Translation and transcultural validation of the Dutch hospital for special surgery paediatric functional activity brief scale (HSS Pedi-FABS).

BACKGROUND: There is a need for a validated simple Dutch paediatric activity scale. The purpose was to translate and transculturally validate the Dutch Hospital for Special Surgery Paediatric Functional Activity Brief Scale (HSS Pedi-FABS) questionnaire in healthy children and adolescents. METHODS: The original HSS Pedi-FABS was translated forward and backward and was transculturally adapted after performing a pilot study among children and professionals. The final version of the Dutch HSS Pedi-FABS was validated in healthy children and adolescents aged 10 to 18 years old. Children who had any condition or injury limiting their normal physical activity were excluded. The interval between the first questionnaire T0 (HSS Pedi-FABS, Physical Activity Questionnaire for children or adolescents (PAQ-C/A) and Tegner activity scale) and the second questionnaire T1 (HSS Pedi-FABS) was 2 weeks. Construct validity, interpretability and reliability were evaluated. Content validity was evaluated through cognitive interviews among a smaller group of children and through a questionnaire among professionals. RESULTS: To evaluate content validity, 9 children and adolescents were interviewed, and 30 professionals were consulted. Content validity among professionals showed a relevance of less than 85% for most items on construct. However, content validity among children was good with a 92% score for item relevance. Readability was scored at a reading level of 11- to 12-year-olds. The validation group consisted of 110 healthy children and adolescents (mean age of 13.9 years ±2.6). Construct validity was considered good as 8 out of 10 hypotheses were confirmed. The Dutch HSS Pedi-FABS showed no floor or ceiling effect. Analysis of the internal consistency in the validation group resulted in a Cronbach's alpha of 0.82. Test-retest reliability was evaluated among 69 children and adolescents and revealed an Intraclass Correlation Coefficient (ICC) of 0.76. CONCLUSION: The Dutch HSS Pedi-FABS showed good psychometric properties in a healthy Dutch paediatric and adolescent population. Limitations of the current Dutch HSS Pedi-FABS are content validity on construct of items reported by professionals.

Study Protocol of the Exercise Study: Unraveling Limitations for Physical Activity in Children With Chronic Diseases in Order to Target Them With Tailored Interventions-A Randomized Cross Over Trial.

Introduction: Physical activity is associated with many physiological and psychological health benefits across the lifespan. Children with a chronic disease often have lower levels of daily physical activity, and a decreased exercise capacity compared to healthy peers. In order to learn more about limitations for physical activity, we investigate children with four different chronic diseases: children with a Fontan circulation, children with Broncho Pulmonary Dysplasia (BPD), Pompe disease and inflammatory bowel disease (IBD). Each of these diseases is likely to interfere with physical activity in a different way. Knowing the specific limitations for physical activity would make it possible to target these, and increase physical activity by a personalized intervention. The aim of this study is to first investigate limitations for physical activity in children with various chronic diseases. Secondly, to measure the effects of a tailored exercise intervention, possibly including a personalized dietary advice and/or psychological counseling, on exercise capacity, endurance, quality of life, fatigue, fear for exercise, safety, muscle strength, physical activity levels, energy balance, and body composition. Methods and Analysis: This randomized crossover trial will aim to include 72 children, aged 6-18 years, with one of the following diagnosis: a Fontan circulation, BPD, Pompe disease and IBD. Eligible patients will participate in the 12-week tailored exercise intervention and are either randomized to start with a control period or start with the intervention. The tailored 12-week exercise interventions, possibly including a personalized dietary advice and/or psychological counseling, will be designed based on the found limitations for physical activity in each disease group during baseline measurements by the Rotterdam Exercise Team. Effects of the tailored training interventions will be measured on the following endpoints: exercise capacity (measured by cardiopulmonary exercise test), endurance, physical activity levels, muscle strength, quality of life, fatigue, fear for exercise, disease activity, cardiac function (in children with a Fontan circulation), energy balance, and body composition. Ethics and Dissemination: Conducted according to the Declaration of Helsinki and Good Clinical Practice. Medical-ethical approval was obtained. Trial Registration Number: NL8181, https://www.trialregister.nl/trial/8181.

A case of adult Pompe disease presenting with severe fatigue and selective involvement of type 1 muscle fibers.

We present a case of adult Pompe disease (acid maltase deficiency) with an uncommon clinical presentation characterized by severe fatigue and myalgia prior to the onset of limb girdle weakness. Remarkably, the muscle biopsy demonstrated selective involvement of type 1 muscle fibers. The cause and clinical effects of fiber type specific involvement are currently unknown, but the phenomenon might contribute to the clinical heterogeneity in Pompe disease and the variable response to enzyme replacement therapy.