Testing and Psychometric Validation of a Pediatric Instrument to Self-Assess Symptoms of the Common Cold.

OBJECTIVES: Given the lack of validated patient-reported outcomes (PRO) instruments assessing cold symptoms, a new pediatric PRO instrument was developed to assess multiple cold symptoms: the Child Cold Symptom Questionnaire (CCSQ). The objective of this research was to evaluate the measurement properties of the CCSQ. METHODS: This observational study involved daily completion of the self-report CCSQ by children aged 6-11 years in their home for 7 days. These data were used to develop a scoring algorithm and item-scale structure and evaluate the psychometric properties of the resulting scores. Analyses included evaluation of item and dimensionality performance (item response distributions and confirmatory factor analysis) and assessment of test-retest reliability in stable patients, construct validity (convergent and known groups validity), and preliminary responsiveness. Qualitative exit interviews in a subgroup of the children with colds and their parents were conducted. RESULTS: More than 90% of children had no missing data during the testing period, reflecting an excellent completion rate. For most items, responses were distributed across the options, with approximately normal distributions. Test-retest reliability was adequate, with intra-class correlation coefficients ranging from 0.63 to 0.83. A logical pattern of correlations with the validated Strep-PRO instrument provided evidence supporting convergent validity. Single- and multi-item symptom scores distinguished between children who differed in their cold severity based on global ratings, providing evidence of known groups validity. Preliminary evidence indicates the CCSQ is responsive to changes over time. CONCLUSIONS: The findings demonstrate that the CCSQ items and multi-item scores provide valid and reliable patient-reported measures of cold symptoms in children aged 6-11 years. They provide strong evidence supporting the validity of these items and multi-item scores for inclusion as endpoints in clinical trials to evaluate the efficacy of cold medicines.

Development and Content Validity Testing of Patient-Reported Outcome Items for Children to Self-Assess Symptoms of the Common Cold.

BACKGROUND AND OBJECTIVE: No pediatric patient-reported outcome instruments specific to the common cold are found in the literature. This study involved development and content validity testing of patient-reported outcome items (questions and response options) assessing cold symptoms in children aged 6-11 years. METHODS: Draft patient-reported outcome instructions, items, response scales, and recall periods were developed based on the literature and existing measures. Qualitative interviews were conducted with children (n = 39) who were currently (n = 31) or had recently (n = 8) experienced a cold and ten parents of a subset of children aged 6-8 years. The interviews were conducted over two rounds and included open-ended concept elicitation questioning, a free-drawing task, a card sorting task, and a task involving circling parts of the body, followed by cognitive debriefing of draft items. Thematic analysis of verbatim transcripts was performed to analyze the qualitative data. The findings were used to support revisions to the draft patient-reported outcome. RESULTS: Ten symptom concepts were reported by the children during concept elicitation. The creative tasks helped the children to describe their symptoms, generally using consistent language to do so, irrespective of age. Nineteen patient-reported outcome items were developed and subject to cognitive debriefing. Debriefing with both children and parents informed several small revisions and provided evidence that the majority of children found most patient-reported outcome items easy to understand, and that the items were mainly interpreted consistently and as intended. CONCLUSIONS: This in-depth qualitative study has supported identification of relevant symptom concepts and the development and refinement of patient-reported outcome items to assess those concepts. The findings support the content validity of the items and suggest that they can be used with confidence in children aged 9 years and older. For children aged 6-8 years, it is recommended the items are administered with initial adult supervision to explain the more difficult concepts or through parent/interviewer administration.

Multisite controlled study of OROS methylphenidate in the treatment of adolescents with attention-deficit/hyperactivity disorder.

BACKGROUND: Despite the persistence of attention-deficit/hyperactivity disorder (ADHD) into adolescence, little is known about the efficacy and tolerability of stimulant medications in this age group. OBJECTIVE: To report the results of a multisite controlled study among adolescents with ADHD evaluating the efficacy and tolerability of osmotic-release oral system (OROS) methylphenidate. DESIGN: Adolescents (N = 220) having a confirmed Diagnostic and Statistical Manual of Mental Disorders, Fourth Edition diagnosis of ADHD underwent dose titration to identify dosages of OROS methylphenidate that improved symptoms to predefined criteria. Subjects successfully completing the dose titration phase (n = 177) (ie, tolerated and responded to treatment and adhered to the protocol) were randomized to receive 2 weeks' treatment with their individualized dosage of OROS methylphenidate (18, 36, 54, or 72 mg once daily) or placebo. Treatment effectiveness was measured using investigator, parent, and adolescent assessments of ADHD. RESULTS: A significant reduction from baseline in the investigator-rated ADHD Rating Scale, the primary efficacy measure, was found with OROS methylphenidate treatment compared with placebo. Similar findings were noted with parent- and adolescent-report measures. Based on a Clinical Global Impression improvement subscale score of much or very much improved, 52% of subjects in the OROS methylphenidate group improved compared with 31% receiving placebo. Thirty-seven percent of subjects required the maximum dosage of 72 mg/d. The incidence of drug-related adverse events was similar between the 2 study groups. CONCLUSION: In adolescents, once-daily OROS methylphenidate significantly reduced ADHD symptoms and was well tolerated using dosages up to 72 mg/d.