title: Common data elements for clinical research in mitochondrial disease: a National Institute for Neurological Disorders and Stroke project.
abstract: The common data elements (CDE) project was developed by the National Institute of Neurological Disorders and Stroke (NINDS) to provide clinical researchers with tools to improve data quality and allow for harmonization of data collected in different research studies. CDEs have been created for several neurological diseases; the aim of this project was to develop CDEs specifically curated for mitochondrial disease (Mito) to enhance clinical research.
  
  
  
  Nine working groups (WGs), composed of international mitochondrial disease experts, provided recommendations for Mito clinical research. They initially reviewed existing NINDS CDEs and instruments, and developed new data elements or instruments when needed. Recommendations were organized, internally reviewed by the Mito WGs, and posted online for external public comment for a period of eight weeks. The final version was again reviewed by all WGs and the NINDS CDE team prior to posting for public use.
  
  
  
  The NINDS Mito CDEs and supporting documents are publicly available on the NINDS CDE website ( https://commondataelements.ninds.nih.gov/ ), organized into domain categories such as Participant/Subject Characteristics, Assessments, and Examinations.
  
  
  
  We developed a comprehensive set of CDE recommendations, data definitions, case report forms (CRFs), and guidelines for use in Mito clinical research. The widespread use of CDEs is intended to enhance Mito clinical research endeavors, including natural history studies, clinical trial design, and data sharing. Ongoing international collaboration will facilitate regular review, updates and online publication of Mito CDEs, and support improved consistency of data collection and reporting.
authors.affiliation: Massachusetts General Hospital, Boston, MA, USA.
authors.forename: Amel
authors.initials: A
authors.lastname: Karaa
authors.affiliation: UCL Great Ormond Street Institute of Child Health, London, UK.
authors.forename: Shamima
authors.initials: S
authors.lastname: Rahman
authors.affiliation: INSERM, Institut Cochin U1016, Paris, France.
authors.forename: Anne
authors.initials: A
authors.lastname: Lombès
authors.affiliation: Wellcome Trust Centre for Mitochondrial Research, Newcastle University, Newcastle upon Tyne, UK.|NIHR Biomedical Research Centre at Moorfields Eye Hospital, London, UK.|UCL Institute of Ophthalmology, London, UK.
authors.forename: Patrick
authors.initials: P
authors.lastname: Yu-Wai-Man
authors.affiliation: The Emmes Corporation, Rockville, MD, USA.
authors.forename: Muniza K
authors.initials: MK
authors.lastname: Sheikh
authors.affiliation: The Emmes Corporation, Rockville, MD, USA.
authors.forename: Sherita
authors.initials: S
authors.lastname: Alai-Hansen
authors.affiliation: Akron Children's Hospital, Akron, OH, USA.
authors.forename: Bruce H
authors.initials: BH
authors.lastname: Cohen
authors.affiliation: Medical College of Wisconsin, Milwaukee, WI, USA.
authors.forename: David
authors.initials: D
authors.lastname: Dimmock
authors.affiliation: Baylor College of Medicine, Houston, TX, USA.
authors.forename: Lisa
authors.initials: L
authors.lastname: Emrick
authors.affiliation: Children's Hospital of Philadelphia, Philadelphia, PA, USA.|University of Pennsylvania Perelman School of Medicine, Philadelphia, PA, USA.
authors.forename: Marni J
authors.initials: MJ
authors.lastname: Falk
authors.affiliation: Children's Hospital of Philadelphia, Philadelphia, PA, USA.|University of Pennsylvania Perelman School of Medicine, Philadelphia, PA, USA.
authors.forename: Shana
authors.initials: S
authors.lastname: McCormack
authors.affiliation: Children's Hospital Colorado, Aurora, CO, USA.
authors.forename: David
authors.initials: D
authors.lastname: Mirsky
authors.affiliation: Institute of Ophthalmology, University College London, London, UK.|Moorfields Eye Hospital, London, UK.|Department of Ophthalmology, University of California, San Francisco, USA.
authors.forename: Tony
authors.initials: T
authors.lastname: Moore
authors.affiliation: Cleveland Clinic, Cleveland, OH, USA.
authors.forename: Sumit
authors.initials: S
authors.lastname: Parikh
authors.affiliation: , Atlanta, GA, USA.
authors.forename: John
authors.initials: J
authors.lastname: Shoffner
authors.affiliation: McGill University, Montreal, CA, USA.
authors.forename: Tanja
authors.initials: T
authors.lastname: Taivassalo
authors.affiliation: McMaster University, Hamilton, ON, Canada.
authors.forename: Mark
authors.initials: M
authors.lastname: Tarnopolsky
authors.affiliation: Hospital for Sick Children, University of Toronto, Toronto, ON, Canada.
authors.forename: Ingrid
authors.initials: I
authors.lastname: Tein
authors.affiliation: National Institute of Neurological Disorders and Stroke, National Institutes of Health, Bethesda, MD, USA.
authors.forename: Joanne C
authors.initials: JC
authors.lastname: Odenkirchen
authors.affiliation: Children's Hospital of Pittsburgh, 4401 Penn Avenue, Pittsburgh, PA, 15224, USA. dramygoldstein@gmail.com.
authors.forename: Amy
authors.identifier: 0000-0002-7675-3063
authors.initials: A
authors.lastname: Goldstein
mesh_terms: D035843:Biomedical Research / Q000592:standards; D065709:Common Data Elements / Q000592:standards*; D003625:Data Collection / Q000592:standards; D006801:Humans; D028361:Mitochondrial Diseases / Q000473:pathology*; D055825:National Institute of Neurological Disorders and Stroke (U.S.); D009422:Nervous System Diseases / Q000473:pathology*; D012107:Research Design / Q000592:standards; D020521:Stroke / Q000473:pathology*; D014481:United States