Journal article
Early Termination of a Trial of Mycophenolate Mofetil for Treatment of Interstitial Cystitis/Painful Bladder Syndrome: Lessons Learned
The Journal of urology, Vol.185(3), pp.901-906
03/01/2011
DOI: 10.1016/j.juro.2010.10.053
PMCID: PMC3408599
PMID: 21238993
Abstract
We evaluated the efficacy and tolerability of mycophenolate mofetil in patients with treatment refractory interstitial cystitis/painful bladder syndrome.
A total of 210 patients with interstitial cystitis/painful bladder syndrome were to be randomized into a multicenter, placebo controlled trial using a 2:1 randomization. Participants in whom at least 3 interstitial cystitis/painful bladder syndrome specific treatments had failed and who had at least moderately severe symptoms were enrolled in a 12-week treatment study. The primary study end point was the global response assessment. Secondary end points were general and disease specific symptom questionnaires, and voiding diaries.
Only 58 subjects were randomized before a black box warning regarding mycophenolate mofetil safety was issued by the manufacturer in October 2007. The trial was halted, and interim analysis was performed and presented to an independent data and safety monitoring board. Six of the 39 subjects (15%) randomized at study cessation were considered responders for mycophenolate mofetil compared to 3 of 19 controls (16%, p = 0.67). Secondary outcome measures reflected more improvement in controls.
In a randomized, placebo controlled trial that was prematurely halted mycophenolate mofetil showed efficacy similar to that of placebo to treat symptoms of refractory interstitial cystitis/painful bladder syndrome. The results of this limited study cannot be used to confirm or refute the hypothesis that immunosuppressive therapy may be beneficial to at least a subgroup of patients with interstitial cystitis/painful bladder syndrome. Despite study termination lessons can be gleaned to inform future investigations.
Details
- Title: Subtitle
- Early Termination of a Trial of Mycophenolate Mofetil for Treatment of Interstitial Cystitis/Painful Bladder Syndrome: Lessons Learned
- Creators
- Claire C. Yang - University of WashingtonDavid A. Burks - Henry Ford HospitalKathleen J. Propert - University of PennsylvaniaRobert D. Mayer - University of RochesterKenneth M. Peters - Beaumont Hospital, TroyJ. Curtis Nickel - Queen's UniversityChristopher K. Payne - Stanford UniversityMary P. FitzGerald - Loyola University ChicagoPhilip M. Hanno - University of PennsylvaniaToby C. Chai - University of Maryland, BaltimoreKarl J. Kreder - University of IowaEmily S. Lukacz - University of California San DiegoHarris E. Foster - University of WashingtonLiyi Cen - University of PennsylvaniaJ. Richard Landis - University of PennsylvaniaJohn W. Kusek - National Institute of Diabetes and Digestive and Kidney DiseasesLeroy M. Nyberg - National Institute of Diabetes and Digestive and Kidney DiseasesInterstitial Cystitis Collaborative Research Network
- Resource Type
- Journal article
- Publication Details
- The Journal of urology, Vol.185(3), pp.901-906
- DOI
- 10.1016/j.juro.2010.10.053
- PMID
- 21238993
- PMCID
- PMC3408599
- NLM abbreviation
- J Urol
- ISSN
- 0022-5347
- eISSN
- 1527-3792
- Publisher
- Elsevier Inc
- Grant note
- U01 DK65209; U01DK65255; U01DK65213; U01DK65214; U01DK65215; U01DK65178; U01DK65190; U01DK65192; U01DK65255; U01DK65267; U01DK65271; U01DK65202 / National Institute of Diabetes and Digestive and Kidney Diseases Cooperative Agreements
- Language
- English
- Date published
- 03/01/2011
- Academic Unit
- Obstetrics and Gynecology; Urology
- Record Identifier
- 9984383292302771
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