A pilot clinical trial of oral pentosan polysulfate and oral hydroxyzine in patients with interstitial cystitis
A pilot clinical trial of oral pentosan polysulfate and oral hydroxyzine in patients with interstitial cystitis
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DOI：
10.1097/01.ju.0000083020.06212.3d
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发表时间：
2003-09-01
影响因子：
6.6
通讯作者：
Nyberg, LM
中科院分区：
文献类型：
--
作者：
Sant, GR;Propert, KJ;Nyberg, LM
目的：这项先导性研究旨在评估间质性膀胱炎(IC)多中心随机临床试验的可行性。第二个目标是评价口服多聚戊聚糖硫酸钠(PPS)、羟基锌及其组合的安全性和有效性，以考虑它们在更大规模的随机临床试验中的应用。材料和方法：采用2×2析因试验设计评价PPS和羟基锌。参与者符合国家卫生研究院-国家糖尿病、消化和肾脏疾病研究所的IC标准，并在进入研究前至少6个月报告至少中度疼痛和频率。主要终点是患者报告的全球反应评估。次要终点包括有效的症状指数和患者关于疼痛、紧迫感和频率的报告。目标样本量是在10个月内招募的136名参与者。结果：总共121名参与者(占目标的89%)在18个月内被随机抽样，其中79%提供了完整的随访数据。羟基锌治疗有效率为31%，未治疗有效率为20%(p=0.26)。PPS治疗组(34%)与无PPS组(18%，p=0.064)相比无明显趋势。对于任何次要终点，都没有治疗差异。结论：PPS和羟基锌的总体应答率较低，表明这两种药物均不能为大多数IC患者提供益处。这项试验证明了在IC中使用统一的程序和结果进行多中心随机临床试验的可行性。然而，缓慢的招募突显了评估常见IC药物的困难。
Purpose: This pilot study was designed to evaluate the feasibility of a multicenter, randomized, clinical trial in interstitial cystitis (IC). Secondary objectives were to evaluate the safety and efficacy of oral pentosan polysulfate sodium (PPS), hydroxyzine, and the combination to consider their use in a larger randomized clinical trial.Materials and Methods: A 2 X 2 factorial study design was used to evaluate PPS and hydroxyzine. Participants 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. The primary end point was a patient reported global response assessment. Secondary end points included validated symptom indexes and patient reports of pain, urgency and frequency. The target sample size was 136 participants recruited during 10 months.Results: A total of 121 (89% of goal) participants were randomized over 18 months and 79% provided complete followup data. The response rate for hydroxyzine was 31% for those treated and 20% for those not treated (p = 0.26). A nonsignificant trend was seen in the PPS treatment groups (34%) as compared to no PPS (18%, p = 0.064). There were no treatment differences for any of the secondary end points. Adverse events were mostly minor and similar to those in previous reports.Conclusions: The low global response rates for PPS and hydroxyzine suggest that neither provided benefit for the-majority of patients with IC. This trial demonstrated the feasibility of conducting a multicenter randomized clinical trial in IC using uniform procedures and outcomes. However, slow recruitment underscored the difficulties of evaluating commonly available IC drugs.