Mulhall John P, Montorsi Francesco
Department of Urology, Weill Medical College of Cornell University, New York Presbyterian Hospital & Memorial Sloan Kettering Cancer Center New York, NY 10021, USA.
Eur Urol. 2006 Jan;49(1):30-7. doi: 10.1016/j.eururo.2005.09.001. Epub 2005 Sep 22.
More treatment options are available now for the treatment of erectile dysfunction (ED) than ever. Treatments include oral phosphodiesterase 5 (PDE5) inhibitors, intracavernosal injections, vacuum constriction devices, and penile implants. Clinicians, researchers, and patients are interested in making direct comparisons between the response of newer treatments and that of established and more developed therapies. Of the currently available treatment options for ED, the most commonly prescribed therapies are oral PDE5 inhibitors, which include sildenafil citrate (Viagra, Pfizer Inc), tadalafil (Cialis, Lilly ICOS), and vardenafil (Levitra, Bayer). However, most patient preference studies of these drugs conducted to date have serious design flaws that hinder interpretation of the data, and thus limit the utility of the results. To make an informed decision on the most appropriate treatment option available, physicians and their patients require a thorough understanding of the methodology of these studies. Clinical comparison or preference trials must establish internal and external validity if the data are to be used in a generalized patient population. We review preference studies that compared sildenafil, tadalafil, and vardenafil, and highlight study designs that can introduce bias. We propose that, like safety and efficacy trials, randomized controlled trials (RCTs) should be the gold standard for evaluating patient preference treatments for ED. We do not wish to discourage individual investigators from performing preference studies, but rather to highlight the features of current preference trials to help patients and clinicians alike become aware of potential biases from independent or industry-sponsored patient preference trials so that they can interpret the results accordingly. Key components of patient preference RCTs are reviewed: period and carryover effects, preference assessments, eligibility criteria, and data analysis. We discuss why these components of patient-preference RCTs are important for evaluating the validity and relevance of patient preference studies. The preference studies discussed in this brief review are summarized in , and the methodological problems with each study are indicated. We provide a recommendation for the design of such trials that can minimize bias and provide better data for physicians and their patients.
如今，治疗勃起功能障碍（ED）的治疗选择比以往任何时候都更多。治疗方法包括口服磷酸二酯酶5（PDE5）抑制剂、海绵体内注射、真空收缩装置和阴茎植入物。临床医生、研究人员和患者都有兴趣对新治疗方法与已确立且更成熟疗法的反应进行直接比较。在目前可用的ED治疗选择中，最常用的疗法是口服PDE5抑制剂，其中包括枸橼酸西地那非（万艾可，辉瑞公司）、他达拉非（希爱力，礼来公司）和伐地那非（艾力达，拜耳公司）。然而，迄今为止对这些药物进行的大多数患者偏好研究都存在严重的设计缺陷，这阻碍了对数据的解读，从而限制了结果的实用性。为了就最合适的可用治疗选择做出明智的决定，医生及其患者需要全面了解这些研究的方法。如果要将数据应用于一般患者群体，临床比较或偏好试验必须确立内部和外部有效性。我们回顾了比较西地那非、他达拉非和伐地那非的偏好研究，并强调了可能引入偏差的研究设计。我们建议，与安全性和有效性试验一样，随机对照试验（RCT）应成为评估ED患者偏好治疗的金标准。我们并非要阻止个别研究人员进行偏好研究，而是要强调当前偏好试验的特点，以帮助患者和临床医生都意识到独立或行业赞助的患者偏好试验中潜在的偏差，以便他们能够相应地解读结果。本文回顾了患者偏好RCT的关键组成部分：周期和残留效应、偏好评估、纳入标准和数据分析。我们讨论了为什么患者偏好RCT的这些组成部分对于评估患者偏好研究的有效性和相关性很重要。本简要综述中讨论的偏好研究总结在[此处可能应有相关表格或内容但未给出]中，并指出了每项研究的方法学问题。我们为这类试验的设计提供了一项建议，该建议可以最大限度地减少偏差，并为医生及其患者提供更好的数据。
