Symptom heterogeneity in interstitial cystitis/bladder pain syndrome and chronic prostatitis/chronic pelvic pain syndrome, collectively termed urologic chronic pelvic pain syndrome (UCPPS), has resulted in difficulty in defining appropriate clinical trial endpoints. We determine clinically important differences (CIDs) for 2 primary symptom measures, Pelvic Pain Severity (PPS) and Urinary Symptom Severity (USS), and evaluate subgroup differences.
The Multidisciplinary Approach to the Study of Chronic Pelvic Pain Symptom Patterns Study enrolled individuals with UCPPS. We defined CIDs by associating changes in PPS and USS over 3 to 6 months with marked improvement on a global response assessment using regression and receiver operating characteristic curves. We evaluated CIDs for absolute and percent change and examined differences in CIDs by sex-diagnosis, presence of Hunner lesions, pain type, pain widespreadness, and baseline symptom severity.
An absolute change of -4 was clinically important in PPS among all patients, but CID estimates differed by pain type, presence of Hunner lesions, and baseline severity. PPS CID estimates for percent change were more consistent across subgroups and ranged from 30 to 57 percent. The absolute change USS CID was -3 for female participants and -2 for male participants with CP/CPPS only. Patients with greater baseline severity required larger decreases in symptoms to feel improved. Estimated CIDs had lower accuracy among participants with low baseline symptoms.
A reduction of 30-50% in PSS is a clinically meaningful endpoint for future therapeutic trials in UCPPS. USS CIDs are more appropriately defined separately for male and female participants.
The Journal of urology. 2023 Feb 27 [Epub ahead of print]
Alisa J Stephens-Shields, H Henry Lai, J Richard Landis, Karl Kreder, Larissa V Rodriguez, Bruce D Naliboff, Niloofar Afari, Siobhan Sutcliffe, Robert Moldwin, James W Griffith, J Quentin Clemens, Catherine S Bradley, Susan Quallich, Priyanka Gupta, Steven E Harte, John T Farrar
University of Pennsylvania, Philadelphia, Pennsylvania., Washington University, St. Louis, Missouri., University of Iowa, Iowa City, Iowa., Weill Cornell Medicine, New York, New York., University of California, Los Angeles, California., University of California, San Diego, California., Zucker School of Medicine at Hofstra-Northwell, Lake Success, New York., Northwestern University, Chicago, Illinois., University of Michigan, Ann Arbor, Michigan.