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Facioscapulohumeral muscular dystrophy functional composite outcome measure.
Eichinger, Katy; Heatwole, Chad; Iyadurai, Stanley; King, Wendy; Baker, Lindsay; Heininger, Susanne; Bartlett, Amy; Dilek, Nuran; Martens, William B; Mcdermott, Michael; Kissel, John T; Tawil, Rabi; Statland, Jeffrey M.
Afiliación
  • Eichinger K; Department of Neurology, University of Rochester Medical Center, 601 Elmwood Avenue, Box 673 Rochester, New York, 14642, USA.
  • Heatwole C; Department of Neurology, University of Rochester Medical Center, 601 Elmwood Avenue, Box 673 Rochester, New York, 14642, USA.
  • Iyadurai S; Department of Neurology, The Ohio State University Wexner Medical Center, Columbus, Ohio, USA.
  • King W; Department of Neurology, The Ohio State University Wexner Medical Center, Columbus, Ohio, USA.
  • Baker L; Department of Neurology, University of Rochester Medical Center, 601 Elmwood Avenue, Box 673 Rochester, New York, 14642, USA.
  • Heininger S; Department of Neurology, University of Rochester Medical Center, 601 Elmwood Avenue, Box 673 Rochester, New York, 14642, USA.
  • Bartlett A; Department of Neurology, The Ohio State University Wexner Medical Center, Columbus, Ohio, USA.
  • Dilek N; Department of Neurology, University of Rochester Medical Center, 601 Elmwood Avenue, Box 673 Rochester, New York, 14642, USA.
  • Martens WB; Department of Neurology, University of Rochester Medical Center, 601 Elmwood Avenue, Box 673 Rochester, New York, 14642, USA.
  • Mcdermott M; Department of Neurology, University of Rochester Medical Center, 601 Elmwood Avenue, Box 673 Rochester, New York, 14642, USA.
  • Kissel JT; Department of Biostatistics and Computational Biology, University of Rochester Medical Center, Rochester, New York, USA.
  • Tawil R; Department of Neurology, The Ohio State University Wexner Medical Center, Columbus, Ohio, USA.
  • Statland JM; Department of Neurology, University of Rochester Medical Center, 601 Elmwood Avenue, Box 673 Rochester, New York, 14642, USA.
Muscle Nerve ; 2018 Jan 30.
Article en En | MEDLINE | ID: mdl-29381807
ABSTRACT

INTRODUCTION:

We developed an evaluator-administered functional facioscapulohumeral muscular dystrophy composite outcome measure (FSHD-COM) comprising patient-identified areas of functional burden for future clinical trials.

METHODS:

We performed a prospective observational study of 41 patients with FSHD at 2 sites. The FSHD-COM includes functional assessment of the legs, shoulders and arms, trunk, hands, and balance/mobility. We determined the test-retest reliability and convergent validity compared to established FSHD disease metrics.

RESULTS:

The FSHD-COM demonstrated excellent test-retest reliability (intraclass correlation coefficient [ICC] 0.96; subscale ICC range, 0.90-0.94). Cross-sectional associations between the FSHD-COM and disease duration, clinical severity, and strength were moderate to strong (Pearson correlation coefficient range |0.51-0.92|).

DISCUSSION:

The FSHD-COM is a disease-relevant, functional composite outcome measure suitable for future FSHD clinical trials that shows excellent test-retest reliability and cross-sectional associations to disease measures. Future directions include determining multisite reliability, sensitivity to change, and the minimal clinically important change in the FSHD-COM. Muscle Nerve, 2018.
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Texto completo: 1 Colección: 01-internacional Base de datos: MEDLINE Tipo de estudio: Observational_studies Idioma: En Revista: Muscle Nerve Año: 2018 Tipo del documento: Article País de afiliación: Estados Unidos
Texto completo
Añadir a Mi BVS
Imprimir
XML
PubMed Links
Buscar en Google

Texto completo: 1 Colección: 01-internacional Base de datos: MEDLINE Tipo de estudio: Observational_studies Idioma: En Revista: Muscle Nerve Año: 2018 Tipo del documento: Article País de afiliación: Estados Unidos