Early termination of a trial of mycophenolate mofetil for treatment of interstitial cystitis/painful bladder syndrome: lessons learned.
Early termination of a trial of mycophenolate mofetil for treatment of interstitial cystitis/painful bladder syndrome: lessons learned.
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DOI：
10.1016/j.juro.2010.10.053
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发表时间：
2011-03
期刊：
影响因子：
--
通讯作者：
Interstitial Cystitis Collaborative Research Network
中科院分区：
文献类型：
--
作者：
Yang CC;Burks DA;Propert KJ;Mayer RD;Peters KM;Nickel JC;Payne CK;FitzGerald MP;Hanno PM;Chai TC;Kreder KJ;Lukacz ES;Foster HE;Cen L;Landis JR;Kusek JW;Nyberg LM;Interstitial Cystitis Collaborative Research Network
评价霉酚酸酯（MMF）治疗难治性间质性膀胱炎/膀胱痛综合征（IC/PBS）患者的疗效和耐受性。210例IC/PBS患者按2:1随机分组，随机分为多中心、安慰剂对照试验。至少三次IC/ pbs特异性治疗失败且至少有中度严重症状的参与者被纳入为期12周的治疗研究。主要研究终点是总体反应评估（GRA）。次要终点包括一般和疾病特异性症状问卷调查和排尿日记。在制造商于2007年10月发布关于MMF安全性的黑框警告之前，只有58名受试者被随机分配。试验被暂停，并进行了中期分析，并提交给独立的数据和安全监测委员会。在研究停止时随机分配的受试者中，6/39（15%）被认为对MMF有反应，而对照组为3/19 (16%)（p=0.67）。次要结果指标在对照组中反映出更多的改善。在一项提前停止的随机安慰剂对照试验中，MMF在治疗难治性IC/PBS症状方面显示出与安慰剂相似的疗效。这项有限的研究结果不能用来证实或反驳免疫抑制治疗可能对至少一个亚组的IC\PBS患者有益的假设。尽管研究终止了，但仍有可以收集到的教训为未来的调查提供信息。
To evaluate the efficacy and tolerability of mycophenolate mofetil (MMF) in patients with treatment refractory interstitial cystitis/painful bladder syndrome (IC/PBS). Two hundred and ten patients with IC/PBS were to be randomized into a multicenter, placebo-controlled trial using a 2:1 randomization. Participants who had failed at least three IC/PBS-specific treatments and had at least moderately severe symptoms were enrolled in a 12-week treatment study. The primary study endpoint was the Global Response Assessment (GRA). Secondary endpoints included general and disease-specific symptom questionnaires and voiding diaries. Only 58 subjects were randomized before a black box warning regarding MMF safety was issued by the manufacturer in October 2007. The trial was halted, and an interim analysis was performed and presented to an independent Data and Safety Monitoring Board. Of the subjects randomized at the time of study cessation, 6/39 (15%) were considered responders for MMF, compared to 3/19 (16%) of controls (p=0.67). Secondary outcome measures reflected more improvement among controls. In a randomized placebo-controlled trial that was prematurely halted, MMF demonstrated efficacy similar to placebo in treating symptoms of refractory IC/PBS. 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 IC\PBS patients. Despite study termination, there are lessons that can be gleaned to inform future investigations.
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影响因子：
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作者：
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通讯作者：
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影响因子：
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