Prophylaxis vs. on‐demand treatment with BAY 81‐8973, a full‐length plasma protein‐free recombinant factor VIII product: results from a randomized trial (LEOPOLD II)

WOS: 000350548500005
PubMed ID: 25546368
BackgroundBAY 81-8973 is a new full-length human recombinant factor VIII product manufactured with technologies to improve consistency in glycosylation and expression to optimize clinical performance. ObjectivesTo demonstrate superiority of prophylaxis vs. on-demand therapy with BAY 81-8973 in patients with severe hemophilia A. Patients/MethodsIn this multinational, randomized, open-label crossover study (LEOPOLD II; ClinicalTrials.gov identifier: NCT01233258), males aged 12-65years with severe hemophilia A were randomized to twice-weekly prophylaxis (20-30IUkg(-1)), 3-times-weekly prophylaxis (30-40IUkg(-1)), or on-demand treatment with BAY 81-8973. Potency labeling for BAY 81-8973 was based on the chromogenic substrate assay or adjusted to the one-stage assay. Primary efficacy endpoint was annualized number of all bleeds (ABR). Adverse events (AEs) andimmunogenicity were also assessed. ResultsEighty patients (on demand, n=21; twice-weekly prophylaxis, n=28; 3-times-weekly prophylaxis, n=31) were treated and analyzed. MeanSD ABR was significantly lower with prophylaxis (twice-weekly, 5.7 +/- 7.2; 3-times-weekly, 4.3 +/- 6.5; combined, 4.9 +/- 6.8) vs. on-demand treatment (57.7 +/- 24.6; P
Bayer HealthCare AG, Leverkusen, GermanyBayer AGBayer Healthcare Pharmaceuticals; Bayer HealthCare PharmaceuticalsBayer AGBayer Healthcare Pharmaceuticals
This study was funded by Bayer HealthCare AG, Leverkusen, Germany. Medical writing assistance was provided by Erin P. Scott for Complete Healthcare Communications, Inc. (Chadds Ford, PA) and was fully funded by Bayer HealthCare Pharmaceuticals.