Paula Bray

Introduction/Aims Young people with Duchenne muscular dystrophy (DMD) are at increased risk of obesity. Weight management is important to families; however, several barriers exist. This pilot study aimed to investigate the feasibility and acceptability of a co‐designed weight management program for DMD. Methods The Supporting Nutrition and Optimizing Wellbeing Program (SNOW‐P) was a single‐arm diet and behavior weight management intervention delivered via weekly telehealth/phone visits over 6 weeks to young people with DMD and obesity (body mass index (BMI) ≥95th percentile) and their caregivers. Using an online survey, caregivers of boys with DMD were consulted on the structure and topics delivered in SNOW‐P. Primary outcomes were feasibility and acceptability; secondary outcomes were weight, physical function, and quality of life at 6‐ and 12‐weeks follow‐up. Results Of nineteen eligible participants, eight were enrolled (median age 11.4 years, range 4.9–15.8), and seven completed the program. Visit attendance was high (88%–100%); most participants reported high satisfaction and that participation was easy. Suggested changes included online and visual DMD‐specific resources. At 6‐weeks, median change in weight z‐scores was −0.01 (IQR: −0.23, 0.17) indicating that on average, weight gain tracked as expected for age. Waist circumference measured by caregivers lacked accuracy and the completion rate of caregiver‐reported secondary outcome measures (e.g., food diaries) was low. Discussion A co‐designed, telehealth/phone weight management program appeared to be feasible and acceptable in a small group of boys with DMD. An adapted, hybrid telehealth and face‐to‐face program is recommended for efficacy testing.

Aim To determine the quality and utility of proxy‐reported sensory measures for children and adolescents with neurodevelopmental disorders (such as autism spectrum disorder, attention‐deficit/hyperactivity disorder, movement disorders, and intellectual disability). Method We systematically searched 11 databases. We applied the updated Consensus‐based Standards for the selection of health Measurement INstruments (COSMIN) Risk of Bias checklist and criteria for good measurement properties to evaluate instrument development and psychometric properties. Findings were summarized using a COSMIN adaptation of Grading of Recommendations, Assessment, Development and Evaluations. Results From 11 databases, 6748 articles were screened. Ninety‐one full‐length articles were reviewed after removing excluded studies and manual searches conducted by two reviewers. Data were extracted for 12 measures from 20 articles. Of the 12 measures, only three provided sufficient data to evaluate content validity and psychometric measurement properties. The Participation and Sensory Environment Questionnaire‐Home (PSEQ‐H) was the only measure that satisfied moderate content validity and moderate‐to‐high quality for measurement properties. These properties included: structural validity, hypothesis testing for construct validity, internal consistency, reliability, and measurement error. Interpretation One measure, the PSEQ‐H, met eight criteria for good measurement properties. To facilitate evidence‐informed clinical decision‐making, all psychometric properties of all 12 sensory‐based, proxy‐reported measures were presented. The importance of consumer engagement in measure development and the need for ongoing evaluation of measures against contemporaneous standards is recommended. What this paper adds Three measures provided studies on content validity and psychometric measurement properties. The Participation and Sensory Environment Questionnaire‐Home had moderate quality for content validity studies and high‐to‐moderate quality evidence for psychometric properties. The Participation and Sensory Environment Questionnaire was the only measure that included consumer involvement through qualitative interviews and pilot testing. Consumer involvement in measure development is important for content validity. Ongoing evaluation of measures against contemporaneous standards is recommended.