Clinical trial readiness in non-ambulatory boys and men with duchenne muscular dystrophy

MDA–DMD network follow-up

the MDA–DMD Clinical Research Network

Research output: Contribution to journalArticle

14 Citations (Scopus)

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.

Original languageEnglish (US)
Pages (from-to)681-689
Number of pages9
JournalMuscle and Nerve
Volume54
Issue number4
DOIs
StatePublished - Oct 1 2016

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Duchenne Muscular Dystrophy
Vital Capacity
Hand Strength
Elbow
Adrenal Cortex Hormones
Clinical Trials
Muscles

Keywords

  • Brooke Scale
  • clinical trial
  • Duchenne muscular dystrophy
  • Egen Klassifikation Scale
  • force vital capacity
  • non-ambulatory
  • outcomes

ASJC Scopus subject areas

  • Physiology
  • Clinical Neurology
  • Cellular and Molecular Neuroscience
  • Physiology (medical)

Cite this

Clinical trial readiness in non-ambulatory boys and men with duchenne muscular dystrophy : MDA–DMD network follow-up. / the MDA–DMD Clinical Research Network.

In: Muscle and Nerve, Vol. 54, No. 4, 01.10.2016, p. 681-689.

Research output: Contribution to journalArticle

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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.",
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