ABSTRACT
BACKGROUND: Spinal muscular atrophy (SMA) is a progressive neuromuscular disorder that causes muscle weakness and is the leading genetic cause of infant mortality worldwide. While no definitive cure exists, the approval of 3 genetic-based therapies in Canada since 2018 has led to significant improvements in muscle function for children with SMA. With that, there are no evidence-based rehabilitation interventions and minimal evidence on the combined effects of genetic-based therapies and rehabilitation. OBJECTIVE: This protocol describes the methodology to assess the feasibility of a twice-weekly outpatient rehabilitation intervention focusing on gross and fine motor function to inform the methodology and sample size of a definitive clinical trial. METHODS: We will conduct a single-center nonrandomized pilot and feasibility trial to explore an outpatient rehabilitation intervention for children aged 6 months to 3 years with SMA treated with genetic-based therapies. Participation in the study will occur over a 25-week period, with a baseline assessment visit followed by a 12-week intervention period and a 12-week nonintervention period. The rehabilitation intervention comprises weekly physical and occupational therapy for 11 weeks. Assessments will occur at baseline (week 0), end of intervention or early withdrawal (week 12), and follow-up (week 24). Predetermined feasibility indicators will evaluate study feasibility across process (recruitment rates, eligibility criteria, adherence rates, retention rates, questionnaire suitability, and acceptability), resource (time, implementation, and execution), management (materials and data), and scientific (safety, tolerability, and preliminary efficacy) domains. RESULTS: This project was funded in March 2022, and data will be collected between March 2023 and December 2023. Data analysis will occur between January 2024 and March 2024, with publication expected in the fall of 2024. The protocol for the feasibility trial will be considered successful if it meets the success criteria set out for the feasibility indicators. Indicators of specific interest include all process indicators, as well as time. Exploratory indicators will be reported. Pragmatically, the results of the feasibility trial will inform changes to the protocol and the start-up of a definitive multisite trial. CONCLUSIONS: This novel twice-weekly outpatient rehabilitation intervention will be the first step toward filling the need for an evidence-based rehabilitation intervention for children with SMA treated with genetic-based therapies. It is expected that consistent and intensive rehabilitation therapy will augment functional gains being observed in this population. In the future, a definitive trial will measure the efficacy of the intervention. TRIAL REGISTRATION: ClinicalTrials.gov NCT05638750; https://clinicaltrials.gov/study/NCT05638750. INTERNATIONAL REGISTERED REPORT IDENTIFIER (IRRID): DERR1-10.2196/46363.
ABSTRACT
Word-finding difficulties for naming everyday objects are often prevalent in aphasia. Traditionally, treating these difficulties has involved repeated drilling of troublesome items with a therapist. Spaced repetition schedules can improve the efficiency of such training. However, spaced repetition in a therapy environment can be both difficult to implement and time-consuming. The current study evaluated the potential utility of automated, asynchronous, online spaced repetition training for the treatment of word-finding difficulties in individuals with aphasia. Twenty-one participants completed a two-week training study, completing approximately 60 minutes per day of asynchronous online drilling. The training items were identified using a pretest, and word-finding difficulties were evaluated both at the end of training (i.e., a post-test) and four weeks later (i.e., a retention test). The trained items were separated into three different spaced-repetition schedules: (1) Short-spacing; (2) Long-spacing; and (3) Adaptive-spacing. At the retention-test, all trained items outperformed non-trained items in terms of accuracy and reaction time. Further, preliminary evidence suggested a potential reaction time advantage for the adaptive-spacing condition. Overall, online, asynchronous spaced repetition training appears to be effective in treating word-finding difficulties in aphasia. Further research will be required to determine if different spaced repetition schedules can be leveraged to enhance this effect.
