Longitudinal Evaluation of Immunological Biomarkers in Previously Untreated/Minimally Treated Patients With Severe and Moderately Severe Haemophilia A During Exposure to Factor VIII: Results From the HEMFIL Study.

ABSTRACT:

Background

Haemophilia A (HA) is an inherited bleeding disorder due to Factor VIII (FVIII) deficiency. Treatment with FVIII can activate immune mechanisms, which may lead to inhibitor development.

Objectives

This study aimed to perform a longitudinal and exploratory analysis of immunological biomarkers during replacement with FVIII concentrate and after immune tolerance induction (ITI) in patients who developed a high-responding inhibitor.

Methods

Biological samples and clinical data from severe and moderately severe persons with HA (PwHA; FVIII < 0.02 IU/mL) were obtained before any or after minimal exposure (≤5 days) to FVIII (T0), at inhibitor development (INB+), at 75 exposure days (ED) without inhibitor (INB-) (T1) and at end of ITI (T2). Biomarkers were assessed

SUBMITTER: Antonio Portugal Santana M 

PROVIDER: S-EPMC12311890 | biostudies-literature | 2025 Jul

REPOSITORIES: biostudies-literature