Anne M Connolly1,2, Julaine M Florence3, Craig M Zaidman3,4, Paul T Golumbek3,4, Jerry R Mendell5, Kevin M Flanigan5, Peter I Karachunski6, John W Day7, Craig M McDonald8, Basil T Darras9, Peter B Kang10, Catherine A Siener3, Rebecca K Gadeken3, Pallavi Anand3, Jeanine R Schierbecker3, Elizabeth C Malkus3, Linda P Lowes5, Lindsay N Alfano5, Linda Johnson8, Alina Nicorici8, Jason M Kelecic6, Janet Quigley9, Amy E Pasternak9, J Philip Miller11. 1. Department of Neurology, Washington University School of Medicine, Saint Louis, Missouri, USA. connollya@neuro.wustl.edu. 2. Department of Pediatrics, Washington University School of Medicine, Saint Louis, Missouri, USA. connollya@neuro.wustl.edu. 3. Department of Neurology, Washington University School of Medicine, Saint Louis, Missouri, USA. 4. Department of Pediatrics, Washington University School of Medicine, Saint Louis, Missouri, USA. 5. Department of Pediatrics, Ohio State University, and the Center for Gene Therapy, Nationwide Children's Hospital, Columbus, Ohio, USA. 6. Department of Neurology, University of Minnesota, Minneapolis, Minnesota, USA. 7. Department of Neurology, Stanford University, Stanford, California, USA. 8. Department Physical Medicine and Rehabilitation, University of California, Davis Medical Center, Sacramento, California, USA. 9. Department of Neurology, Harvard University, Boston Children's Hospital, Boston, Massachusetts, USA. 10. Division of Pediatric Neurology, University of Florida College of Medicine, Gainesville, Florida, USA. 11. Division of Biostatistics, Washington University School of Medicine, Saint Louis, Missouri, USA.
Abstract
INTRODUCTION: Outcomes sensitive to change over time in non-ambulatory boys/men with Duchenne muscular dystrophy (DMD) are not well-established. METHODS: Subjects (n = 91; 16.8 ± 4.5 years old) were assessed at baseline and 6-month intervals for 2 years. We analyzed all subjects using an intent-to-treat model and a subset of stronger subjects with Brooke Scale score ≤4, using repeated measures. RESULTS: Eight patients (12-33 years old) died during the study. Sixty-six completed 12-month follow-up, and 51 completed 24-month follow-up. Those taking corticosteroids performed better at baseline, but rates of decline were similar. Forced vital capacity percent predicted (FVC% predicted) declined significantly only after 2 years. However, Brooke and Egen Klassifikation (EK) Scale scores, elbow flexion, and grip strength declined significantly over both 1 and 2 years. CONCLUSION: Brooke and EK Scale scores, elbow flexion, and grip strength were outcomes most responsive to change. FVC% predicted was responsive to change over 2 years. Corticosteroids benefited non-ambulatory DMD subjects but did not affect decline rates of measures tested here. Muscle Nerve 54: 681-689, 2016.
INTRODUCTION: Outcomes sensitive to change over time in non-ambulatory boys/men with Duchenne muscular dystrophy (DMD) are not well-established. METHODS: Subjects (n = 91; 16.8 ± 4.5 years old) were assessed at baseline and 6-month intervals for 2 years. We analyzed all subjects using an intent-to-treat model and a subset of stronger subjects with Brooke Scale score ≤4, using repeated measures. RESULTS: Eight patients (12-33 years old) died during the study. Sixty-six completed 12-month follow-up, and 51 completed 24-month follow-up. Those taking corticosteroids performed better at baseline, but rates of decline were similar. Forced vital capacity percent predicted (FVC% predicted) declined significantly only after 2 years. However, Brooke and Egen Klassifikation (EK) Scale scores, elbow flexion, and grip strength declined significantly over both 1 and 2 years. CONCLUSION: Brooke and EK Scale scores, elbow flexion, and grip strength were outcomes most responsive to change. FVC% predicted was responsive to change over 2 years. Corticosteroids benefited non-ambulatory DMD subjects but did not affect decline rates of measures tested here. Muscle Nerve 54: 681-689, 2016.
Authors: David J Birnkrant; Katharine Bushby; Carla M Bann; Susan D Apkon; Angela Blackwell; David Brumbaugh; Laura E Case; Paula R Clemens; Stasia Hadjiyannakis; Shree Pandya; Natalie Street; Jean Tomezsko; Kathryn R Wagner; Leanne M Ward; David R Weber Journal: Lancet Neurol Date: 2018-02-03 Impact factor: 44.182
Authors: Craig M Zaidman; Jim S Wu; Kush Kapur; Amy Pasternak; Lavanya Madabusi; Sung Yim; Adam Pacheck; Heather Szelag; Tim Harrington; Basil T Darras; Seward B Rutkove Journal: Ann Neurol Date: 2017-05-04 Impact factor: 10.422
Authors: Lindsay N Alfano; Jay S Charleston; Anne M Connolly; Linda Cripe; Cas Donoghue; Robert Dracker; Johannes Dworzak; Helen Eliopoulos; Diane E Frank; Sarah Lewis; Karin Lucas; Jessie Lynch; A J Milici; Amy Flynt; Emily Naughton; Louise R Rodino-Klapac; Zarife Sahenk; Frederick J Schnell; G David Young; Jerry R Mendell; Linda P Lowes Journal: Medicine (Baltimore) Date: 2019-06 Impact factor: 1.817