Extension Study With rVIII-SingleChain in Previously Untreated Patients (PUPs) With Severe Haemophilia A.

ABSTRACT:

Introduction

Clinical trials and real-world evidence have demonstrated the efficacy and safety of rVIII-SingleChain in previously treated patients with haemophilia A.

Aim

To investigate the safety and efficacy of rVIII-SingleChain in previously untreated patients (PUPs).

Methods

In an open-label, phase 3, extension study, PUPs with severe haemophilia A (FVIII <1%) received rVIII-SingleChain prophylactically or on-demand. The primary endpoints were incidence of high-titre (HT) inhibitor formation to FVIII, treatment success for major bleeding episodes and annualised spontaneous bleeding rate (AsBR).

Results

Twenty-four PUPs (median age 1 year [range 0-5]) were treated with rVIII-SingleChain; median time on study was 35.0 months (range 2.4-54.0). Overall, six PUP

SUBMITTER: Mahlangu J 

PROVIDER: S-EPMC11957422 | biostudies-literature | 2025 Mar

REPOSITORIES: biostudies-literature