Plasma metabolomics profile alterations in the onset and progression of paediatric immune thrombocytopenia

Immune thrombocytopenia (ITP) is an autoimmune disorder, characterized by immune-mediated platelet destruction and decreased platelet production. To investigate metabolic alterations associated with paediatric ITP and disease chronicity, we performed untargeted plasma metabolomics analysis in 60 newly diagnosed ITP (nITP) patients, 39 chronic ITP (cITP) patients and 39 healthy controls (HC) from Beijing Children's Hospital between October 2020 and August 2024. A total of 30 differential metabolites were identified between ITP patients and HC, with altered tryptophan metabolism among the most significantly enriched metabolic pathways. Random forest analysis achieved an accuracy of 83.9% and a precision of 95.1%. Glycocholic acid demonstrated strong discriminatory performance, with an area under the receiver operating characteristic curve of 0.882 (95% confidence interval: 0.814-0.950). In addition, phosphatidylcholine-related metabolites and sphingolipid-related metabolites were associated with metabolic alterations observed between nITP and cITP. Overall, paediatric ITP was associated with distinct plasma metabolic alterations, particularly involving tryptophan metabolism, bile acid metabolism and lipid metabolism. These findings provide additional insights into metabolic alterations associated with paediatric ITP and disease chronicity.