Treatment and Disease Burden in a Cohort of People With Haemophilia Without Inhibitors in the United States.

ABSTRACT:

Introduction

Haemophilia is a bleeding disorder caused by a deficiency in coagulation factors VIII or IX. Great advances in haemophilia treatment have been achieved in recent decades, with a range of products for prophylaxis now available, the ability to individualise therapy, and resultant improvements in life expectancy and quality of life. Despite these advances, there remains an unresolved burden, both from the disease and its treatment. Real-world data provide an opportunity to understand the experiences of people with haemophilia and gain insight into the areas of greatest unmet need.

Aim

To assess the treatment and disease burden of haemophilia using real-world data.

Methods

Medical records data and patient-reported outcomes from a self-selected cohort of 446 p

SUBMITTER: Wheeler AP 

PROVIDER: S-EPMC12462568 | biostudies-literature | 2025 Sep

REPOSITORIES: biostudies-literature