Literature DB >> 23239403

Common data elements for clinical research in Friedreich's ataxia.

David R Lynch1, Massimo Pandolfo, Jorg B Schulz, Susan Perlman, Martin B Delatycki, R Mark Payne, Robert Shaddy, Kenneth H Fischbeck, Jennifer Farmer, Paul Kantor, Subha V Raman, Lisa Hunegs, Joanne Odenkirchen, Kristy Miller, Petra Kaufmann.   

Abstract

To reduce study start-up time, increase data sharing, and assist investigators conducting clinical studies, the National Institute of Neurological Disorders and Stroke embarked on an initiative to create common data elements for neuroscience clinical research. The Common Data Element Team developed general common data elements, which are commonly collected in clinical studies regardless of therapeutic area, such as demographics. In the present project, we applied such approaches to data collection in Friedreich's ataxia (FRDA), a neurological disorder that involves multiple organ systems. To develop FRDA common data elements, FRDA experts formed a working group and subgroups to define elements in the following: ataxia and performance measures; biomarkers; cardiac and other clinical outcomes; and demographics, laboratory tests, and medical history. The basic development process included identification of international experts in FRDA clinical research, meeting by teleconference to develop a draft of standardized common data elements recommendations, vetting of recommendations across the subgroups, and dissemination of recommendations to the research community for public comment. The full recommendations were published online in September 2011 at http://www.commondataelements.ninds.nih.gov/FA.aspx. The subgroups' recommendations are classified as core, supplemental, or exploratory. Template case report forms were created for many of the core tests. The present set of data elements should ideally lead to decreased initiation time for clinical research studies and greater ability to compare and analyze data across studies. Their incorporation into new, ongoing studies will be assessed in an ongoing fashion to define their utility in FRDA.
Copyright © 2012 Movement Disorders Society.

Entities:  

Mesh:

Substances:

Year:  2012        PMID: 23239403      PMCID: PMC3581713          DOI: 10.1002/mds.25201

Source DB:  PubMed          Journal:  Mov Disord        ISSN: 0885-3185            Impact factor:   10.338


  32 in total

Review 1.  Friedreich ataxia: effects of genetic understanding on clinical evaluation and therapy.

Authors:  David R Lynch; Jennifer M Farmer; Laura J Balcer; Robert B Wilson
Journal:  Arch Neurol       Date:  2002-05

2.  Common data elements for pediatric traumatic brain injury: recommendations from the working group on demographics and clinical assessment.

Authors:  P David Adelson; Jose Pineda; Michael J Bell; Nicholas S Abend; Rachel P Berger; Christopher C Giza; Gillian Hotz; Mark S Wainwright
Journal:  J Neurotrauma       Date:  2011-11-07       Impact factor: 5.269

3.  Aconitase and mitochondrial iron-sulphur protein deficiency in Friedreich ataxia.

Authors:  A Rötig; P de Lonlay; D Chretien; F Foury; M Koenig; D Sidi; A Munnich; P Rustin
Journal:  Nat Genet       Date:  1997-10       Impact factor: 38.330

4.  International Cooperative Ataxia Rating Scale (ICARS): appropriate for studies of Friedreich's ataxia?

Authors:  Stefan J Cano; Jeremy C Hobart; Paul E Hart; L V Prasad Korlipara; Anthony H V Schapira; J Mark Cooper
Journal:  Mov Disord       Date:  2005-12       Impact factor: 10.338

5.  Performance measures in Friedreich ataxia: potential utility as clinical outcome tools.

Authors:  David R Lynch; Jennifer M Farmer; Robert L Wilson; Laura J Balcer
Journal:  Mov Disord       Date:  2005-07       Impact factor: 10.338

6.  Glucose metabolism alterations in Friedreich's ataxia.

Authors:  G Finocchiaro; G Baio; P Micossi; G Pozza; S di Donato
Journal:  Neurology       Date:  1988-08       Impact factor: 9.910

7.  Regulation of mitochondrial iron accumulation by Yfh1p, a putative homolog of frataxin.

Authors:  M Babcock; D de Silva; R Oaks; S Davis-Kaplan; S Jiralerspong; L Montermini; M Pandolfo; J Kaplan
Journal:  Science       Date:  1997-06-13       Impact factor: 47.728

8.  Glucose intolerance in Friedreich's ataxia: association with insulin resistance and decreased insulin binding.

Authors:  R J Khan; E Andermann; I G Fantus
Journal:  Metabolism       Date:  1986-11       Impact factor: 8.694

9.  Friedreich's ataxia: a clinical and genetic study of 90 families with an analysis of early diagnostic criteria and intrafamilial clustering of clinical features.

Authors:  A E Harding
Journal:  Brain       Date:  1981-09       Impact factor: 13.501

10.  Evidence for abnormal regulation of insulin receptors in Friedreich's ataxia.

Authors:  I G Fantus; M H Seni; E Andermann
Journal:  J Clin Endocrinol Metab       Date:  1993-01       Impact factor: 5.958

View more
  10 in total

Review 1.  Common data elements for spinal cord injury clinical research: a National Institute for Neurological Disorders and Stroke project.

Authors:  F Biering-Sørensen; S Alai; K Anderson; S Charlifue; Y Chen; M DeVivo; A E Flanders; L Jones; N Kleitman; A Lans; V K Noonan; J Odenkirchen; J Steeves; K Tansey; E Widerström-Noga; L B Jakeman
Journal:  Spinal Cord       Date:  2015-02-10       Impact factor: 2.772

2.  Adaptive semantic tag mining from heterogeneous clinical research texts.

Authors:  T Hao; C Weng
Journal:  Methods Inf Med       Date:  2014-10-20       Impact factor: 2.176

3.  Common Data Elements for Muscle Biopsy Reporting.

Authors:  Jahannaz Dastgir; Anne Rutkowski; Rachel Alvarez; Stacy A Cossette; Ke Yan; Raymond G Hoffmann; Caroline Sewry; Yukiko K Hayashi; Hans-Hilmar Goebel; Carsten Bonnemann; Michael W Lawlor
Journal:  Arch Pathol Lab Med       Date:  2015-07-01       Impact factor: 5.534

4.  Report of a workshop on research gaps in the treatment of cerebral palsy.

Authors:  Codrin Lungu; Deborah Hirtz; Diane Damiano; Paul Gross; Jonathan W Mink
Journal:  Neurology       Date:  2016-08-24       Impact factor: 9.910

5.  Establishment and Maintenance of Primary Fibroblast Repositories for Rare Diseases-Friedreich's Ataxia Example.

Authors:  Yanjie Li; Urszula Polak; Amanda D Clark; Angela D Bhalla; Yu-Yun Chen; Jixue Li; Jennifer Farmer; Lauren Seyer; David Lynch; Jill S Butler; Marek Napierala
Journal:  Biopreserv Biobank       Date:  2016-03-22       Impact factor: 2.300

6.  Establishment of Kawasaki disease database based on metadata standard.

Authors:  Yu Rang Park; Jae-Jung Kim; Young Jo Yoon; Young-Kwang Yoon; Ha Yeong Koo; Young Mi Hong; Gi Young Jang; Soo-Yong Shin; Jong-Keuk Lee
Journal:  Database (Oxford)       Date:  2016-07       Impact factor: 3.451

7.  An approach for transgender population information extraction and summarization from clinical trial text.

Authors:  Boyu Chen; Hao Jin; Zhiwen Yang; Yingying Qu; Heng Weng; Tianyong Hao
Journal:  BMC Med Inform Decis Mak       Date:  2019-04-09       Impact factor: 2.796

8.  Measuring peripheral nerve involvement in Friedreich's ataxia.

Authors:  Peter D Creigh; Joan Mountain; Janet E Sowden; Katy Eichinger; Bernard Ravina; Jane Larkindale; David N Herrmann
Journal:  Ann Clin Transl Neurol       Date:  2019-08-15       Impact factor: 4.511

Review 9.  Standardized Assessment of Hereditary Ataxia Patients in Clinical Studies.

Authors:  Brigitte K Paap; Sandra Roeske; Alexandra Durr; Ludger Schöls; Tetsuo Ashizawa; Sylvia Boesch; Lisa M Bunn; Martin B Delatycki; Paola Giunti; Stéphane Lehéricy; Caterina Mariotti; Jörg Melegh; Massimo Pandolfo; Chantal M E Tallaksen; Dagmar Timmann; Shoji Tsuji; Jörg Bela Schulz; Bart P van de Warrenburg; Thomas Klockgether
Journal:  Mov Disord Clin Pract       Date:  2016-02-11

10.  Iron Hack - A symposium/hackathon focused on porphyrias, Friedreich's ataxia, and other rare iron-related diseases.

Authors:  Gloria C Ferreira; Jenna Oberstaller; Renée Fonseca; Thomas E Keller; Swamy Rakesh Adapa; Justin Gibbons; Chengqi Wang; Xiaoming Liu; Chang Li; Minh Pham; Guy W Dayhoff Ii; Ben Busby; Rays H Y Jiang; Linh M Duong; Luis Tañón Reyes; Luciano Enrique Laratelli; Douglas Franz; Segun Fatumo; Atm Golam Bari; Audrey Freischel; Lindsey Fiedler; Omkar Dokur; Krishna Sharma; Deborah Cragun
Journal:  F1000Res       Date:  2019-07-19
  10 in total

北京卡尤迪生物科技股份有限公司 © 2022-2023.