Clinical trial readiness in non-ambulatory boys and men with duchenne muscular dystrophy: MDA-DMD network follow-up

Anne M Connolly^{1

2}, Julaine M Florence³, Craig M Zaidman^{3

4}, Paul T Golumbek^{3

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; MDA-DMD Clinical Research Network

Affiliations

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.

PMID: 26930423
DOI: 10.1002/mus.25089

Clinical trial readiness in non-ambulatory boys and men with duchenne muscular dystrophy: MDA-DMD network follow-up

Anne M Connolly et al. Muscle Nerve. 2016 Oct.

. 2016 Oct;54(4):681-9.

doi: 10.1002/mus.25089. Epub 2016 May 10.

Authors

Affiliations

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.

PMID: 26930423
DOI: 10.1002/mus.25089

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.

Keywords: Brooke Scale; Duchenne muscular dystrophy; Egen Klassifikation Scale; clinical trial; force vital capacity; non-ambulatory; outcomes.

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Clinical trial readiness in non-ambulatory boys and men with duchenne muscular dystrophy: MDA-DMD network follow-up

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Clinical trial readiness in non-ambulatory boys and men with duchenne muscular dystrophy: MDA-DMD network follow-up

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