Reevaluating measures of disease progression in facioscapulohumeral muscular dystrophy
Recent advances in the understanding of the molecular pathophysiology of facioscapulohumeral muscular dystrophy (FSHD) have identified potential therapeutic targets. Consequently, an accurate understanding of disease progression in FSHD is crucial for the design of future clinical trials. Data from...
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| Published in | Neuromuscular disorders : NMD Vol. 23; no. 4; pp. 306 - 312 |
|---|---|
| Main Authors | , , , , , , , , |
| Format | Journal Article |
| Language | English |
| Published |
England
Elsevier B.V
01.04.2013
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| Subjects | |
| Online Access | Get full text |
| ISSN | 0960-8966 1873-2364 1873-2364 |
| DOI | 10.1016/j.nmd.2013.01.008 |
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| Abstract | Recent advances in the understanding of the molecular pathophysiology of facioscapulohumeral muscular dystrophy (FSHD) have identified potential therapeutic targets. Consequently, an accurate understanding of disease progression in FSHD is crucial for the design of future clinical trials. Data from 228 subjects in 3 clinical trials and 1 natural history study were compared to examine disease progression in FSHD. All studies utilized the same techniques for manual muscle testing and maximum voluntary isometric contraction testing. Both techniques yield a total strength score that can be followed over time as an indicator of disease progression. Whereas natural history data showed a decrease in strength over 1year, there was an apparent increase in strength at 6months in 2 of the 3 clinical trials in both the placebo and treatment groups, that persisted for up to 1year for maximum voluntary isometric contraction testing. Variability estimates from the clinical trial data were consistent with those seen in the natural history data. Patients in clinical trials in FSHD may have better outcomes than those in natural history studies, regardless of treatment assignment, emphasizing the importance of placebo groups and the need for caution when interpreting the strength results of controlled and uncontrolled trials. |
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| AbstractList | Recent advances in the understanding of the molecular pathophysiology of facioscapulohumeral muscular dystrophy (FSHD) have identified potential therapeutic targets. Consequently, an accurate understanding of disease progression in FSHD is crucial for the design of future clinical trials. Data from 228 subjects in 3 clinical trials and 1 natural history study were compared to examine disease progression in FSHD. All studies utilized the same techniques for manual muscle testing and maximum voluntary isometric contraction testing. Both techniques yield a total strength score that can be followed over time as an indicator of disease progression. Whereas natural history data showed a decrease in strength over 1 year, there was an apparent increase in strength at 6 months in 2 of the 3 clinical trials in both the placebo and treatment groups, that persisted for up to 1 year for maximum voluntary isometric contraction testing. Variability estimates from the clinical trial data were consistent with those seen in the natural history data. Patients in clinical trials in FSHD may have better outcomes than those in natural history studies, regardless of treatment assignment, emphasizing the importance of placebo groups and the need for caution when interpreting the strength results of controlled and uncontrolled trials.Recent advances in the understanding of the molecular pathophysiology of facioscapulohumeral muscular dystrophy (FSHD) have identified potential therapeutic targets. Consequently, an accurate understanding of disease progression in FSHD is crucial for the design of future clinical trials. Data from 228 subjects in 3 clinical trials and 1 natural history study were compared to examine disease progression in FSHD. All studies utilized the same techniques for manual muscle testing and maximum voluntary isometric contraction testing. Both techniques yield a total strength score that can be followed over time as an indicator of disease progression. Whereas natural history data showed a decrease in strength over 1 year, there was an apparent increase in strength at 6 months in 2 of the 3 clinical trials in both the placebo and treatment groups, that persisted for up to 1 year for maximum voluntary isometric contraction testing. Variability estimates from the clinical trial data were consistent with those seen in the natural history data. Patients in clinical trials in FSHD may have better outcomes than those in natural history studies, regardless of treatment assignment, emphasizing the importance of placebo groups and the need for caution when interpreting the strength results of controlled and uncontrolled trials. Recent advances in the understanding of the molecular pathophysiology of facioscapulohumeral muscular dystrophy (FSHD) have identified potential therapeutic targets. Consequently, an accurate understanding of disease progression in FSHD is crucial for the design of future clinical trials. Data from 228 subjects in 3 clinical trials and 1 natural history study were compared to examine disease progression in FSHD. All studies utilized the same techniques for manual muscle testing and maximum voluntary isometric contraction testing. Both techniques yield a total strength score that can be followed over time as an indicator of disease progression. Whereas natural history data showed a decrease in strength over 1 year, there was an apparent increase in strength at 6 months in 2 of the 3 clinical trials in both the placebo and treatment groups, that persisted for up to 1 year for maximum voluntary isometric contraction testing. Variability estimates from the clinical trial data were consistent with those seen in the natural history data. Patients in clinical trials in FSHD may have better outcomes than those in natural history studies, regardless of treatment assignment, emphasizing the importance of placebo groups and the need for caution when interpreting the strength results of controlled and uncontrolled trials. Recent advances in the understanding of the molecular pathophysiology of facioscapulohumeral muscular dystrophy (FSHD) have identified potential therapeutic targets. Consequently, an accurate understanding of disease progression in FSHD is crucial for the design of future clinical trials. Data from 228 subjects in 3 clinical trials and 1 natural history study were compared to examine disease progression in FSHD. All studies utilized the same techniques for manual muscle testing and maximum voluntary isometric contraction testing. Both techniques yield a total strength score that can be followed over time as an indicator of disease progression. Whereas natural history data showed a decrease in strength over 1year, there was an apparent increase in strength at 6months in 2 of the 3 clinical trials in both the placebo and treatment groups, that persisted for up to 1year for maximum voluntary isometric contraction testing. Variability estimates from the clinical trial data were consistent with those seen in the natural history data. Patients in clinical trials in FSHD may have better outcomes than those in natural history studies, regardless of treatment assignment, emphasizing the importance of placebo groups and the need for caution when interpreting the strength results of controlled and uncontrolled trials. Abstract Recent advances in the understanding of the molecular pathophysiology of facioscapulohumeral muscular dystrophy (FSHD) have identified potential therapeutic targets. Consequently, an accurate understanding of disease progression in FSHD is crucial for the design of future clinical trials. Data from 228 subjects in 3 clinical trials and 1 natural history study were compared to examine disease progression in FSHD. All studies utilized the same techniques for manual muscle testing and maximum voluntary isometric contraction testing. Both techniques yield a total strength score that can be followed over time as an indicator of disease progression. Whereas natural history data showed a decrease in strength over 1 year, there was an apparent increase in strength at 6 months in 2 of the 3 clinical trials in both the placebo and treatment groups, that persisted for up to 1 year for maximum voluntary isometric contraction testing. Variability estimates from the clinical trial data were consistent with those seen in the natural history data. Patients in clinical trials in FSHD may have better outcomes than those in natural history studies, regardless of treatment assignment, emphasizing the importance of placebo groups and the need for caution when interpreting the strength results of controlled and uncontrolled trials. |
| Author | Heatwole, Chad Wagner, Kathryn R. Statland, Jeffrey M. Martens, William B. Pandya, Shree McDermott, Michael P. van der Kooi, E.L. Kissel, John T. Tawil, Rabi |
| AuthorAffiliation | 1 Department of Neurology, University of Rochester Medical Center, Rochester, NY USA 4 Departments of Neurology and Pediatrics, The Ohio State University and Nationwide Children's Hospital, Columbus, OH USA 5 Center for Genetic Muscle Disorders, The Kennedy Krieger Institute, and Departments of Neurology and Neuroscience The Johns Hopkins School of Medicine, Baltimore, MD USA 2 Department of Biostatistics and Computational Biology, University of Rochester Medical Center, Rochester, NY USA 3 Department of Neurology, Medical Center Leeuwarden, Leeuwarden, The Netherlands |
| AuthorAffiliation_xml | – name: 5 Center for Genetic Muscle Disorders, The Kennedy Krieger Institute, and Departments of Neurology and Neuroscience The Johns Hopkins School of Medicine, Baltimore, MD USA – name: 3 Department of Neurology, Medical Center Leeuwarden, Leeuwarden, The Netherlands – name: 4 Departments of Neurology and Pediatrics, The Ohio State University and Nationwide Children's Hospital, Columbus, OH USA – name: 1 Department of Neurology, University of Rochester Medical Center, Rochester, NY USA – name: 2 Department of Biostatistics and Computational Biology, University of Rochester Medical Center, Rochester, NY USA |
| Author_xml | – sequence: 1 givenname: Jeffrey M. surname: Statland fullname: Statland, Jeffrey M. email: Jeffrey_Statland@URMC.Rochester.edu organization: Department of Neurology, University of Rochester Medical Center, Rochester, NY, USA – sequence: 2 givenname: Michael P. surname: McDermott fullname: McDermott, Michael P. organization: Department of Neurology, University of Rochester Medical Center, Rochester, NY, USA – sequence: 3 givenname: Chad surname: Heatwole fullname: Heatwole, Chad organization: Department of Neurology, University of Rochester Medical Center, Rochester, NY, USA – sequence: 4 givenname: William B. surname: Martens fullname: Martens, William B. organization: Department of Neurology, University of Rochester Medical Center, Rochester, NY, USA – sequence: 5 givenname: Shree surname: Pandya fullname: Pandya, Shree organization: Department of Neurology, University of Rochester Medical Center, Rochester, NY, USA – sequence: 6 givenname: E.L. surname: van der Kooi fullname: van der Kooi, E.L. organization: Department of Neurology, Medical Center Leeuwarden, Leeuwarden, The Netherlands – sequence: 7 givenname: John T. surname: Kissel fullname: Kissel, John T. organization: Departments of Neurology and Pediatrics, The Ohio State University and Nationwide Children’s Hospital, Columbus, OH, USA – sequence: 8 givenname: Kathryn R. surname: Wagner fullname: Wagner, Kathryn R. organization: Center for Genetic Muscle Disorders, The Kennedy Krieger Institute, and Departments of Neurology and Neuroscience, The Johns Hopkins School of Medicine, Baltimore, MD, USA – sequence: 9 givenname: Rabi surname: Tawil fullname: Tawil, Rabi organization: Department of Neurology, University of Rochester Medical Center, Rochester, NY, USA |
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| SubjectTerms | Adult All clinical trials All neuromuscular disease Clinical trials methodology/study design Disease Progression Female Humans Isometric Contraction - physiology Longitudinal Studies Male Middle Aged Muscle disease Muscle Strength - physiology Muscle, Skeletal - physiopathology Muscular Dystrophy, Facioscapulohumeral - physiopathology Neurology Outcome research Randomized Controlled Trials as Topic |
| Title | Reevaluating measures of disease progression in facioscapulohumeral muscular dystrophy |
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