PDF(Pubmed)
Abstract：
BACKGROUND: Wilson disease (WD) is a rare metabolic disorder of impaired copper transport manifesting in hepatic, neurological, and psychiatric symptoms. To evaluate the clinical symptoms of WD in clinical trials, a group of clinicians created the Unified Wilson Disease Rating Scale (UWDRS). Content validity of this scale has not been established. The aim of this study was to evaluate the content validity of the UWDRS Part II from the patient perspective.
METHODS: This study utilized multiple qualitative research methods including concept elicitation interviews, concept/instrument mapping, and cognitive debriefing interviews.
RESULTS: Concept elicitation interviews with a sample of patients with WD and one or more neurological signs/symptoms identified several signs, symptoms, and impacts related to neurological dysfunction, strengthening our understanding of the importance of the neurological aspects of the WD patient experience. Mapping neurological concepts to Part II and III items of the UWDRS showed complete coverage of all salient neurological concepts and near complete coverage of all neurological concepts reported by patients in concept elicitation interviews. Item debriefing of Part II of the UWDRS revealed that patients generally found the items clear and personally relevant to their experience with WD.
CONCLUSIONS: Overall, the findings from this study provide evidence for the content validity of the UWDRS Part II and supportive evidence for the content validity of Part III. The UWDRS should be used in conjunction with additional clinical outcomes assessments, specifically those evaluating the hepatic and psychiatric signs/symptoms of WD, to provide a comprehensive evaluation of the WD patient experience.
METHODS: This study utilized multiple qualitative research methods including concept elicitation interviews, concept/instrument mapping, and cognitive debriefing interviews.
RESULTS: Concept elicitation interviews with a sample of patients with WD and one or more neurological signs/symptoms identified several signs, symptoms, and impacts related to neurological dysfunction, strengthening our understanding of the importance of the neurological aspects of the WD patient experience. Mapping neurological concepts to Part II and III items of the UWDRS showed complete coverage of all salient neurological concepts and near complete coverage of all neurological concepts reported by patients in concept elicitation interviews. Item debriefing of Part II of the UWDRS revealed that patients generally found the items clear and personally relevant to their experience with WD.
CONCLUSIONS: Overall, the findings from this study provide evidence for the content validity of the UWDRS Part II and supportive evidence for the content validity of Part III. The UWDRS should be used in conjunction with additional clinical outcomes assessments, specifically those evaluating the hepatic and psychiatric signs/symptoms of WD, to provide a comprehensive evaluation of the WD patient experience.
摘要：
背景：威尔逊病（WD）是一种罕见的代谢紊乱，表现为肝铜转运受损，神经学,和精神症状。在临床试验中评估WD的临床症状，一组临床医生创建了统一的威尔逊病评定量表（UWDRS）。该量表的内容效度尚未确定。这项研究的目的是从患者的角度评估UWDRS第二部分的内容有效性。
方法：本研究采用了多种定性研究方法，包括概念启发访谈，概念/仪器映射，和认知汇报访谈。
结果：对WD患者样本的概念启发访谈以及一种或多种神经系统体征/症状确定了几种体征，症状,以及与神经功能障碍有关的影响，加强我们对WD患者体验的神经系统方面的重要性的理解。将神经系统概念映射到UWDRS的第II部分和第III部分项目显示，患者在概念启发访谈中报告的所有神经系统概念都已完全覆盖，并且几乎完全覆盖了所有神经系统概念。UWDRS第二部分的项目汇报显示，患者通常发现这些项目清晰且与他们的WD经验有关。
结论：总体而言，这项研究的结果为UWDRS第二部分的内容有效性提供了证据，并为第三部分的内容有效性提供了支持性证据。UWDRS应与其他临床结果评估结合使用，特别是那些评估WD的肝脏和精神体征/症状的人，提供对WD患者体验的全面评估。
方法：本研究采用了多种定性研究方法，包括概念启发访谈，概念/仪器映射，和认知汇报访谈。
结果：对WD患者样本的概念启发访谈以及一种或多种神经系统体征/症状确定了几种体征，症状,以及与神经功能障碍有关的影响，加强我们对WD患者体验的神经系统方面的重要性的理解。将神经系统概念映射到UWDRS的第II部分和第III部分项目显示，患者在概念启发访谈中报告的所有神经系统概念都已完全覆盖，并且几乎完全覆盖了所有神经系统概念。UWDRS第二部分的项目汇报显示，患者通常发现这些项目清晰且与他们的WD经验有关。
结论：总体而言，这项研究的结果为UWDRS第二部分的内容有效性提供了证据，并为第三部分的内容有效性提供了支持性证据。UWDRS应与其他临床结果评估结合使用，特别是那些评估WD的肝脏和精神体征/症状的人，提供对WD患者体验的全面评估。
