Low-dose immune tolerance induction in children with severe hemophilia A with high-titer inhibitors: Type of factor 8 mutation and outcomes.

ABSTRACT:

Background

No studies evaluated the role of F8 mutations in outcomes for low-dose immune tolerance induction (ITI) in people with severe hemophilia A (SHA) with high-titer inhibitors.

Objectives

To explore the association between F8 mutation types and low-dose ITI outcomes in children with SHA with high-titer inhibitors.

Methods

Children SHA with high-titer inhibitors who received low-dose ITI therapy at least for 1 year were included in this study. Based on the risk of inhibitor development, F8 mutations were classified into a high-risk group and a non-high-risk group. Rapid tolerance and the final ITI outcomes were assessed at the 12th and 24th month of treatment, respectively, and the predictor of outcomes was analyzed.

Results

Of 104 ch

SUBMITTER: Sun J 

PROVIDER: S-EPMC9606347 | biostudies-literature | 2022 Oct

REPOSITORIES: biostudies-literature