Integrated proteomic and metabolomic profiling reveals sex-stratified biomarkers predicting chronicity in paediatric primary immune thrombocytopenia.

This study aimed to develop prediction models for chronic immune thrombocytopenia (ITP) using a sex-stratified proteomic and metabolomic approach, providing a framework for individualized prognosis evaluation and timely clinical management. This investigation was designed as a non-interventional, prospective observational cohort. Plasma samples were collected from 67 children initially diagnosed with ITP along with 40 healthy controls. After a minimum of 1 year of regular follow-up, participants were classified according to sex and disease progression. The male and female cohorts each comprised individuals with chronic ITP, non-chronic ITP and healthy controls. From each subgroup, three peripheral blood samples were randomly chosen for proteomic and metabolomic profiling. Integrative omics were analysed for correlations using Pearson's coefficient (threshold: |r| > 0.8, p < 0.05). Predictive models were constructed using sex-specific biomarkers associated with chronic progression. The analysis identified intercellular adhesion molecule-1 (ICAM-1) and biopterin in males and actin, alpha 2, smooth muscle, aorta (ACTA-2) and N6-acetyl-L-lysine in females as associated factors. Cross-sex applications of these biomarkers revealed limited predictive value. Furthermore, the receiver operating characteristics curves, calibration curves and clinical decision curve analysis demonstrated good predictive efficacy of these predictive models. The underlying mechanisms of interaction between these biomarkers, sex differences and ITP chronicity progression warrant further investigation.