Adverse events should be reported. Reporting forms and information can be found at www.mhra.gov.uk/yellowcard or search for MHRA Yellow Card in the Google Play or Apple App Store. Adverse events should also be reported to Roche Products Ltd. Please contact Roche Drug Safety Centre by emailing firstname.lastname@example.org or calling +44 (0)1707 367554.
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: September 2021