Electrical impedance myography for reducing sample size in Duchenne muscular dystrophy trials
Abstract Objective To evaluate the sensitivity of electrical impedance myography (EIM) to disease progression in both ambulatory and non‐ambulatory boys with DMD. Methods and Participants A non‐blinded, longitudinal cohort study of 29 ambulatory and 15 non‐ambulatory boys with DMD and age‐similar he...
| Main Authors: | , , , , , , , , , , , |
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| Format: | Article |
| Language: | English |
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Wiley
2020-01-01
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| Series: | Annals of Clinical and Translational Neurology |
| Online Access: | https://doi.org/10.1002/acn3.50958 |
| _version_ | 1819091583668060160 |
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| author | Melanie L. Leitner Kush Kapur Basil T. Darras Michele Yang Brenda Wong Laura Dalle Pazze Julaine Florence Martin Buck Laura Freedman Jose Bohorquez Seward Rutkove Craig Zaidman |
| author_facet | Melanie L. Leitner Kush Kapur Basil T. Darras Michele Yang Brenda Wong Laura Dalle Pazze Julaine Florence Martin Buck Laura Freedman Jose Bohorquez Seward Rutkove Craig Zaidman |
| author_sort | Melanie L. Leitner |
| collection | DOAJ |
| description | Abstract Objective To evaluate the sensitivity of electrical impedance myography (EIM) to disease progression in both ambulatory and non‐ambulatory boys with DMD. Methods and Participants A non‐blinded, longitudinal cohort study of 29 ambulatory and 15 non‐ambulatory boys with DMD and age‐similar healthy boys. Subjects were followed for up to 1 year and assessed using the Myolex® mViewTM EIM system as part of a multicenter study. Results In the ambulatory group, EIM 100 kHz resistance values showed significant change compared to the healthy boys. For example, in lower extremity muscles, the average change in EIM 100 kHz resistance values over 12 months led to an estimated effect size of 1.58. Based on these results, 26 DMD patients/arm would be needed for a 12‐month clinical trial assuming a 50% treatment effect. In non‐ambulatory boys, EIM changes were greater in upper limb muscles. For example, biceps at 100kHz resistance gave an estimated effect size of 1.92 at 12 months. Based on these results, 18 non‐ambulatory DMD patients/arm would be needed for a 12‐month clinical trial assuming a 50% treatment effect. Longitudinal changes in the 100 kHz resistance values for the ambulatory boys correlated with the longitudinal changes in the timed supine‐to‐stand test. EIM was well‐tolerated throughout the study. Interpretation This study supports that EIM 100 kHz resistance is sensitive to DMD progression in both ambulatory and non‐ambulatory boys. Given the technology’s ease of use and broad age range of utility it should be employed as an exploratory endpoint in future clinical therapeutic trials in DMD. Trial Registration: Clincialtrials.gov registration #NCT02340923 |
| first_indexed | 2024-12-21T22:42:02Z |
| format | Article |
| id | doaj.art-0f3e4106a6a4413997711be74d88d6bd |
| institution | Directory Open Access Journal |
| issn | 2328-9503 |
| language | English |
| last_indexed | 2024-12-21T22:42:02Z |
| publishDate | 2020-01-01 |
| publisher | Wiley |
| record_format | Article |
| series | Annals of Clinical and Translational Neurology |
| spelling | doaj.art-0f3e4106a6a4413997711be74d88d6bd2022-12-21T18:47:49ZengWileyAnnals of Clinical and Translational Neurology2328-95032020-01-017141410.1002/acn3.50958Electrical impedance myography for reducing sample size in Duchenne muscular dystrophy trialsMelanie L. Leitner0Kush Kapur1Basil T. Darras2Michele Yang3Brenda Wong4Laura Dalle Pazze5Julaine Florence6Martin Buck7Laura Freedman8Jose Bohorquez9Seward Rutkove10Craig Zaidman11Accelerating NeuroVentures LLC Needham MassachusettsDepartment of Neurology Boston Children’s Hospital Boston MassachusettsDepartment of Neurology Boston Children’s Hospital Boston MassachusettsDepartment of Neurology Children’s Hospital Colorado Denver ColoradoDepartment of Pediatrics University of Massachusetts Medical School Worcester MassachusettsCharley’s Fund New York New YorkDepartment of Neurology Washington University in St. Louis St. Louis MissouriMyolex Inc Brookline MassachusettsMyolex Inc Brookline MassachusettsMyolex Inc Brookline MassachusettsMyolex Inc Brookline MassachusettsDepartment of Neurology Washington University in St. Louis St. Louis MissouriAbstract Objective To evaluate the sensitivity of electrical impedance myography (EIM) to disease progression in both ambulatory and non‐ambulatory boys with DMD. Methods and Participants A non‐blinded, longitudinal cohort study of 29 ambulatory and 15 non‐ambulatory boys with DMD and age‐similar healthy boys. Subjects were followed for up to 1 year and assessed using the Myolex® mViewTM EIM system as part of a multicenter study. Results In the ambulatory group, EIM 100 kHz resistance values showed significant change compared to the healthy boys. For example, in lower extremity muscles, the average change in EIM 100 kHz resistance values over 12 months led to an estimated effect size of 1.58. Based on these results, 26 DMD patients/arm would be needed for a 12‐month clinical trial assuming a 50% treatment effect. In non‐ambulatory boys, EIM changes were greater in upper limb muscles. For example, biceps at 100kHz resistance gave an estimated effect size of 1.92 at 12 months. Based on these results, 18 non‐ambulatory DMD patients/arm would be needed for a 12‐month clinical trial assuming a 50% treatment effect. Longitudinal changes in the 100 kHz resistance values for the ambulatory boys correlated with the longitudinal changes in the timed supine‐to‐stand test. EIM was well‐tolerated throughout the study. Interpretation This study supports that EIM 100 kHz resistance is sensitive to DMD progression in both ambulatory and non‐ambulatory boys. Given the technology’s ease of use and broad age range of utility it should be employed as an exploratory endpoint in future clinical therapeutic trials in DMD. Trial Registration: Clincialtrials.gov registration #NCT02340923https://doi.org/10.1002/acn3.50958 |
| spellingShingle | Melanie L. Leitner Kush Kapur Basil T. Darras Michele Yang Brenda Wong Laura Dalle Pazze Julaine Florence Martin Buck Laura Freedman Jose Bohorquez Seward Rutkove Craig Zaidman Electrical impedance myography for reducing sample size in Duchenne muscular dystrophy trials Annals of Clinical and Translational Neurology |
| title | Electrical impedance myography for reducing sample size in Duchenne muscular dystrophy trials |
| title_full | Electrical impedance myography for reducing sample size in Duchenne muscular dystrophy trials |
| title_fullStr | Electrical impedance myography for reducing sample size in Duchenne muscular dystrophy trials |
| title_full_unstemmed | Electrical impedance myography for reducing sample size in Duchenne muscular dystrophy trials |
| title_short | Electrical impedance myography for reducing sample size in Duchenne muscular dystrophy trials |
| title_sort | electrical impedance myography for reducing sample size in duchenne muscular dystrophy trials |
| url | https://doi.org/10.1002/acn3.50958 |
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