AOZORA study: 3-year interim analysis of safety and joint health in pediatric people with hemophilia A receiving emicizumab prophylaxis.

ABSTRACT:

Background

Recurrent joint bleeding in people with hemophilia A (PwHA) can cause hemophilic arthropathy, resulting in limited movement and chronic pain. Emicizumab is a bispecific monoclonal antibody bridging activated factor (F)IX and FX to substitute for deficient activated FVIII in PwHA, thereby improving hemostasis.

Objectives

This 3-year interim analysis of the ongoing, open-label, phase IV AOZORA study (jRCT1080224629) analyzes medium-term safety and joint health in pediatric PwHA without FVIII inhibitors receiving emicizumab.

Methods

PwHA aged <12 years with severe hemophilia A without FVIII inhibitors were eligible. Participants entered AOZORA as emicizumab-naïve or having previously initiated emicizumab during the HOHOEMI study. Endpoints included safety, and

SUBMITTER: Shima M 

PROVIDER: S-EPMC12681530 | biostudies-literature | 2025 Nov

REPOSITORIES: biostudies-literature