The Hunter Syndrome-Functional Outcomes for Clinical Understanding Scale (HS-FOCUS) Questionnaire: item reduction and further validation

PURPOSE: The Hunter Syndrome-Functional Outcomes for Clinical Understanding Scale (HS-FOCUS) Questionnaire is a patient and parent-completed disease-specific instrument used in Hunter syndrome (mucopolysaccharidosis II), a rare paediatric progressive multi-systemic lysosomal storage disease. The objective of this study was to shorten the number of items of the Questionnaire to reduce response burden while maintaining its content validity. METHODS: Data collected in a clinical trial were used. An iterative process helped identifying redundant or low performing items based on content validity and psychometric properties. Validation on the retained items was assessed using patients and parent’s responses in terms of reliability, validity and responsiveness. RESULTS: The HS-FOCUS was completed by 49 patients and 84 parents. Items were mainly removed owing to high floor effects, high inter-item correlations (>0.80) or inadequate content. The shortened patient and parent versions (18 and 21 items) each contained five function domains. Internal consistency and test–retest reliability were >0.70 for most domains, except Breathing and School/work. Concurrent validity was demonstrated by significant correlations (>0.30) with similar concepts of previously validated measures. Significant differences were found in all domain scores across levels of disability. CONCLUSIONS: The shortened HS-FOCUS is a reliable, valid and responsive measure, where burden in answering the Questionnaire was reduced without compromising its validity

Children's Healthy Living (CHL) Program for remote underserved minority populations in the Pacific region: Rationale and design of a community randomized trial to prevent early childhood obesity

Background: Although surveillance data are limited in the US Affiliated Pacific, Alaska, and Hawaii, existing data suggest that the prevalence of childhood obesity is similar to or in excess of other minority groups in the contiguous US. Strategies for addressing the childhood obesity epidemic in the region support the use of community-based, environmentally targeted interventions. The Children's Healthy Living Program is a partnership formed across institutions in the US Affiliated Pacific, Alaska, and Hawaii to design a community randomized environmental intervention trial and a prevalence survey to address childhood obesity in the region through affecting the food and physical activity environment. Methods/Design. The Children's Healthy Living Program community randomized trial is an environmental intervention trial in four matched-pair communities in American Samoa, the Commonwealth of the Northern Mariana Islands, Guam, and Hawaii and two matched-pair communities in Alaska. A cross-sectional sample of children (goal n = 180) in each of the intervention trial communities is being assessed for outcomes at baseline and at 24 months (18 months post-intervention). In addition to the collection of the participant-based measures of anthropometry, diet, physical activity, sleep and acanthosis nigricans, community assessments are also being conducted in intervention trial communities. The Freely Associated States of Micronesia (Federated States of Micronesia, and Republics of Marshall Islands and Palau) is only conducting elements of the Children's Healthy Living Program sampling framework and similar measurements to provide prevalence data. In addition, anthropometry information will be collected for two additional communities in each of the 5 intervention jurisdictions to be included in the prevalence survey. The effectiveness of the environmental intervention trial is being assessed based on the RE-AIM (reach, effectiveness, adoption, implementation, maintenance) framework. Discussion. The Children's Healthy Living Program environmental trial is designed to focus on capacity building and to maximize the likelihood of sustainable impact on childhood obesity-related behaviors and outcomes. The multiple measures at the individual, community, and environment levels are designed to maximize the likelihood of detecting change. This approach enhances the likelihood for identifying and promoting the best methods to promote health and well-being of the children in the underserved US Affiliated Pacific Region. Trial registration. NIH clinical trial # NCT01881373. © 2013 Wilken et al.; licensee BioMed Central Ltd