Real-world evidence on Kovaltry (81-8973) in children with moderate or severe hemophilia A in Europe: a nested cohort analysis

Authors: O’Hara, J, Hirst, C, Cabre M, Jose F, and Burke, T.

Publication: Orphanet Journal of Rare Diseases; 16,1:33; January 2021

Affiliations: Department of Health and Social Care, University of Chester, Chester, UK; HCD Economics, Daresbury, UK ; Real World Evidence, Bayer, Basel, Switzerland ; Global Medical Affairs, Bayer, Mexico City, Mexico

Abstract: Untreated hemophilia A patients may experience recurrent bleeding events leading to debilitating joint damages. While RCT and pharmacokinetic data support the value of Kovaltry [an unmodified full-length recombinant factor VIII (FVIII) product], real world evidence in children is lacking. This report describes a descriptive and multivariate analysis of the effectiveness of Kovaltry in children with hemophilia A in the real-world setting, using data from medical chart abstraction and cross-sectional surveys of physicians, patients, and caregivers.