Prophylactic therapy with emicizumab may demonstrate superior efficacy compared with factor VIII (FVIII) prophylaxis in patients with hemophilia A, according to study results published in Current Medical Research and Opinion.

The study involved a combined analysis of data from multiple trials, designed to compare bleed rates between emicizumab prophylaxis and FVIII prophylaxis in patients with hemophilia without inhibitors.

A network meta-analysis was performed on data from 4 studies found by literature review. These were the A-LONG, LEOPOLD 2, SPINART, and HAVEN 3 studies. The first 3 emphasized FVIII-based prophylaxis, and the phase 3 HAVEN 3 trial (152 patients; ClinicalTrials.gov Identifier: NCT02847637) focused on emicizumab prophylaxis.

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Subgroup analyses using results from the HAVEN 3 trial were also performed. Two subgroups were analyzed that featured patients categorized by prophylactic treatment patterns prior to trial initiation. Although the HAVEN 3 trial examined emicizumab prophylaxis, the patients in this trial had previously been treated with FVIII prophylaxis, with data available for direct comparisons of bleed rates with either prophylaxis treatment.

The network meta-analysis indicated that among the 4 examined trials, emicizumab prophylaxis enabled a lower total treated bleed rate compared with FVIII prophylaxis, with a rate ratio of 0.36 (95% CI, 0.13-0.95).

In the subgroup analyses of patients on the HAVEN 3 trial, similar results were obtained, regardless of subgroup. Rate ratios from subgroup analyses were 0.380 (95% CI, 0.186-0.790) and 0.472 (95% CI, 0.258-0.866).

The researchers investigated efficacy in this study, but comparison of safety profiles was not performed.

The authors stated in their report that this set of results “supports lower bleed rates with emicizumab prophylaxis [compared] with FVIII prophylaxis in patients with hemophilia A without inhibitors.”

Reference

  1. Reyes A, Révil C, Niggli M, et al. Efficacy of emicizumab prophylaxis versus factor VIII prophylaxis for treatment of hemophilia A without inhibitors: network meta-analysis and subgroup analyses of the intra-patient comparison of the HAVEN 3 trial [published online July 29, 2019]. Curr Med Res Opin. doi:10.1080/03007995.2019.1649378