Article

Early termination of a trial of mycophenolate mofetil for treatment of interstitial cystitis/painful bladder syndrome: lessons learned.

Department of Urology, University of Washington, Seattle, Washington 98195-0510, USA.
The Journal of urology (Impact Factor: 4.02). 03/2011; 185(3):901-6. DOI:10.1016/j.juro.2010.10.053
Source: PubMed

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.

0 0
 · 
0 Bookmarks
 · 
101 Views
  • [show abstract] [hide abstract]
    ABSTRACT: This review reflects the presentations and subsequent discussions at the International consultation on Incontinence Research Society's annual meeting. It updates the current definitions and diagnostic and treatment algorithms for bladder pain syndrome and chronic pelvic pain syndrome (non-bacterial prostatitis), highlights some specific basic research findings from discussion participants, looks at what we can hope to eventually learn from a large multicenter National Institutes of Health study, reviews future research pathways as articulated by the National Urologic Research Agenda of the American Urological Association and others, discusses recent therapeutic efforts, and concludes with discussion points from the ICI-RS meeting.
    Neurourology and Urodynamics 03/2012; 31(3):375-83. · 2.67 Impact Factor
  • [show abstract] [hide abstract]
    ABSTRACT: Cyclosporine A is a fifth-tier treatment option in the American Urological Association guidelines for interstitial cystitis/bladder pain syndrome. It was more effective than pentosanpolysulfate in a Finnish trial, but experience elsewhere is limited. Some centers use cyclosporine A off label for carefully selected patients but the number of patients in each center is small. We performed a retrospective review combining data from 3 tertiary centers that focus on interstitial cystitis/bladder pain syndrome. Charts were reviewed for patients with interstitial cystitis/bladder pain syndrome who received cyclosporine A. Response was defined as markedly improved on the 7-point global response assessment (2 centers) or as at least a 50% decrease in Interstitial Cystitis Symptom Index score (1 center). The study included 14 men and 30 women. Mean patient age was 55.5 years (range 27 to 75) and mean followup was 20.8 months (range 3 to 81). A total of 34 patients had Hunner lesions. Of these patients 29 (85%) responded but 6 eventually stopped cyclosporine A for adverse events, resulting in a success rate of 68% (23 of 34) for patients with Hunner lesions. In contrast, only 3 of 10 patients without Hunner lesions responded (30%). For all responders, the response occurred within 4 months. Cyclosporine A had a high success rate for patients with Hunner lesions in whom more conservative options, including endoscopic treatment, had failed. The success rate was low for patients without Hunner lesions. A 3 to 4-month trial is sufficient time to assess response. Adverse events were common and led to discontinuation of cyclosporine A for some patients. Close monitoring is needed, especially for blood pressure and renal function.
    The Journal of urology 08/2012; 188(4):1186-91. · 4.02 Impact Factor

Full-text (2 Sources)

View
6 Downloads
Available from
Oct 7, 2013