Title

Comparison of strength testing modalities in dysferlinopathy

Authors

Natalie F. Reash, The Abigail Wexner Research Institute at Nationwide Children's Hospital, Columbus, Ohio.
Meredith K. James, The John Walton Muscular Dystrophy Research Centre, Translational and Clinical Research Institute, Newcastle University and Newcastle Hospitals NHS Foundation Trust, Newcastle upon Tyne, UK.
Lindsay N. Alfano, The Abigail Wexner Research Institute at Nationwide Children's Hospital, Columbus, Ohio.
Anna G. Mayhew, The John Walton Muscular Dystrophy Research Centre, Translational and Clinical Research Institute, Newcastle University and Newcastle Hospitals NHS Foundation Trust, Newcastle upon Tyne, UK.
Marni Jacobs, Center for Translational Science, Division of Biostatistics and Study Methodology, Children's National Health System, Washington, DC.
Megan A. Iammarino, The Abigail Wexner Research Institute at Nationwide Children's Hospital, Columbus, Ohio.
Scott Holsten, Neuroscience Institute, Carolinas Neuromuscular/ALS-MDA Center, Carolinas HealthCare System, Charlotte, North Carolina.
Chikako Sakamoto, Department of Physical Rehabilitation, National Center Hospital, National Center of Neurology and Psychiatry, Tokyo, Japan.
Takayuki Tateishi, Department of Physical Rehabilitation, National Center Hospital, National Center of Neurology and Psychiatry, Tokyo, Japan.
Hiroyuki Yajima, Department of Physical Rehabilitation, National Center Hospital, National Center of Neurology and Psychiatry, Tokyo, Japan.
Tina Duong, Cooperative International Neuromuscular Research Group, Children's National Health System, Washington, DC.
Brittney de Wolf, Cooperative International Neuromuscular Research Group, Children's National Health System, Washington, DC.
Richard Gee, Lucile Salter Packard Children's Hospital at Stanford, Neurology, Palo Alto, California.
Diana X. Bharucha-Goebel, Department of Neurology Children's National Health System, Washington, DC.
Elena Bravver, Neuroscience Institute, Carolinas Neuromuscular/ALS-MDA Center, Carolinas HealthCare System, Charlotte, North Carolina.
Madoka Mori-Yoshimura, Department of Neurology, National Center Hospital, National Center of Neurology and Psychiatry, Tokyo, Japan.
Kate Bushby, The John Walton Muscular Dystrophy Research Centre, Translational and Clinical Research Institute, Newcastle University and Newcastle Hospitals NHS Foundation Trust, Newcastle upon Tyne, UK.
Laura E. Rufibach, The Jain Foundation, Seattle, Washington.
Volker Straub, The John Walton Muscular Dystrophy Research Centre, Translational and Clinical Research Institute, Newcastle University and Newcastle Hospitals NHS Foundation Trust, Newcastle upon Tyne, UK.
Linda P. Lowes, The Abigail Wexner Research Institute at Nationwide Children's Hospital, Columbus, Ohio.

Document Type

Journal Article

Publication Date

5-4-2022

Journal

Muscle & nerve

DOI

10.1002/mus.27570

Keywords

dysferlinopathy; handheld dynamometry; limb girdle muscular dystrophy; outcome measure; strength

Abstract

INTRODUCTION/AIMS: Dysferlinopathy demonstrates heterogeneity in muscle weakness between patients, which can progress at different rates over time. Changing muscle strength due to disease progression or from an investigational product is associated with changing functional ability. The purpose of this study was to compare three methods of strength testing used in the Clinical Outcome Study (COS) for dysferlinopathy to understand which method and which muscle groups were most sensitive to change over time. METHODS: Patients were evaluated at each study visit using functional scales, manual muscle testing, and handheld dynamometry (HHD) at all 15 sites. A fixed-frame system (Fixed) was used at a subset of seven sites. Screening and baseline visits were evaluated for reliability. Data over a 1-year period were analyzed to determine sensitivity to change among strength modalities and individual muscle groups. RESULTS: HHD and Fixed captured significant change across 1 year in summed muscle strength score of four muscle groups (P < .01). Strength summed scores were significantly correlated with functional scales (rho = 0.68-0.92, P < .001). Individual muscle groups, however, showed high levels of variability between visits. DISCUSSION: Although both HHD and Fixed demonstrate change over 12 months, HHD is a less expensive option that provides data on a continuous scale and may be easier to implement. Due to variability in strength measures, researchers should carefully consider use of strength testing as an outcome and may wish to select functional measures with less variability as clinical trial endpoints.

Department

Pediatrics

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