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Clinical trial readiness in non-ambulatory boys and men with duchenne muscular dystrophy: MDA-DMD network follow-up.
Connolly, Anne M; Florence, Julaine M; Zaidman, Craig M; Golumbek, Paul T; Mendell, Jerry R; Flanigan, Kevin M; Karachunski, Peter I; Day, John W; McDonald, Craig M; Darras, Basil T; Kang, Peter B; Siener, Catherine A; Gadeken, Rebecca K; Anand, Pallavi; Schierbecker, Jeanine R; Malkus, Elizabeth C; Lowes, Linda P; Alfano, Lindsay N; Johnson, Linda; Nicorici, Alina; Kelecic, Jason M; Quigley, Janet; Pasternak, Amy E; Miller, J Philip.
Afiliação
  • Connolly AM; Department of Neurology, Washington University School of Medicine, Saint Louis, Missouri, USA. connollya@neuro.wustl.edu.
  • Florence JM; Department of Pediatrics, Washington University School of Medicine, Saint Louis, Missouri, USA. connollya@neuro.wustl.edu.
  • Zaidman CM; Department of Neurology, Washington University School of Medicine, Saint Louis, Missouri, USA.
  • Golumbek PT; Department of Neurology, Washington University School of Medicine, Saint Louis, Missouri, USA.
  • Mendell JR; Department of Pediatrics, Washington University School of Medicine, Saint Louis, Missouri, USA.
  • Flanigan KM; Department of Neurology, Washington University School of Medicine, Saint Louis, Missouri, USA.
  • Karachunski PI; Department of Pediatrics, Washington University School of Medicine, Saint Louis, Missouri, USA.
  • Day JW; Department of Pediatrics, Ohio State University, and the Center for Gene Therapy, Nationwide Children's Hospital, Columbus, Ohio, USA.
  • McDonald CM; Department of Pediatrics, Ohio State University, and the Center for Gene Therapy, Nationwide Children's Hospital, Columbus, Ohio, USA.
  • Darras BT; Department of Neurology, University of Minnesota, Minneapolis, Minnesota, USA.
  • Kang PB; Department of Neurology, Stanford University, Stanford, California, USA.
  • Siener CA; Department Physical Medicine and Rehabilitation, University of California, Davis Medical Center, Sacramento, California, USA.
  • Gadeken RK; Department of Neurology, Harvard University, Boston Children's Hospital, Boston, Massachusetts, USA.
  • Anand P; Division of Pediatric Neurology, University of Florida College of Medicine, Gainesville, Florida, USA.
  • Schierbecker JR; Department of Neurology, Washington University School of Medicine, Saint Louis, Missouri, USA.
  • Malkus EC; Department of Neurology, Washington University School of Medicine, Saint Louis, Missouri, USA.
  • Lowes LP; Department of Neurology, Washington University School of Medicine, Saint Louis, Missouri, USA.
  • Alfano LN; Department of Neurology, Washington University School of Medicine, Saint Louis, Missouri, USA.
  • Johnson L; Department of Neurology, Washington University School of Medicine, Saint Louis, Missouri, USA.
  • Nicorici A; Department of Pediatrics, Ohio State University, and the Center for Gene Therapy, Nationwide Children's Hospital, Columbus, Ohio, USA.
  • Kelecic JM; Department of Pediatrics, Ohio State University, and the Center for Gene Therapy, Nationwide Children's Hospital, Columbus, Ohio, USA.
  • Quigley J; Department Physical Medicine and Rehabilitation, University of California, Davis Medical Center, Sacramento, California, USA.
  • Pasternak AE; Department Physical Medicine and Rehabilitation, University of California, Davis Medical Center, Sacramento, California, USA.
  • Miller JP; Department of Neurology, University of Minnesota, Minneapolis, Minnesota, USA.
Muscle Nerve ; 54(4): 681-9, 2016 10.
Article em En | MEDLINE | ID: mdl-26930423
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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Texto completo: 1 Coleções: 01-internacional Base de dados: MEDLINE Tipo de estudo: Observational_studies / Prognostic_studies / Risk_factors_studies Limite: Adolescent / Adult / Child / Humans / Male Idioma: En Ano de publicação: 2016 Tipo de documento: Article
Texto completo
Imprimir
XML
PubMed Links
Buscar no Google

Texto completo: 1 Coleções: 01-internacional Base de dados: MEDLINE Tipo de estudo: Observational_studies / Prognostic_studies / Risk_factors_studies Limite: Adolescent / Adult / Child / Humans / Male Idioma: En Ano de publicação: 2016 Tipo de documento: Article