Low-dose immune tolerance induction for children with hemophilia A with poor-risk high-titer inhibitors: A pilot study in China.

ABSTRACT:

Background

Immune tolerance induction (ITI) therapy is currently unaffordable in China. Management of hemophilia A children with high-titer inhibitor is therefore a challenge.

Aim

To describe the ITI strategy using plasma-derived factor VIII/von Willebrand factor concentrate (pdFVIII/VWF) +/- immunosuppression and to report its efficacy in children with hemophilia A having poor-risk status for ITI success.

Methods

A prospective pilot study on children with hemophilia A having poor-risk status (all with at least inhibitor titer > 10 BU pre-ITI initiation). Patients received ~50 IU/kg FVIII every other day using domestic intermediate purity pdFVIII/VWF products, either alone or in combination with rituximab +/- prednisone.

Results

Sixteen patients with median age

SUBMITTER: Li Z 

PROVIDER: S-EPMC6781932 | biostudies-literature | 2019 Oct

REPOSITORIES: biostudies-literature