Development of a Symptom-Based Patient-Reported Outcome Instrument for Functional Dyspepsia: A Preliminary Conceptual Model and an Evaluation of the Adequacy of Existing Instruments.
Development of a Symptom-Based Patient-Reported Outcome Instrument for Functional Dyspepsia: A Preliminary Conceptual Model and an Evaluation of the Adequacy of Existing Instruments.
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DOI：
10.1007/s40271-016-0164-1
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发表时间：
2016-10
影响因子：
3.6
通讯作者：
Silberg, Debra G.
中科院分区：
文献类型：
--
作者：
Taylor, Fiona;Reasner, David S.;Carson, Robyn T.;Deal, Linda S.;Foley, Catherine;Iovin, Ramon;Lundy, J. Jason;Pompilus, Farrah;Shields, Alan L.;Silberg, Debra G.
其目的是从经验性文献的角度记录功能性消化不良(FD)的主要症状，评估现有问卷针对这些症状的程度，并最终确定任何可能影响问卷在规范临床试验中的使用的缺失证据，以评估用于产品标签的治疗效果声明。通过文献回顾来确定FD的主要症状和现有的基于症状的FD患者报告结果(PRO)工具。在数据库搜索之后，对摘要进行筛选，检索文章以供审查。FD的主要症状被组织到一个概念模型中，评估PRO工具的概念性覆盖范围，并与FDA的PRO行业指南中提出的证据要求进行比较。对56篇文献和16种评估FD症状的工具进行综述。在FD概念模型中总结了罗马III FD标准中列出的概念(n=7)、由现有FD仪器评估的概念(n=34)、以及已发表的定性研究中患者报告的症状(n=)。除了呕吐，所有从已发表的定性研究报告中确定的症状也都在罗马III标准中具体说明。在16种工具中，只有3种，消化不良症状严重程度指数(DSSI)、Nepean消化不良指数(NDI)和简式Nepean消化不良指数(SF-NDI)测量了罗马III标准定义的所有7种FD症状。在这三个人中，每个人都使用了2周的回忆期和5分Likert类型的量表，并有证据表明患者参与了发育。尽管这些工具的覆盖面很广，但在根据监管机构的期望对这些工具进行评估时，有几个问题危及它们对标签索赔进行证实的潜在资格。现有的测量所有七种症状的PRO仪器都没有遵守支持产品标签所必需的监管原则。因此，支持开发一种新的FD症状PRO仪器。
The aim was to document, from the perspective of the empirical literature, the primary symptoms of functional dyspepsia (FD), evaluate the extent to which existing questionnaires target those symptoms, and, finally, identify any missing evidence that would impact the questionnaires’ use in regulated clinical trials to assess treatment efficacy claims intended for product labeling. A literature review was conducted to identify the primary symptoms of FD and existing symptom-based FD patient-reported outcome (PRO) instruments. Following a database search, abstracts were screened and articles were retrieved for review. The primary symptoms of FD were organized into a conceptual model and the PRO instruments were evaluated for conceptual coverage as well as compared against evidentiary requirements presented in the FDA’s PRO Guidance for Industry. Fifty-six articles and 16 instruments assessing FD symptoms were reviewed. Concepts listed in the Rome III criteria for FD (n = 7), those assessed by existing FD instruments (n = 34), and symptoms reported by patients in published qualitative research (n = 6) were summarized in the FD conceptual model. Except for vomiting, all of the identified symptoms from the published qualitative research reports were also specified in the Rome III criteria. Only three of the 16 instruments, the Dyspepsia Symptom Severity Index (DSSI), Nepean Dyspepsia Index (NDI), and Short-Form Nepean Dyspepsia Index (SF-NDI), measure all seven FD symptoms defined by the Rome III criteria. Among these three, each utilizes a 2-week recall period and 5-point Likert-type scale, and had evidence of patient involvement in development. Despite their coverage, when these instruments were evaluated in light of regulatory expectations, several issues jeopardized their potential qualification for substantiation of a labeling claim. No existing PRO instruments that measured all seven symptoms adhered to the regulatory principles necessary to support product labeling. As such, the development of a new FD symptom PRO instrument is supported.
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影响因子：
3.6
作者：
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通讯作者：
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DOI：
10.1097/00042737-199610000-00006
发表时间：
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