Facioscapulohumeral Muscular Dystrophy

Data Standards

The National Institute of Neurological Disorders and Stroke (NINDS), other Federal agencies, and international organizations have the common mission of developing data standards for clinical research. Through the efforts of subject-specific working groups, topic-driven data elements have been created. The first set of Common Data Elements (CDEs) for Facioscapulohumeral Muscular Dystrophy was developed in 2014-2015. The Core CDEs to be used by an investigator when beginning a research study in this disease/disorder are listed in the Start-up Resource Listing.

Many of the CDEs will overlap across study types, which allows for comparisons and meta-analyses across studies. Consistency of the data elements and the CDE formats is kept in order to ensure the ability to transfer critical medical information electronically from one center to another. This consistency also allows for continuity across different disease areas. The goals of the NINDS CDE initiative are to increase the efficiency and effectiveness of clinical research studies and clinical treatment, increase data quality, facilitate data sharing and help educate new clinical investigators.

Organized by domains and sub-domains, often used in clinical studies, data standards include:

An overview of all Facioscapulohumeral Muscular Dystrophy CDE recommendations can be found in the Facioscapulohumeral Muscular Dystrophy Disease CDE Highlight Summary document. For your reference, a zip file containing all the current Facioscapulohumeral Muscular Dystrophy CDE template CRF modules can be downloaded below.

The outline that follows includes all the CDEs associated with the CRF modules, organized by domain and sub-domain.

Overview of Working Group Recommendations
Summary Documents
CRF Module/Guideline CDEs
Summary of Recommendations for FSHD N/A
Participant Characteristics
Demographics
CRF Module/Guideline CDEs
General Core CDE Details
Demographics CDE Details
Social Status
CRF Module/Guideline CDEs
Social Status CDE Details
Participant History and Family History
Epidemiology/Environmental History
CRF Module/Guideline CDEs
World Health Organization Disability Assessment Schedule (WHODAS) N/A
General Health History
CRF Module/Guideline CDEs
Family History CDE Details
Medical History CDE Details
Pregnancy CDE Details
Assessments and Examinations
Physical/Neurological Examination
CRF Module/Guideline CDEs
Electrical Impedance Myography (EIM) N/A
Polysomnography CDE Details
Non-Imaging Diagnostics
CRF Module/Guideline CDEs
Electrocardiogram (ECG) CDE Details
Treatment/Intervention Data
Therapies
CRF Module/Guideline CDEs
Respiratory Interventions CDE Details
Drugs
CRF Module/Guideline CDEs
Prior and Concomitant Medications CDE Details

Overview

To develop the Facioscapulohumeral Muscular Dystrophy (FSHD) CDEs, the FSHD CDE Working Group met from April 2014 to October 2014. The recommendations were posted for public comment in November 2014 and version 1.0 was released at the end of February 2015.

History and Acknowledgements

The Facioscapulohumeral Muscular Dystrophy (FSHD) CDE Working Group has developed the CDEs for their recommendations. The FSHD CDE Working Group is supported by the NINDS CDE Team. The Working Group began meeting by teleconference in May 2014 and held calls every four to six weeks to define the CDEs for their domains and to recommend standardized, validated instruments for FSHD research.

Complete Facioscapulohumeral Muscular Dystrophy (FSHD) CDE Roster

The complete FSHD CDE Working Group roster is shown below.

FSHD Working Group

  • John T. Kissel, MD
    Ohio State University, Columbus, Ohio
  • George W.A.M. Padberg, MD
    Nijmegen, Netherlands
  • Enzo Ricci, MD
    Universita Cattolica del Sacro Cuore, Rome, Italy
  • Sabrina Sacconi, MD
    Centre Hopitalier Universitaire de Nice Sophia Antipolis, Nice, France
  • Jeffrey Statland, MD
    University of Kansas Medical Center, Kansas
  • Rabi Tawil, MD
    University of Rochester Medical Center, Rochester, New York
  • Baziel G. M. van Engelen, MD
    Neuromuscular Centre Nijmegen, Nijmegen, Netherlands
  • Kathryn R. Wagner, MD, PhD
    Kennedy Krieger Institute, Baltimore, Maryland

NIH Coordinating Team

  • Robin Conwit, MD
    National Institute of Neurological Disorders and Stroke (NINDS), National Institutes of Health (NIH), Bethesda, MD, USA
  • Elizabeth McNeil, MD, MSc
    National Institute of Neurological Disorders and Stroke (NINDS), National Institutes of Health (NIH), Bethesda, Maryland
  • Glen Nuckolls, PhD
    National Institute of Neurological Disorders and Stroke (NINDS), National Institutes of Health (NIH), Bethesda, Maryland
  • Joanne Odenkirchen, MPH (Until 2016)
    NINDS CDE Project Officer, National Institute of Neurological Disorders and Stroke (NINDS), National Institutes of Health (NIH), Bethesda, MD, USA
  • Yaffa Rubenstein, PhD
    National Center for Advancing Translational Sciences (NCATS), National Institutes of Health (NIH), Bethesda, Maryland
  • Tiina Urv, PhD
    National Institute of Child Health and Human Development (NICHD), National Institutes of Health (NIH), Bethesda, Maryland

NINDS CDE Team

  • Sherita Ala'I, MS
    The Emmes Corporation
  • Robin Feldman, BS, MBA
    The Emmes Corporation
  • Muniza Sheikh, MS, MBA
    The Emmes Corporation

References

A publication related to the Facioscapulohumeral muscular dystrophy (FSHD) CDE initiative is in development. The citation for this publication will be included on this site as soon as it is available.

Updates

Please see attached revision history document. Please contact NINDSCDE@emmes.com if you require further information or have any questions about the revision history.

Facioscapulohumeral CDE Revision History Document