Common data elements for clinical research in mitochondrial disease

a National Institute for Neurological Disorders and Stroke project

on behalf of the Mito Working Group Member Participants

Research output: Contribution to journalArticle

5 Citations (Scopus)

Abstract

Objectives: 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. Methods: 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. Results: 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. Conclusion: 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.

Original languageEnglish (US)
Pages (from-to)403-414
Number of pages12
JournalJournal of Inherited Metabolic Disease
Volume40
Issue number3
DOIs
StatePublished - May 1 2017

Fingerprint

National Institute of Neurological Disorders and Stroke
Mitochondrial Diseases
Research
Common Data Elements
Information Dissemination
Natural History
Publications
Research Design
Research Personnel
Clinical Trials
Guidelines

ASJC Scopus subject areas

  • Genetics
  • Genetics(clinical)

Cite this

Common data elements for clinical research in mitochondrial disease : a National Institute for Neurological Disorders and Stroke project. / on behalf of the Mito Working Group Member Participants.

In: Journal of Inherited Metabolic Disease, Vol. 40, No. 3, 01.05.2017, p. 403-414.

Research output: Contribution to journalArticle

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title = "Common data elements for clinical research in mitochondrial disease: a National Institute for Neurological Disorders and Stroke project",
abstract = "Objectives: 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. Methods: 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. Results: 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. Conclusion: 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.",
author = "{on behalf of the Mito Working Group Member Participants} and Amel Karaa and Shamima Rahman and Anne Lomb{\`e}s and Patrick Yu-Wai-Man and Sheikh, {Muniza K.} and Sherita Alai-Hansen and Cohen, {Bruce H.} and David Dimmock and Lisa Emrick and Falk, {Marni J.} and Shana McCormack and David Mirsky and Tony Moore and Sumit Parikh and John Shoffner and Tanja Taivassalo and Mark Tarnopolsky and Ingrid Tein and Odenkirchen, {Joanne C.} and Amy Goldstein and Abdenur, {J. E.} and Vernon Anderson and Cristy Balcells and Mark Bamberger and Piero Barboni and Laurence Bindoff and Kathryn Camp and Valerio Carelli and Patrick Chinnery and Abigail Collins and Copeland, {William C.} and Kristin Fiorino and Xiaowu Gai and Kerry Goetz and Bret Goodpaster and Gropman, {Andrea L.} and Gwinn, {Katrina A.} and Ronald Haller and Heuckeroth, {Robert O.} and Michio Hirano and Holder, {Graham E.} and Petra Kaufmann and Kierstin Keller and Keltner, {John L.} and Keltner, {John L} and Thomas Klopstock and Saskia Koene and Koenig, {Mary Kay} and Yasutoshi Koga and Danuta Krotoski",
year = "2017",
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language = "English (US)",
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T1 - Common data elements for clinical research in mitochondrial disease

T2 - a National Institute for Neurological Disorders and Stroke project

AU - on behalf of the Mito Working Group Member Participants

AU - Karaa, Amel

AU - Rahman, Shamima

AU - Lombès, Anne

AU - Yu-Wai-Man, Patrick

AU - Sheikh, Muniza K.

AU - Alai-Hansen, Sherita

AU - Cohen, Bruce H.

AU - Dimmock, David

AU - Emrick, Lisa

AU - Falk, Marni J.

AU - McCormack, Shana

AU - Mirsky, David

AU - Moore, Tony

AU - Parikh, Sumit

AU - Shoffner, John

AU - Taivassalo, Tanja

AU - Tarnopolsky, Mark

AU - Tein, Ingrid

AU - Odenkirchen, Joanne C.

AU - Goldstein, Amy

AU - Abdenur, J. E.

AU - Anderson, Vernon

AU - Balcells, Cristy

AU - Bamberger, Mark

AU - Barboni, Piero

AU - Bindoff, Laurence

AU - Camp, Kathryn

AU - Carelli, Valerio

AU - Chinnery, Patrick

AU - Collins, Abigail

AU - Copeland, William C.

AU - Fiorino, Kristin

AU - Gai, Xiaowu

AU - Goetz, Kerry

AU - Goodpaster, Bret

AU - Gropman, Andrea L.

AU - Gwinn, Katrina A.

AU - Haller, Ronald

AU - Heuckeroth, Robert O.

AU - Hirano, Michio

AU - Holder, Graham E.

AU - Kaufmann, Petra

AU - Keller, Kierstin

AU - Keltner, John L.

AU - Keltner, John L

AU - Klopstock, Thomas

AU - Koene, Saskia

AU - Koenig, Mary Kay

AU - Koga, Yasutoshi

AU - Krotoski, Danuta

PY - 2017/5/1

Y1 - 2017/5/1

N2 - Objectives: 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. Methods: 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. Results: 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. Conclusion: 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.

AB - Objectives: 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. Methods: 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. Results: 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. Conclusion: 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.

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EP - 414

JO - Journal of Inherited Metabolic Disease

JF - Journal of Inherited Metabolic Disease

SN - 0141-8955

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