Efficacy, safety, and pharmacokinetics of emicizumab prophylaxis given every 4 weeks in people with haemophilia A (HAVEN 4): a multicentre, open-label, non-randomised phase 3 study

This study is registered with ClinicalTrials.gov, number NCT03020160, and is ongoing.FindingsBetween Jan 30, 2017, and Feb 27, 2017, seven patients were enrolled into the initial run-in cohort, which confirmed the expected pharmacokinetic profile and safety of the regimen based on model-based simulations, providing sufficient evidence for opening of the expansion cohort (n=41), which was recruited and enrolled between May 24, 2017, and June 30, 2017. The annualised rate of treated bleeds was 2·4 (95% CI 1·4–4·3). 23 (56·1%; 95% CI 39·7–71·5) of 41 reported no treated bleeds and 37 (90%; 76·9–97·3) reported zero to three treated bleeds. The annualised bleed rate was 4·5 (95% CI 3·1–6·6) for all bleeds, 0·6 (0·3–1·5), for treated spontaneous bleeds, 1·7 (0·8–3·7) for treated joint bleeds, and 1·0 (0·3–3·3) for treated target joint bleeds. The most frequent treatment-related adverse event was injection-site reaction (nine [22%] of 41 patients). We observed no thrombotic events or development of de-novo antidrug antibodies with neutralising potential or FVIII inhibitors.InterpretationEmicizumab given once every 4 weeks showed clinically meaningful bleed control while being well tolerated. This regimen could improve patient care by decreasing treatment burden and increasing adherence to effective prophylaxis, potentially decreasing the development of secondary complications for people with haemophilia A.FundingF Hoffmann-La Roche and Chugai Pharma...
Source: The Lancet Haematology - Category: Hematology Source Type: research