AstraZeneca, Gaithersburg, MD, USA.
IQVIA, Real World Solutions, Patient Centered Solutions, New York, NY, USA.
Orphanet J Rare Dis. 2021 Nov 24;16(1):495. doi: 10.1186/s13023-021-02107-6.
A patient reported outcome (PRO) instrument with evidence of validity and reliability for assessing symptoms of eosinophilic gastritis (EG) and eosinophilic gastroenteritis (EGE) is needed to measure treatment benefit in clinical trials. The aim of this research is to develop an EG/EGE symptom PRO instrument for patients aged 12 and above.
The Symptom Assessment for Gastrointestinal Eosinophilic Diseases (SAGED) was developed through a literature review, discussions with expert clinicians, and concept elicitation and cognitive debriefing interviews with patients. Patients (n = 28) were recruited based on confirmed diagnosis and self-reported symptoms. The final instrument was translated and linguistically validated with additional cognitive debriefing interviews (n = 105).
SAGED is a 24-h recall questionnaire consisting of eight items evaluating the core symptoms of EG and EGE (abdominal pain, nausea, bloating, early satiety, loss of appetite, vomiting, and diarrhea). Seven of the eight items are evaluated on an 11-point numerical rating scale ranging from 'none' to 'worst imaginable'. Cognitive debriefing interviews showed that adults and adolescents understand the content and are able to select a response that reflects their experience. The linguistic validation process produced 21 translations that are understandable to patients and conceptually equivalent to the source version.
SAGED is suitable for measuring symptom improvement in adult and adolescent patients with EG and/or EGE. The content validity of SAGED has been established through best practices in qualitative research for PRO instrument development. The psychometric properties of SAGED will be evaluated in a future study.
需要一种具有有效性和可靠性证据的患者报告结局（PRO）工具来评估嗜酸性胃炎（EG）和嗜酸性胃肠炎（EGE）的症状，以便在临床试验中衡量治疗效果。本研究旨在为 12 岁及以上的 EG/EGE 患者开发一种症状 PRO 工具。
通过文献回顾、与专家临床医生的讨论以及对患者的概念启发和认知访谈，开发了症状评估用于胃肠道嗜酸性疾病（SAGED）。根据确诊诊断和自我报告的症状招募患者（n=28）。最终工具经过翻译和语言验证，并进行了额外的认知访谈（n=105）。
SAGED 是一个 24 小时回忆问卷，包含 8 个项目，评估 EG 和 EGE 的核心症状（腹痛、恶心、腹胀、早饱、食欲不振、呕吐和腹泻）。八个项目中的七个项目使用 11 点数字评分量表进行评估，范围从“无”到“想象中最严重”。认知访谈显示，成年人和青少年理解内容，并能够选择反映他们体验的反应。语言验证过程产生了 21 种翻译，这些翻译对患者来说是可以理解的，并且与原始版本在概念上是等效的。
SAGED 适用于测量 EG 和/或 EGE 成年和青少年患者的症状改善。通过 PRO 工具开发的定性研究最佳实践，已经确立了 SAGED 的内容有效性。SAGED 的心理测量特性将在未来的研究中进行评估。
