Trial design
A randomized, multicenter, open-label, clinical trial 


Patient population

  • 152 adult and adolescent males (aged ≥12 years and ≥40 kg)
  • With hemophilia A without FVIII inhibitors
  • Randomized comparison: 89 patients previously treated with on-demand FVIII
  • Intra-patient comparison: 63 patients previously treated with prophylactic FVIII

Maintenance doses

  • Randomized comparison: HEMLIBRA 1.5 mg/kg QW, HEMLIBRA 3 mg/kg Q2W, or no prophylaxis
  • Intra-patient comparison: HEMLIBRA 1.5 mg/kg QW

Aim for zero treated bleeds

The majority of hemophilia A patients receiving HEMLIBRA had zero bleeds requiring treatment.

 


Long-term descriptive analysis of zero treated bleeds in adults and adolescents without FVIII inhibitors (HAVEN 3). 10

 


Making a change can make a difference

Patients without FVIII inhibitors receiving HEMLIBRA saw a 68% reduction in treated bleed rate vs prior FVIII prophylaxis. 1

  • Median ABR in the intra-patient comparison was 0 (IQR: 0; 2.1) with HEMLIBRA vs 1.8 (IQR: 0; 7.6) with prior FVIII prophylaxis

ABR was calculated with a negative binomial regression model, accounting for the difference in follow-up times. 1

“I think you’ll agree, that this is also clinically important…to go from roughly 5 bleeds a year to 1-2 [treated] bleeds per year.” 2

—Dr Guy Young, MD, Keck School of Medicine of the University of Southern California

Target joint bleeds

As observed over at least 24 weeks (HAVEN 3), patients taking HEMLIBRA saw a reduction in treated target joint bleeds.

A post hoc, descriptive analysis of target joint resolution§ in patients taking HEMLIBRA (HAVEN 3). 10

  • 99.2% target joint resolution in patients taking HEMLIBRA
  • At study entry, 97 patients reported a combined total of 238 target joints
  • The median duration of exposure was 84 weeks
  • This analysis was conducted post hoc. There was no pre-specified statistical procedure controlling for type 1 error rate. Such analyses do not ascertain whether the findings were attributable to treatment with HEMLIBRA, or merely due to change, and are susceptible to bias. Conclusions based on this analysis may not be valid

Target joints were defined as major joints (eg, hip, elbow, wrist, shoulder, knee, and ankle) in which ≥3 spontaneous bleeds occurred over a 24-week period. 10
§Target joint resolution was defined as ≤2 spontaneous bleeding events in a 52-week period in a joint previously defined as a target joint. 10ABR=annualized bleed rate; CI=confidence interval; FVIII=factor VIII; IQR=interquartile range; QW=once weekly; Q2W=once every 2 weeks.

Adults and Adolescents With Inhibitors

Learn about HEMLIBRA for adults and adolescents with FVIII inhibitors