We compared intravesical bacillus Calmette-Guerin (BCG) to placebo instillations in patients with treatment refractory interstitial cystitis (IC).
Subjects who met the National Institutes of Health-National Institute for Diabetes and Digestive and Kidney Diseases criteria for IC, and reported at least moderate pain and frequency for a minimum of 6 months before study entry, were randomized to 6 weekly double-blinded intravesical instillations of either BCG or placebo, and then followed for a total of 34 weeks. The primary outcome was a patient reported global response assessment at week 34, supplemented with medications for IC during weeks 31 to 34. Secondary outcomes included a 24-hour voiding diary, pain, urgency, validated IC symptom indexes and adverse events. The target sample size was 260 participants, designed to detect a difference in response rates between placebo and BCG of 30% and 50%, respectively.
A total of 265 participants were randomized and 17 (6%) patients withdrew from study. The response rates for the primary outcome were 12% for placebo and 21% for BCG (p = 0.062). Small improvements were observed for all secondary outcomes, some more so with BCG, but these differences were of borderline statistical significance. Although a large number of adverse events were reported in the BCG arm, there was no statistically significant difference between the treatment arms in overall adverse event rates.
Although the BCG safety profile was acceptable, the response rate for the primary outcome was low. Effective medical treatment for patients with moderate to severe interstitial cystitis remains elusive.
"Une étude récente, conduite par l'ICCTG, randomisée prospective en double insu versus placebo a été réalisé chez 265 patients sans montrer de différence significative . La réponse globale a été de 21 % dans le groupe BCG versus 12 % dans le groupe placebo avec un recul de 34 semaines (p = 0,062) . La série a été réactualisée chez les patients initialement répondeurs (12 dans le groupe placebo et 19 dans le groupe BCG) avec un suivi plus long de 68 semaines. "
[Show abstract][Hide abstract] ABSTRACT: IntroductionPainful bladder syndrome is defined as chronic pelvic pain present for more than 6 months, causing discomfort perceived as being related to the bladder and accompanied by a persistent and strong urge to urinate or urinary frequency. The purpose of this article is to review the treatment of painful bladder syndrome.Material and methodsA comprehensive review of the literature was performed by searching PUBMED for articles on specific treatments for painful bladder syndrome.ResultsMany treatments have been proposed for the management of painful bladder syndrome: local intravesical treatments (glucosaminoglycan [pentosan polysulfate], dimethylsulfoxide [DMSO], heparin, bacillus Calmette-Guérin [BCG], anticholinergic agents [oxybutynin, etc.] or oral treatments [glucosaminoglycan (pentosan polysulfate), antihistamines, antidepressants, immunosuppressives, etc.]) with an action on the pathophysiology of this syndrome. The efficacy of these various treatments has been limited, with trials based on small numbers of patients and not always conducted according to a randomized, prospective design. Other salvage treatments (neuromodulation, botulinum toxin, surgery, etc.) have also been reported with limited efficacy, but allowing salvage of treatment failures.Conclusion
The therapeutic management of painful bladder syndrome is complex. The large number of proposed treatment modalities present a limited efficacy with discordant results from one study to another making comparisons and analyses difficult.
Progrès en Urologie 11/2010; 20(12):1044-1053. DOI:10.1016/j.purol.2010.08.045 · 0.66 Impact Factor
"This difference did not achieve the prespecified threshold for statistical significance (p=0.06). Longitudinal analyses of the two secondary outcomes pain and urgency to void showed that the interaction between treatment assignment and time was not significant for either outcome, giving further evidence that treatment was not effective (Mayer et al., 2005). The goal of our analysis was to use the multivariate symptom profiles to classify patients in hopes of identifying a subset of true treatment responders. "
[Show abstract][Hide abstract] ABSTRACT: In this paper, we propose a multivariate growth curve mixture model that groups subjects based on multiple symptoms measured repeatedly over time. Our model synthesizes features of two models. First, we follow Roy and Lin (2000) in relating the multiple symptoms at each time point to a single latent variable. Second, we use the growth mixture model of Muthén and Shedden (1999) to group subjects based on distinctive longitudinal profiles of this latent variable. The mean growth curve for the latent variable in each class defines that class's features. For example, a class of "responders" would have a decline in the latent symptom summary variable over time. A Bayesian approach to estimation is employed where the methods of Elliott et al (2005) are extended to simultaneously estimate the posterior distributions of the parameters from the latent variable and growth curve mixture portions of the model. We apply our model to data from a randomized clinical trial evaluating the efficacy of Bacillus Calmette-Guerin (BCG) in treating symptoms of Interstitial Cystitis. In contrast to conventional approaches using a single subjective Global Response Assessment, we use the multivariate symptom data to identify a class of subjects where treatment demonstrates effectiveness. Simulations are used to confirm identifiability results and evaluate the performance of our algorithm. The definitive version of this paper is available at onlinelibrary.wiley.com.
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