Treatment-Specific Clinical and Urinary Biomarker Signatures Associated With Response to Intravesical Botulinum Toxin...

To estimate response to intravesical onabotulinumtoxinA (BoNT-A) injection and platelet-rich plasma (PRP) therapy in females with bladder pain syndrome (BPS) without Hunner lesions, we developed and internally validated treatment-specific predictive models integrating clinical characteristics, bladder functional parameters, and urinary biomarkers.

Females with BPS who underwent intravesical BoNT-A injection or PRP therapy were retrospectively analyzed. A total of 273 female patients were included in the final analysis. Multivariable logistic regression models were constructed to predict treatment response, defined as a Global Response Assessment score ≥ 2. Model discrimination was assessed using the area under the receiver operating characteristic (ROC) curve. For interpretability, nomogram visualizations and a conceptual framework are provided in the Supporting Information.

The BoNT-A model demonstrated acceptable discrimination (area under the ROC curve: 0.789), whereas the PRP model showed superior discriminatory performance (area under the ROC curve: 0.895). Distinct clinical features and biomarker patterns contributed to each treatment-specific model, suggesting differential underlying mechanisms. Internal validation revealed higher observed response rates when the administered therapy matched the model-predicted higher probability of response, supporting internal model concordance.

Treatment-specific models combining clinical, urodynamic, and urinary biomarker data provide mechanistic insight into heterogeneous response profiles to BoNT-A and PRP in females with BPS. While these models may inform future phenotype-guided research and prospective validation, external validation is required before broader clinical implementation.