Validity and Reliability of the Neuromuscular Gross Motor Outcome - 13/08/21
, Megan A. Iammarino, DPT a, Natalie F. Reash, DPT a, Brenna R. Powers, DPT a, Kiana Shannon, BS a, Anne M. Connolly, MD a, b, c, Megan A. Waldrop, MD a, b, c, Garey H. Noritz, MD b, Richard Shell, MD b, Chang-Yong Tsao, MD b, c, Kevin M. Flanigan, MD a, b, c, Jerry R. Mendell, MD a, b, c, Linda P. Lowes, PT, PhD a, bAbstract |
Background |
Approved treatments in spinal muscular atrophy (SMA) have resulted in unprecedented gains for many individuals. Use of available outcomes, typically developed for a specific type of SMA, do not cover the range of progression, often resulting in a battery of functional testing being completed at visits. Our objective was to validate the Neuromuscular Gross Motor Outcome (GRO) as a tool to quantify function in SMA across the span of abilities.
Methods |
Patients with genetically confirmed SMA completed functional testing at each visit including the Neuromuscular GRO and other appropriate gross motor outcomes.
Results |
We enrolled 91 patients with SMA types 1 to 3 between 8 days and 32.1 years. The GRO utilizes a 0- to 2-point scale with scores in our cohort ranging from 1 to 95 points with no floor or ceiling effect. GRO scores were significantly different across functional categories (P < 0.001) and treatment status (P = 0.01) and correlated to other functional assessments (P ≤ 0.001). All patients were measured using the GRO, whereas traditional outcomes were only appropriate on 36% to 59% of our cohort.
Conclusion |
The Neuromuscular GRO quantifies function across the span of age and abilities included in our cohort, allowing for continuous longitudinal monitoring on one scale to reduce the burden of testing in our heterogeneous clinic population.
Le texte complet de cet article est disponible en PDF.Keywords : Spinal muscular atrophy, Outcome measure, Function, Natural history, Neuromuscular disease, Validation, Neuromuscular GRO
Plan
| Author Contributions: The neuromuscular physical therapy team at Nationwide Children's Hospital, consisting of authors Lindsay N. Alfano, Megan A. Iammarino, Natalie F. Reash, Brenna R. Powers, Kiana Shannon, and Linda P. Lowes participated in all aspects of data collection, analysis and review of the submitted manuscript. Physicians Dr. Anne Connolly, Dr. Megan Waldrop, Dr. Garey Noritz, Dr. Richard Shell, Dr. Chang-Yong Tsao, Dr. Kevin Flanigan, and Dr. Jerry Mendell participated in routine clinical assessments of the subjects recruited from Nationwide Children's Hospital's spinal muscular atrophy clinic, as well as provided significant contributions to the revision of the manuscript. All authors have had complete access to the study data for review and development of this submission. |
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| Conflict of interest: Dr. Alfano reports other support from Novartis Gene Therapies through Nationwide Children’s Hospital, personal fees from Biogen, outside the submitted work. Dr. Iammarino reports personal fees from Genentech, outside the submitted work. Dr. Reash has nothing to disclose. Dr. Powers has nothing to disclose. Ms. Shannon has nothing to disclose. Dr. Connolly reports personal fees from Sarepta Therapeutics, Novartis Gene Therapies, Genentech-Roche, Catabasis, and NS Pharma, outside the submitted work. Dr. Waldrop reports personal fees from Novartis Gene Therapies and Sarepta Therapeutics, outside the submitted work. Dr. Noritz has nothing to disclose. Dr. Shell reports personal fees from Novartis Gene Therapies, outside the submitted work. Dr. Tsao has nothing to disclose. Dr. Flanigan reports other from Sarepta Therapeutics, other from Audentes Therapeutics, outside the submitted work. Dr. Mendell has nothing to disclose. Dr. Lowes has nothing to disclose. |
Vol 122
P. 21-26 - septembre 2021 Retour au numéro
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