A new paradigm for personalized prophylaxis for patients with severe haemophilia A.

ABSTRACT:

Aim

For patients with severe haemophilia A, guidelines recommend prophylactic treatment with FVIII, with dose calculations targeting a predetermined FVIII trough level. However, this pharmacokinetic (PK) approach is suboptimal, with some patients experiencing breakthrough bleeds. We aimed to improve FVIII dosing by incorporating the thrombin generation assay, a global haemostasis assay whose main pharmacodynamic (PD) parameter, endogenous thrombin potential (ETP), predicts spontaneous bleeding risk.

Methods

We performed post hoc combined PK-PD modelling using data from 66 adults who received human-cl rhFVIII (Nuwiq^® , Octapharma AG) in a phase IIIb study. Time-to-event analyses simulated the probability of spontaneous bleeding for different FVIII exposures and bas

SUBMITTER: Delavenne X 

PROVIDER: S-EPMC7154752 | biostudies-literature | 2020 Mar

REPOSITORIES: biostudies-literature