The American neurogastroenterology and motility society gastroparesis cardinal symptom index‐daily diary (ANMS GCSI‐DD): Psychometric evaluation in patients with idiopathic or diabetic gastroparesis

Abstract

Background 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. Methods 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. Key Results 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.

Randomised clinical trial: safety, pharmacokinetics and pharmacodynamics of trazpiroben (TAK‐906), a dopamine D2/D3 receptor antagonist, in patients with gastroparesis

“…Neurogastroenterology and Motility Society Gastroparesis Cardinal Symptom Index-Daily Diary scores in patients over the course of 8 weeks. 45,62,63 In conclusion, the results from this short-term, phase 2a, pilot study demonstrate that trazpiroben is well tolerated with a favourable safety profile, without any evidence of cardiovascular or central nervous system effects. Despite the absence of a significant effect on gastric emptying, trazpiroben 25 mg produced significant benefits in volume to fullness and demonstrated numerical improvements in symptom scores.…”

“…For treatments that are intended to be used in a chronic and/or continuous setting, longer treatment periods of at least 8 weeks would be preferable to evaluate changes in symptoms of gastroparesis and possibly the effect of trazpiroben on gastric emptying. This short treatment period (9 days) may also have impacted the outcomes of the American Neurogastroenterology and Motility Society Gastroparesis Cardinal Symptom Index‐Daily Diary symptom scoring tool, which recommends that symptoms are recorded over a 4‐ to 8‐week period, because treatment for gastroparesis is associated with a progressive decline in American Neurogastroenterology and Motility Society Gastroparesis Cardinal Symptom Index‐Daily Diary scores in patients over the course of 8 weeks 45,62,63 …”

“…The effect of trazpiroben on gastroparesis symptoms was assessed using the American Neurogastroenterology and Motility Society Gastroparesis Cardinal Symptom Index‐Daily Diary validated measure of gastroparesis symptoms, a patient‐reported outcome instrument, which assesses nausea, early satiety, postprandial fullness and upper abdominal pain on a severity score from none (0) to very severe (4), in addition to the number of vomiting episodes during the previous 24 hours. A composite score is generated from the first four symptoms (nausea, early satiety, postprandial fullness and upper abdominal pain), with the core symptom score including all five symptoms (the first four symptoms plus vomiting frequency) 45 . American Neurogastroenterology and Motility Society Gastroparesis Cardinal Symptom Index‐Daily Diaries were completed daily, beginning 7 days prior to day 1 and continuing throughout the trial until the end of trial visit.…”

“…A composite score is generated from the first four symptoms (nausea, early satiety, postprandial fullness and upper abdominal pain), with the core symptom score including all five symptoms (the first four symptoms plus vomiting frequency). 45…”

Role of Gastric Emptying in Symptoms of Gastroparesis

“…This questionnaire grades the severity of nausea, early satiety, postprandial fullness and upper abdominal pain on a 0 (none), 1 (mild), 2 (moderate), 3 (severe), 4 (very severe) scale, and also records the number of episodes of vomiting to assess these symptoms over the past 24-h [13]. The GCSI-DD has excellent reliability and validity in assessing symptoms in patients with either idiopathic or diabetic Gp [14][15][16]. In patients with diabetic Gp, the Diabetic Gastroparesis Symptom Severity Diary (DGSSD) has been shown to be a reliable and valid tool to assess Gp symptoms [17].…”