Individualised prophylaxis based on personalised target trough FVIII level optimised clinical outcomes in paediatric patients with severe haemophilia A.

ABSTRACT:

Introduction

As standard care of severe haemophilia A (SHA), prophylaxis should be individualised.

Aim

This study aimed to investigate the effectiveness of this new-proposed individualised prophylaxis protocol.

Methods

Boys with SHA were enrolled and followed a PK-guided, trough-level escalating protocol of prophylaxis after a six-month observational period. In the next 2 years, clinical assessments including joint bleeds, ultrasound (US) scores and Haemophilia Joint Health Score (HJHS) in both sides of ankles, knees and elbows were conducted every 6 months as a scoring system, which determined whether the trough level's escalation. Adjustment of dosing regimen was based on WAPPS-Hemo.

Results

Fifty-eight SHA boys were finally analysed. Their age and bodyweight

SUBMITTER: Huang K 

PROVIDER: S-EPMC9796890 | biostudies-literature | 2022 Nov

REPOSITORIES: biostudies-literature