Dose tailoring of human cell line-derived recombinant factor VIII simoctocog alfa: Using a limited sampling strategy in patients with severe haemophilia A.

ABSTRACT:

Aims

The use of factor VIII (FVIII) prophylaxis in haemophilia A is considered the standard of care, particularly in children. Despite adjustment of doses for body weight and/or age, a large pharmacokinetic (PK) variability between patients has been observed. PK-tailored prophylaxis may help clinicians adjust coagulation factor FVIII activity (FVIII:C) to the desired level, which may differ in individual patients. The objective was to develop a population PK model for simoctocog alfa based on pooled clinical trial data and to develop a Bayesian estimator to allow PK parameters in individual patients to be estimated using a reduced number of blood samples.

Methods

PK data from 86 adults and 29 children/adolescents with severe haemophilia A were analysed. The FVIII data measur

SUBMITTER: Delavenne X 

PROVIDER: S-EPMC6422655 | biostudies-literature | 2019 Apr

REPOSITORIES: biostudies-literature