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Reliability of 4 outcome measures in pediatric spinal muscular atrophy
Susan T Iannaccone1, Linda S Hynan,
1Department of Neurology, Neuromuscular Disease and Neurorehabilitation, Texas Scottish Rite Hospital for Children, 2222 Welborn Street, Dallas, TX 75219, USA. susan.iannaccone@tsrh.org
Insights
Four outcome measures, including the Gross Motor Function Measure, are reliable for clinical trials in pediatric spinal muscular atrophy (SMA) patients. This study confirms their utility for evaluating treatment efficacy in this vulnerable population.
Area of Science:
- Neurology
- Pediatrics
- Clinical Trials
Background:
- Spinal muscular atrophy (SMA) is a frequent and often fatal neurologic disorder in children.
- Clinical trials for SMA in pediatric populations are a recent development.
Purpose of the Study:
- To establish the reliability of four key outcome measures for use in clinical trials involving pediatric SMA patients.
- To validate assessment tools for future therapeutic evaluations.
Main Methods:
- A reliability study involving 38 children diagnosed with SMA across five pediatric centers.
- Paired samples statistics were employed to compare data from different assessment visits.
- Evaluated measures included the Gross Motor Function Measure, pulmonary function tests, quantitative muscle testing, and quality of life surveys.
Main Results:
- Thirty-four patients and seven evaluators completed the study, with participants aged 2 to 17 years.
- High levels of agreement were observed between repeated measures, confirmed by intraclass correlation coefficients and Bradley-Blackwood procedures.
- The Gross Motor Function Measure, pulmonary function tests, quantitative muscle testing, and PedsQL Neuromuscular Module demonstrated strong reliability.
Conclusions:
- The Gross Motor Function Measure, pulmonary function tests, quantitative muscle testing, and quality of life assessments are reliable outcome measures for pediatric SMA clinical trials.
- These validated measures can confidently be used to assess treatment effectiveness in children with spinal muscular atrophy.
Background:
Spinal muscular atrophy is a common neurologic disorder of infants and children with a high mortality rate. Clinical trials have not been attempted in this population until recently.
Objective:
To demonstrate that 4 outcome measures are reliable for use in clinical trials in patients with spinal muscular atrophy.
Design, Setting, Patients:
Thirty-eight children with spinal muscular atrophy who fulfilled inclusion and exclusion criteria were enrolled at 5 pediatric centers for a reliability study. Paired samples statistics were performed comparing results of the qualifying variance visit with a fourth visit.
Main Outcome Measures:
Quantitative muscle testing and the Gross Motor Function Measure.
Results:
Thirty-four patients and 7 evaluators completed the study. Thirteen patients were aged 2 through 4 years and 21 were 5 through 17 years. The Gross Motor Function Measure was completed by 34 subjects. Six variables for pulmonary function tests were measured in 20 subjects. Quantitative muscle testing was performed on 21 subjects in 8 muscle groups. Thirty-three subjects completed the PedsQL Neuromuscular Module for Parents. The intraclass correlation coefficient and Bradley-Blackwood procedures indicated a very high level of agreement between measures.
Conclusion:
The Gross Motor Function Measure, pulmonary function tests, quantitative muscle testing, and quality of life are reliable outcome measures for clinical trials in pediatric spinal muscular atrophy.
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