Real-world bleeding rates on emicizumab: the value of using nationwide digital treatment diary data in clinical research.

ABSTRACT:

Background

People with hemophilia in the Netherlands log bleeds and infusions through a digital treatment diary. With the current innovations in hemophilia treatments, the use of patient-reported bleeding data will become increasingly important.

Objective

To assess real-world bleeding rates on emicizumab in a nationwide cohort of people with severe hemophilia A, and assess the value of digital treatment diary data.

Methods

People with severe hemophilia A of all ages with and without inhibitors using emicizumab who use the digital treatment diary were included. From 2018 to October 2023, data on bleeds treated with clotting factor concentrate were collected from digital treatment diaries and electronic health records. Mean (95% CI) annualized (joint) bleeding rates wer

SUBMITTER: Brands MR 

PROVIDER: S-EPMC11981740 | biostudies-literature | 2025 Feb

REPOSITORIES: biostudies-literature