Evidera, Bethesda, Maryland.
Takeda Pharmaceuticals, London, UK.
Neurogastroenterol Motil. 2019 Apr;31(4):e13553. doi: 10.1111/nmo.13553. Epub 2019 Feb 7.
The purpose of this study was to evaluate the measurement properties of the American Neurogastroenterology and Motility Society Gastroparesis Cardinal Symptom Index-Daily Diary, a patient-reported outcome instrument developed to meet US FDA recommendations for a symptom-based clinical trial endpoint in gastroparesis. The ANMS GCSI-DD assesses nausea, early satiety, postprandial fullness, and upper abdominal pain on a severity score from none (0) to very severe (4) and number of vomiting episodes during the past 24 hours. The composite score includes the first four symptoms, the core symptom score includes all five symptoms.
Seventy-one patients diagnosed with idiopathic or diabetic gastroparesis were recruited for a four-week observational study. Patients completed the ANMS GCSI-DD at home between Baseline and Week 4. Statistical analyses included confirmatory factor analysis, item response theory analysis, internal consistency, test-retest reliability, and construct and known-groups validity.
Unidimensionality for the composite and core symptom scores was supported, and items exhibited good fit. Internal consistency (Cronbach's alpha =0.85 and 0.83) and test-retest reliability were 0.89 and 0.88, for composite and core symptom scores, respectively. Convergent validity was supported by strong correlations with patient-reported and clinician measures. Baseline and Week 4 scores differed for three measures used to define disease severity status (P < 0.0001), supporting known-groups validity.
CONCLUSIONS & INFERENCES: The ANMS GCSI-DD has excellent reliability and validity, supporting its use to assess symptom-based endpoints in gastroparesis clinical studies. Further analyses will be conducted using clinical trial data to ascertain treatment responsiveness and define a responder.
本研究旨在评估美国神经胃肠病学和动力学会胃轻瘫 cardinal 症状指数-日常日记的测量特性，这是一种患者报告的结局工具，旨在满足美国 FDA 的建议，即胃轻瘫基于症状的临床试验终点。ANMS GCSI-DD 评估严重程度（从无[0]到非常严重[4]）和过去 24 小时内呕吐发作次数的恶心、早饱、餐后饱胀和上腹痛。综合评分包括前四个症状，核心症状评分包括所有五个症状。
71 名被诊断为特发性或糖尿病性胃轻瘫的患者被招募参加为期四周的观察性研究。患者在基线和第 4 周在家中完成 ANMS GCSI-DD。统计分析包括验证性因子分析、项目反应理论分析、内部一致性、测试-重测信度以及结构和已知组有效性。
复合和核心症状评分的单维性得到支持，项目表现出良好的拟合度。内部一致性（Cronbach's alpha=0.85 和 0.83）和测试-重测信度分别为 0.89 和 0.88，用于复合和核心症状评分。与患者报告和临床医生测量的强相关性支持了收敛有效性。用于定义疾病严重程度的三种测量方法的基线和第 4 周评分存在差异（P<0.0001），支持已知组有效性。
ANMS GCSI-DD 具有极好的可靠性和有效性，支持其在胃轻瘫临床研究中评估基于症状的终点。将使用临床试验数据进一步分析以确定治疗反应和定义反应者。
