Abstract
INTRODUCTION: Haemophilia A is a rare inherited bleeding disease caused by the deficiency of coagulation factor VIII (FVIII). The main treatment protocol is to administer regular exogenous FVIII concentrate infusions. With the discovery of variability in individualised pharmacokinetics (PK) and bleeding phenotype, the previous weight-based approach needs to be replaced by more advanced PK-tailored prophylaxis with an accurate evaluation system. In this study, we combine individualised PK profiles and a complementary evaluation system to guide prophylaxis in paediatric patients with haemophilia A. METHODS AND ANALYSIS: This is a single-centre, prospective single-arm study. The aim of this study is to assess the effectiveness of a new strategy combining PK and a complementary evaluation system to treat haemophilia A in Chinese paediatric patients. Sixty paediatric patients with haemophilia will be recruited. After PK testing, they will receive a PK-guided stepup prophylaxis in the next 2 years. The dosing regimen will be determined according to individualised PK profiles and complementary evaluation findings. Related indicators at the end of the study will be compared with the values at treatment initiation to examine the effectiveness of this new strategy. The demographic data of the investigated patients will be summarised by descriptive statistics. Quantitative data will be described by summary statistics, including arithmetic median, range, mean and arithmetic SD. Analyses will use t-test to compare indicators such as bleeding rate and imaging score at both ends of the study as well as during follow-up. ETHICS AND DISSEMINATION: The study has been approved by the Ethics Committee of Beijing Children's Hospital (Number 2020-Z-095). The findings will be presented at international meetings such as World Federation of Hemophilia World Congress. Related manuscripts will be submitted to peer-review journals such as Blood and Hemophilia. TRIAL REGISTRATION NUMBER: ChiCTR2000037821; Pre-results.