Performance of a clinical risk prediction model for inhibitor formation in severe haemophilia A.

ABSTRACT:

Background

There is a need to identify patients with haemophilia who have a very low or high risk of developing inhibitors. These patients could be candidates for personalized treatment strategies.

Aims

The aim of this study was to externally validate a previously published prediction model for inhibitor development and to develop a new prediction model that incorporates novel predictors.

Methods

The population consisted of 251 previously untreated or minimally treated patients with severe haemophilia A enrolled in the SIPPET study. The outcome was inhibitor formation. Model discrimination was measured using the C-statistic, and model calibration was assessed with a calibration plot. The new model was internally validated using bootstrap resampling.

Results

Fir

SUBMITTER: Hassan S 

PROVIDER: S-EPMC8360203 | biostudies-literature | 2021 Jul

REPOSITORIES: biostudies-literature