Emicizumab for preventing intracranial hemorrhage in infants with severe hemophilia A: a cost-effectiveness analysis.

ABSTRACT:

Abstract

Intracranial hemorrhage (ICH) and resulting neurologic disability are severe complications for a subset of infants with severe hemophilia A (HA). Although prophylactic factor replacement reduces bleeding risk, it is typically delayed until after age 1 year due to risks associated with central venous access placement. Emicizumab, a subcutaneous activated factor VIII (FVIII) mimetic, has demonstrated safety and efficacy in preventing ICH in infants aged <12 months in the HAVEN 7 trial. Despite its high cost, the cost-effectiveness of emicizumab prophylaxis initiated during the first year of life for infants with severe HA is not known. We developed a Markov cohort model to compare emicizumab prophylaxis to standard care (no prophylaxis) in infants aged 0 to 1 year with sever

SUBMITTER: Glaeser-Khan S 

PROVIDER: S-EPMC12744270 | biostudies-literature | 2025 Dec

REPOSITORIES: biostudies-literature