Identification of key biomarkers for initial corticosteroid resistance in newly diagnosed primary immune thrombocytopenia

Corticosteroid resistance is a major obstacle to the management of patients with primary immune thrombocytopenia (ITP), as the factors contributing to the variability in treatment response remain largely unknown. We analyzed a broad set of plasma protein biomarkers from corticosteroid-sensitive patients (CSp; n = 30), corticosteroid-resistant patients (CRp; n = 26), and healthy controls (HCs; n = 25) using a 92-plex immunoassay from Olink Proteomics Technology. A total of 54 inflammation-related proteins demonstrated significant differences among the 3 groups. Twenty-seven biomarkers showed statistical differences between CSp and CRp. Machine learning-based feature selection identified 4 potential biomarkers which were closely related to corticosteroid resistance: CXCL10, IL-1α, glial-cell-line-derived neurotrophic factor (GDNF), and CCL11. A Nomograph model was developed based on these 4 biomarkers, demonstrating remarkable discriminative ability, with an area under the curve (AUC) of 0.920 (95% confidence interval: 0.830 – 1.000) in this exploratory cohort. While requiring external validation in larger studies, the identification of the 4 potential biomarkers suggested their value in predicting corticosteroid resistance in patients with newly diagnosed ITP, and might guide the initial choice of treatment.