Recombinant single-chain factor VIII in severe hemophilia: Long-term safety and efficacy in previously treated patients in the AFFINITY extension study.

ABSTRACT:

Background

rVIII-SingleChain is a recombinant single-chain factor VIII used to treat people with hemophilia A.

Objectives

The aim of this extension study was to investigate the long-term safety and efficacy of rVIII-SingleChain prophylaxis in ≥200 previously treated patients (PTPs) with hemophilia A with ≥100 exposure days (EDs).

Methods

In total, 222 patients were enrolled, of which 204 rolled over from prior rVIII-SingleChain studies. The median age was 21 years (range, 2-65 years), including 155 patients ≥12 years and 67 patients <12 years. Patients continued with their previously assigned dose and regimen, or switched at the investigator's discretion. Patients were treated for a mean duration of 31 months (range, 1-47 months), the mean ED was 342 (standard deviati

SUBMITTER: Mahlangu J 

PROVIDER: S-EPMC8844480 | biostudies-literature | 2022 Feb

REPOSITORIES: biostudies-literature