Measuring the symptoms of pediatric constipation and irritable bowel syndrome with constipation: expert commentary and literature review.

BACKGROUND: Symptom measurement in pediatric chronic idiopathic constipation (CIC) and irritable bowel syndrome with constipation (IBS-C) trials requires appropriately developed clinical outcome assessments (COAs). METHODS: Literature was reviewed to identify symptom COAs meeting regulatory standards. Searches were conducted in Pubmed/Medline, EMBASE, and PsychINFO. Title/abstracts were reviewed to identify qualitative studies and those using COAs to measure pediatric CIC/IBS symptoms. Pediatric functional gastrointestinal experts provided input on relevant symptom-concepts to measure. RESULTS: Review of 1,105 abstracts identified 1 relevant qualitative article and 113 articles including COAs. Symptoms most frequently measured in CIC studies were frequency of bowel movements, fecal incontinence/encopresis, abdominal pain, stool consistency, and painful defecation. Symptoms most frequently measured in IBS were abdominal pain, abdominal distention/bloating, stool consistency, frequency of bowel movements, and gas. Evidence of development/validity of COAs was limited. Expert feedback was broadly consistent with the literature. CONCLUSION: Findings demonstrate consistency in the literature on key CIC/IBS symptoms to measure in pediatric trials, but existing COAs do not meet regulatory standards.

Pediatric restless legs syndrome: analysis of symptom descriptions and drawings.

The specific aims of this study were to collect and analyze detailed symptom descriptions from patients with pediatric restless legs syndrome, ages 6 to 17 years, as well as assess symptom impact and the usefulness of drawings. Trained qualitative interviewers conducted face-to-face audio-recorded interviews of children and adolescents who met criteria for definite restless legs syndrome. Thirty-three patients in 3 age groups used 16 different categories of descriptors for restless legs sensations, with a mean of 3 or more categories used per patient in each age group. "Need to move/kick," "pain/hurts," "uncomfortable/cannot get comfortable," and "like bugs or ants/crawling" were the most common descriptors. Two-thirds reported daytime sensations, and nearly half had arm involvement. They described impact on sleep, cognitive function, and affect. Drawings provided useful diagnostic information. These detailed empirical data will be useful in clinical practice, as well as in the development of formal diagnostic tools and severity measures.

Psychometric evaluation of the Diabetes Symptom Checklist-Revised (DSC-R)--a measure of symptom distress.

OBJECTIVE: To assess the psychometric validity, reliability, responsiveness, and minimal important differences of the Diabetes Symptoms Checklist-Revised (DSC-R), a widely used patient-reported outcome measure of diabetes symptom distress. RESEARCH DESIGN AND METHODS: Psychometric validity of the DSC-R was assessed using blinded data from a large-scale trial of approximately 4000 type 2 diabetes patients. Confirmatory factorial analysis (CFA) and multitrait analysis were used to examine the construct validity of the structure of DSC-R. DSC-R internal consistency, discriminative validity, and responsiveness were also assessed. Distribution and anchor-based methods were used to estimate minimal important differences for DSC-R domains. RESULTS: Mean age of the sample was 56 years, 42% were female, 88% were Caucasian. Patients had a mean body mass index (BMI) of 32.2 and mean glucose-fasting level of 151.7 md/dl. CFA and multitrait analysis indicated that the scoring of the DSC-R has acceptable construct validity. Item-scale correlations ranged from 0.44 to 0.78. Cronbach's alpha coefficients ranged from 0.69 to 0.87. At baseline, DSC-R scores were higher among patients with higher BMI scores (P < 0.0001), supporting the discriminative validity of the DSC-R. Minimal important difference estimates ranged from 0.39 to 0.60 points when using distribution methods and from 0.00 to 0.33 when estimated using anchor-based methods. CONCLUSIONS: The DSC-R demonstrated excellent psychometric properties when tested in a large-scale diabetes clinical trial. Responsiveness and test-retest reliability of the DSC-R warrant further evaluation.