AMEsobreRuedas Early Powered Mobility in Children with Spinal Muscular Atrophy Type I: Protocol of a Randomized Controlled Trial †
Abstract
:1. Introduction
2. Materials and Methods
2.1. Study Sample
2.2. Outcome Variables
- Primary outcomes: measured at baseline, at weeks 8 and 12—during and after the structured intervention—and at week 16 after the follow-up period.
- Participation: measured with the Spanish version of the Young Children’s Participation and Environment Measure (YC-PEM) [30]. The YC-PEM provides insights into family members’ perspectives on the participation of children from 0 to 5 years old in various activities at home, preschool, and the community. For each activity type, the frequency of participation is rated on a scale from 0 to 7 (0 = never; 7 = one or more times a day). Participation level is rated on a 5-point scale (1 = little involvement; 5 = very involved), and desired changes in participation are recorded as yes or no. Additionally, the impact of environmental characteristics and resources on participation is rated on a scale from 1 to 3 (1 = usually difficult/usually not; 3 = generally helps/usually yes).
- Functional ability: assessed using the Pediatric Evaluation of Disability Inventory–Computer Adaptive Test (PEDI-CAT) questionnaire [31]. Parents or caregivers complete the PEDI-CAT through an adaptive computer platform. The PEDI-CAT includes a bank of items across two domains: Mobility (75 items, ranging from basic motor skills to more complex skills, including the use of walking devices) and Social/Cognitive (60 items covering interaction, communication, daily cognition, and self-control).
- Quality of life: evaluated using the Spanish version of the Pediatric Quality of Life Inventory (PedsQL)-Neuromuscular module [32]. The PedsQL is a parent-reported tool designed to evaluate perceptions of the child’s quality of life. It includes three dimensions: 17 questions on neuromuscular disease, 3 questions on communication, and 5 questions about family resources. Responses range from 0 (never) to 4 (almost always), which are converted into a score from 0 to 100. Scores are interpreted as follows: 100—never; 75—almost never; 50—sometimes; 25—frequently; and 0—almost always.
- Secondary outcomes:
- Learning of powered mobility: assessed with the Assessment of Learning Powered Mobility (ALP) [33]. The ALP describes 8 levels of performance (1 = beginner; 8 = expert) in using a powered mobility device, based on five categories: attention level; activity and movement; comprehension of the mobility device; expressions and emotions; and interaction and communication [34]. The ALP levels will be assessed weekly to help guide families on the strategies to use during their sessions and ensure consistent progress.
- Functional goals: measured with the goal attainment scale (GAS) [35] at baseline and at weeks 4, 8, 12, and 16 assessments. The goals focus on the use of powered mobility in the child’s natural environment and will be developed in collaboration with the child’s family and/or caregivers.
- Parental stress: assessed with the Spanish version of the Parenting Score Index-Short Form (PSI-SF) [36] at baseline and weeks 8, 12, and 16. The PSI-SF is a 36-item self-reported questionnaire that includes three subscales: parental distress (PD), parent–child dysfunctional interaction (PCDI), and difficult child (DC).
- Satisfaction with home intervention: scored with the Spanish version of the Client Satisfaction Questionnaire (CSQ-8) [37] at weeks 4, 8, and 12. The CSQ-8 measures overall satisfaction with the received service through 8 items scored on a scale from 1 to 4. The total score ranges from 8 to 32, with higher scores indicating greater satisfaction.
- Satisfaction with the use of the powered mobility device: assessed with the Spanish version of the Quebec User Evaluation of Satisfaction with assistive Technology Version 2.0 (QUEST 2.0) [38] at week 16 by the end of this study. The QUEST 2.0 is a self-administered instrument designed to assess satisfaction with various assistive technologies and related services. It comprises two subscales for devices and services. Each item is rated from 1 (not satisfied) to 5 (very satisfied). The device subscale contains 8 questions addressing the following dimensions: size, durability, weight, safety, fit, comfort, ease of use, and effectiveness. The services subscale contains 4 questions evaluating the provision of services, professional services, repairs, and follow-up support for the device.
2.3. Study Flow
2.4. Intervention Features
2.5. Adaptation of the Powered Mobility Device
2.6. Structured Intervention (Weeks 1 to 12) with the Powered Mobility Device
- First Weekly Session: each week, the therapist will conduct the session for approximately one hour. The first 30 min will be dedicated to addressing the family’s needs, evaluating the child’s progress using the ALP, and teaching the family the strategies and structure for the remaining 30 min of training. It is essential for a family member to be present in this session to learn the strategies to be used in the subsequent sessions.
- Second and Third Weekly Sessions: these sessions will be conducted by the families and last for 30 min each. They will carry out the training with the MROC, using the strategies they learned during the first session of the week with the therapist.
2.7. Follow-up Period (Weeks 13 to 16)—Free Use of Powered Mobility Devices
2.8. Statistical Analysis
3. Results
4. Discussion
5. Conclusions
Author Contributions
Funding
Institutional Review Board Statement
Informed Consent Statement
Data Availability Statement
Conflicts of Interest
References
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Palomo-Carrión, R.; López-Muñoz, P.; Longo, E.; Romay-Barrero, H.; Ródenas-Martínez, M.; Plasencia-Robledo, M.; de Andrés-Beltrán, B.; Coello-Villalón, M.; Díaz-López, C.; Pacheco-da-Costa, S. AMEsobreRuedas Early Powered Mobility in Children with Spinal Muscular Atrophy Type I: Protocol of a Randomized Controlled Trial. J. Clin. Med. 2024, 13, 4875. https://doi.org/10.3390/jcm13164875
Palomo-Carrión R, López-Muñoz P, Longo E, Romay-Barrero H, Ródenas-Martínez M, Plasencia-Robledo M, de Andrés-Beltrán B, Coello-Villalón M, Díaz-López C, Pacheco-da-Costa S. AMEsobreRuedas Early Powered Mobility in Children with Spinal Muscular Atrophy Type I: Protocol of a Randomized Controlled Trial. Journal of Clinical Medicine. 2024; 13(16):4875. https://doi.org/10.3390/jcm13164875
Chicago/Turabian StylePalomo-Carrión, Rocío, Purificación López-Muñoz, Egmar Longo, Helena Romay-Barrero, Maribel Ródenas-Martínez, María Plasencia-Robledo, Beatriz de Andrés-Beltrán, María Coello-Villalón, Cristina Díaz-López, and Soraya Pacheco-da-Costa. 2024. "AMEsobreRuedas Early Powered Mobility in Children with Spinal Muscular Atrophy Type I: Protocol of a Randomized Controlled Trial" Journal of Clinical Medicine 13, no. 16: 4875. https://doi.org/10.3390/jcm13164875