Journal article
Towards precision medicine in clinical trials for the treatment of urologic chronic pelvic pain syndrome: lessons from the MAPP Research Network
Nature reviews. Urology, Vol.22(9), pp.632-642
09/2025
DOI: 10.1038/s41585-025-01030-w
PMID: 40307537
Abstract
Randomized clinical trials have resulted in few approved therapies for the treatment of interstitial cystitis/bladder pain syndrome and chronic prostatitis/chronic pelvic pain syndrome, collectively referred to as urologic chronic pelvic pain syndrome. Heterogenous patient populations, mismatches of treatments to patient phenotypes, non-specific outcomes and use of standard study designs not leveraging phenotypic heterogeneity might have contributed to the inability of previous trials to demonstrate existing efficacy. The Multidisciplinary Approach to the Study of Chronic Pelvic Pain Research Network has identified important phenotypic characteristics associated with differential symptom severity and treatment responsiveness. Based on Multidisciplinary Approach to the Study of Chronic Pelvic Pain findings and external research, empirically informed strategies were generated for defining patient populations, specifying treatments and selecting primary outcomes for future randomized clinical trials in urologic chronic pelvic pain syndrome. Explicitly specifying the scope of eligibility criteria across heterogeneous patient subgroups defined by pain widespreadness, the presence of Hunner lesions, the presence of pain with bladder filling or relieved by voiding, the extent of chronic overlapping pain conditions, and pelvic floor tenderness is needed. Therapies should be selected based on the mechanism of action and relevance to the mechanism of pain and dominant symptomology that the patient experiences. Evidence suggests that pain and urinary symptoms should be evaluated separately. Promising trial designs for identifying effective therapies in this heterogeneous patient population include sequential multiple assignment randomized trials and adaptive designs.
Details
- Title: Subtitle
- Towards precision medicine in clinical trials for the treatment of urologic chronic pelvic pain syndrome: lessons from the MAPP Research Network
- Creators
- Alisa J Stephens Shields - University of PennsylvaniaJ Quentin Clemens - University of MichiganMichel A Pontari - Temple UniversityH Henry Lai - Division of Urologic Surgery, Departments of Surgery and Anaesthesiology, Washington University, St. Louis, MO, USARobert Moldwin - Zucker School of Medicine at Hofstra-Northwell, Lake Success, NY, USADavid A Williams - University of MichiganCatherine S Bradley - Department of Obstetrics and Gynecology, University of Iowa, Iowa City, IA, USAJohn T Farrar - University of PennsylvaniaJ Richard Landis - University of PennsylvaniaChris Mullins - National Institute of Diabetes and Digestive and Kidney DiseasesBruce D Naliboff - University of California, Los AngelesSiobhan Sutcliffe - Division of Urologic Surgery, Departments of Surgery and Anaesthesiology, Washington University, St. Louis, MO, USAStephen J Walker - Forest InstituteClaire C Yang - University of WashingtonDaniel J Clauw - University of Michigan
- Resource Type
- Journal article
- Publication Details
- Nature reviews. Urology, Vol.22(9), pp.632-642
- DOI
- 10.1038/s41585-025-01030-w
- PMID
- 40307537
- NLM abbreviation
- Nat Rev Urol
- ISSN
- 1759-4820
- eISSN
- 1759-4820
- Publisher
- NATURE PORTFOLIO
- Grant note
- Multidisciplinary Approach to the Study of Chronic Pelvic Pain (MAPP) Research Network (Supplementary information 1) - National Institute of Diabetes and Digestive and Kidney Diseases: U01DK82315, U01DK82316, U01DK82325, U01DK82333, U01DK82342, U01DK82344, U01DK82345, U01DK82370, U01DK103227, U01DK103260, U01DK103271
This work was carried out on behalf of the Multidisciplinary Approach to the Study of Chronic Pelvic Pain (MAPP) Research Network (Supplementary information 1). The MAPP Research Network was funded by the National Institute of Diabetes and Digestive and Kidney Diseases (U01DK82315, U01DK82316, U01DK82325, U01DK82333, U01DK82342, U01DK82344, U01DK82345, U01DK82370, U01DK103227, U01DK103260, U01DK103271).
- Language
- English
- Electronic publication date
- 04/30/2025
- Date published
- 09/2025
- Academic Unit
- Epidemiology; Obstetrics and Gynecology; Urology
- Record Identifier
- 9984816009702771
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