Risk Factors Analysis and Validation of Early Treatment Non-response in Children with Primary ITP: A Multicenter Retrospective Cohort Study

Abstract Objective: To identify factors associated with early non-response to first-line treatment in children with primary immune thrombocytopenia (ITP), and to develop and externally validate a prediction model.

Methods

This multicenter retrospective cohort study included 335 children with newly diagnosed primary ITP as the derivation cohort, including 255 responders and 80 non-responders. Clinical data obtained after admission were collected. Candidate variables were screened using correlation analysis and least absolute shrinkage and selection operator (LASSO) regression, followed by multivariable logistic regression to identify factors associated with early treatment non-response. Model discrimination was evaluated using the area under the receiver operating characteristic curve (AUC). An independent external validation cohort of 78 children was additionally included to assess model discrimination.