Perceive, Recall, Plan and Perform (PRPP)-Assessment Based on Parent-Provided Videos of Children with Mitochondrial Disorder: Action Design Research on Implementation Challenges.

AIMS: Performing the Perceive, Recall, Plan and Perform (PRPP)-Assessment, using video material of everyday life, seems sensible to lower the patient burden, enhance ecological validity, and provide care at a distance. However, receipt of adequate video material is not self-evident and assessing videos can be challenging. Therefore, this study aims to optimize the process of gaining video material and to optimize the PRPP-Assessment based on parent-provided videos. METHODS: An action design research method was used, focusing on implementation of the PRPP-Assessment based on parent-provided videos within the care of children with a mitochondrial disorder or similar symptoms. RESULTS: Five cycles were conducted. To receive input, the cycles used videos of nine children performing activities, written feedback, and semi-structured interviews and focus groups comprising parents (n = 13), a teacher (n = 1), occupational therapists (n = 16), and other professionals (n = 2) . This led to successful implementation of the PRPP-Assessment. General lessons were learned on (1) instructing parents; (2) handling video material; (3) PRPP-Assessment based on parent-provided videos; and (4) PRPP-Assessment of children (with limited functional abilities). CONCLUSIONS: Lessons learned should be implemented in practice and are incorporated into a manual to guide the implementation of video-based observations with PRPP-Assessment in practice.

Assisted 6-minute cycling test: An exploratory study in children.

INTRODUCTION: The 6-minute walk test (6MWT) is frequently used as an outcome measure for clinical trials in neuromuscular disease. Because this submaximal endurance test is not feasible for nonambulatory patients, the motor-assisted 6-minute cycling test (A6MCT) was developed. METHODS: Nineteen children with neuromuscular disorders and children with OXPHOS-dysfunction performed the a6MCT and the 6MWT to explore feasibility and construct validity. Test-retest reproducibility was evaluated within 3 weeks. RESULTS: The assisted 6-minute cycling test was feasible in 90% and 78% of the patients with a neuromuscular disorder and OXPHOS-dysfunction, respectively. The A6MCT for legs correlated with the 6MWT in both patient groups. The assisted 6-minute cycling showed good reproducibility for both legs and arms. CONCLUSIONS: This exploratory study indicates that the assisted 6-minute cycling test is a promising outcome measure for patients with a neuromuscular disorder and patients with OXPHOS-dysfunction. Muscle Nerve, 2015. Muscle Nerve 54: 232-238, 2016.

Physical Activity in Boys With Duchenne Muscular Dystrophy Is Lower and Less Demanding Compared to Healthy Boys.

This study describes the amount of physical activity and perception of physical activity in boys with Duchenne muscular dystrophy (DMD) compared to healthy boys. A questionnaire described 6 domains of physical activity. Four Duchenne muscular dystrophy subgroups were made: early and late ambulatory, nonambulatory with relative good, or limited arm function. Eighty-four boys with Duchenne muscular dystrophy (15.0 ± 6.4 years) and 198 healthy boys (14.0 ± 4.3 years) participated. Daily activities were more passive for boys with Duchenne muscular dystrophy. Physical activity was less and low demanding compared to healthy boys. It decreased with disease severity ( P < .05), whereas screen time increased ( P < .05). Benefits of physical activity in boys with Duchenne muscular dystrophy were having fun and making friends. Barriers were lack of sport facilities and insufficient health. This study helps to quantify poor engagement in physical activity by boys with Duchenne muscular dystrophy, and demonstrates factors that contribute to it. Suggestions to stimulate physical activity are made.