Low-dose immune tolerance induction alone or with immunosuppressants according to prognostic risk factors in Chinese children with hemophilia A inhibitors.

ABSTRACT:

Background

In developing countries, children with hemophilia A (HA) with high-titer inhibitor and poor immune tolerance induction (ITI) prognostic risk(s) cannot afford the recommended high- or intermediate-dose ITI.

Objectives

To determine the efficacy of low-dose ITI (plasma-derived factor VIII [FVIII]/von Willebrand factor at 50 FVIII IU/kg every other day) by itself (ITI-alone) or combined with immunosuppressants rituximab and prednisone (ITI-IS) in children with HA with high-titer inhibitor.

Methods

All enrolled patients had pre-ITI inhibitor ≥10 BU. We used ITI-alone if inhibitor titer was <40 BU pre-ITI and during ITI, and ITI-IS if titer was ≥100 BU (historic) or ≥40 BU (pre- or during ITI) or if the patient was nonresponsive on ITI-alone.

Results

Fifty

SUBMITTER: Li Z 

PROVIDER: S-EPMC8279128 | biostudies-literature | 2021 Jul

REPOSITORIES: biostudies-literature