e295–e305.
Date of preparation: August 2020