Serum Metabolomic Profiling Reveals Potential Biomarkers in Assessing The Management of Women with Polycystic Ovary Syndrome: A Randomized Controlled Trial

Abstract Background: As one of the most common endocrinal disorder for the women at childbearing age, the diagnostic criteria of polycystic ovary syndrome (PCOS) have been defined distinctly among different international health organizations. The diverse manifestations and heterogenic etiology of PCOS also bring about difficulties for its diagnosis and management assessment. Though the elevated androgen level is acknowledged to be the most obvious biomarker for PCOS diagnosis and management evaluation, it is not always present in patients and the mechanism of hyperandrogenism has not been fully elucidated. Therefore, more efficient biomarkers representing progression of PCOS are expected to be integrated into monitoring the management process using metabolomic approach. Methods: In this prospective randomized controlled trial, classical diagnostic parameters, blood glucose, and metabolome were measured in 123 PCOS patients simultaneously before and at 2 and 3 months of different medical interventions. The metabolite fingerprints were detected filtered out by comparing data at baseline and 3 months based on multivariate statistical analysis, followed by validation with receiver operating characteristic (ROC) curve on data collected at 2 months. Results: A set of metabolites including glutamic acid, aspartic acid, 1-methylnicotinamide, acetylcarnitine, glycerophosphocholine, and oleamide, were filtered out with high performance in representing the improvement through three-month management of PCOS with high sensitivity and specificity in ROC analysis. Conclusions: The 6 metabolites were representative for the remission of PCOS through medical intervention, making them a set of potential biomarkers on assessing the outcome of PCOS management. Trial registration: ClinicalTrials.gov, NCT03264638. Registered 29 August 2017 - Retrospectively registered, https://clinicaltrials.gov/ct2/show/NCT03264638