Evaluating symptom outcomes in gastroparesis clinical trials: validity and responsiveness of the Gastroparesis Cardinal Symptom Index-Daily Diary (GCSI-DD)
Evaluating symptom outcomes in gastroparesis clinical trials: validity and responsiveness of the Gastroparesis Cardinal Symptom Index-Daily Diary (GCSI-DD)
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DOI：
10.1111/j.1365-2982.2012.01879.x
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发表时间：
2012-05-01
影响因子：
3.5
通讯作者：
Parkman, H. P.
中科院分区：
文献类型：
--
作者：
Revicki, D. A.;Camilleri, M.;Parkman, H. P.
背景：需要患者报告的症状量表来评估胃轻瘫的治疗。胃轻瘫主要症状指数-每日日记（GCSI-DD）用于评估胃轻瘫的每日症状。本研究评价了GCSI-DD在胃轻瘫患者中的有效性和反应性。方法对有症状的胃轻瘫患者采用新的治疗方法。患者在基线周和治疗8周期间每晚完成GCSI-DD。根据患者和临床医生对变化的总体评级定义应答者。根据基线至4周症状评分的变化估计最小重要差异（MID），以获得微小改善。关键结果在69名参与研究的患者中，46名患有特发性胃轻瘫，19名患有糖尿病，4名患有胃底折叠术后胃轻瘫。GCSI-DD评分具有良好的重测信度，并且GCSI-DD评分与临床医生对症状严重程度的评分之间存在显著相关性。治疗应答者报告恶心改善[效应量（ES）= 0.42，P <0.001]，餐后饱胀，ES = 0.83，P <0.001），腹胀早饱感（ES = 0.53，P <0.001），但上腹痛（ES = 0.29）和呕吐（ES = 0.22; P = 0.119）的反应较低。恶心的MID为0.55，过度饱胀为0.97，腹胀为0.63，餐后饱胀为0.77，腹痛为0.30。四种症状的综合评分（综合评分-1;恶心、腹胀、过度饱胀、餐后饱胀）的ES为0.61，MID为0.73。复合2评分（恶心、早饱、腹胀、腹痛）的ES较低，为0.47。结论和推论早饱、恶心、餐后饱胀和腹胀症状对胃轻瘫的治疗有反应。这些症状的复合也证明了胃轻瘫治疗的有效性和反应性，并且可能代表了在胃轻瘫临床试验中评价药物治疗有效性的可接受终点。
Background Patient-reported symptom scales are needed to evaluate treatments for gastroparesis. The Gastroparesis Cardinal Symptom Index-Daily Diary (GCSI-DD) was developed to assess daily symptoms of gastroparesis. This study evaluated the validity and responsiveness of the GCSI-DD in patients with gastroparesis. Methods Symptomatic patients were started with a new treatment for gastroparesis. Patients completed the GCSI-DD each evening during a baseline week and for 8 weeks of treatment. Responders were defined based on patient and clinician global rating of change. Minimal important differences (MID) were estimated based on baseline to 4 week changes in symptoms scores for small improvements. Key Results Of 69 patients participating, 46 had idiopathic, 19 diabetic, and four postfundoplication gastroparesis. Excellent test-retest reliability was seen for GCSI-DD scores, and there were significant correlations between GCSI-DD scores and clinician ratings of symptom severity. Responders to treatment reported improvements in nausea [effect size (ES) = 0.42, P < 0.001], postprandial fullness, ES = 0.83, P < 0.001), bloating (ES = 0.34, P < 0.001), early satiety (ES = 0.53, P < 0.001), but lower responses for upper abdominal pain (ES = 0.29), and vomiting (ES = 0.22; P = 0.119). MIDs were 0.55 for nausea, 0.97 for excessive fullness, 0.63 for bloating, 0.77 for postprandial fullness, and 0.30 for abdominal pain. A composite score of four symptoms (Composite-1; nausea, bloating, excessive fullness, postprandial fullness) had ES of 0.61 and MID of 0.73. Composite-2 score (nausea, early satiety, bloating, abdominal pain) had a lower ES of 0.47. Conclusions & Inferences Symptoms of early satiety, nausea, postprandial fullness, and bloating were responsive to treatment for gastroparesis. A composite of these symptoms also demonstrates validity and responsiveness to treatment for gastroparesis, and may represent an acceptable endpoint for evaluating the effectiveness of medical treatments in clinical trials for gastroparesis.