Cargando…
Developing a provisional, international Minimal Dataset for Juvenile Dermatomyositis: for use in clinical practice to inform research
BACKGROUND: Juvenile dermatomyositis (JDM) is a rare but severe autoimmune inflammatory myositis of childhood. International collaboration is essential in order to undertake clinical trials, understand the disease and improve long-term outcome. The aim of this study was to propose from existing coll...
Autores principales: | , , , , , , , |
---|---|
Formato: | Online Artículo Texto |
Lenguaje: | English |
Publicado: |
BioMed Central
2014
|
Materias: | |
Acceso en línea: | https://www.ncbi.nlm.nih.gov/pmc/articles/PMC4113599/ https://www.ncbi.nlm.nih.gov/pubmed/25075205 http://dx.doi.org/10.1186/1546-0096-12-31 |
_version_ | 1782328311377035264 |
---|---|
author | McCann, Liza J Arnold, Katie Pilkington, Clarissa A Huber, Adam M Ravelli, Angelo Beard, Laura Beresford, Michael W Wedderburn, Lucy R |
author_facet | McCann, Liza J Arnold, Katie Pilkington, Clarissa A Huber, Adam M Ravelli, Angelo Beard, Laura Beresford, Michael W Wedderburn, Lucy R |
author_sort | McCann, Liza J |
collection | PubMed |
description | BACKGROUND: Juvenile dermatomyositis (JDM) is a rare but severe autoimmune inflammatory myositis of childhood. International collaboration is essential in order to undertake clinical trials, understand the disease and improve long-term outcome. The aim of this study was to propose from existing collaborative initiatives a preliminary minimal dataset for JDM. This will form the basis of the future development of an international consensus-approved minimum core dataset to be used both in clinical care and inform research, allowing integration of data between centres. METHODS: A working group of internationally-representative JDM experts was formed to develop a provisional minimal dataset. Clinical and laboratory variables contained within current national and international collaborative databases of patients with idiopathic inflammatory myopathies were scrutinised. Judgements were informed by published literature and a more detailed analysis of the Juvenile Dermatomyositis Cohort Biomarker Study and Repository, UK and Ireland. RESULTS: A provisional minimal JDM dataset has been produced, with an associated glossary of definitions. The provisional minimal dataset will request information at time of patient diagnosis and during on-going prospective follow up. At time of patient diagnosis, information will be requested on patient demographics, diagnostic criteria and treatments given prior to diagnosis. During on-going prospective follow-up, variables will include the presence of active muscle or skin disease, major organ involvement or constitutional symptoms, investigations, treatment, physician global assessments and patient reported outcome measures. CONCLUSIONS: An internationally agreed minimal dataset has the potential to significantly enhance collaboration, allow effective communication between groups, provide a minimal standard of care and enable analysis of the largest possible number of JDM patients to provide a greater understanding of this disease. This preliminary dataset can now be developed into a consensus-approved minimum core dataset and tested in a wider setting with the aim of achieving international agreement. |
format | Online Article Text |
id | pubmed-4113599 |
institution | National Center for Biotechnology Information |
language | English |
publishDate | 2014 |
publisher | BioMed Central |
record_format | MEDLINE/PubMed |
spelling | pubmed-41135992014-07-30 Developing a provisional, international Minimal Dataset for Juvenile Dermatomyositis: for use in clinical practice to inform research McCann, Liza J Arnold, Katie Pilkington, Clarissa A Huber, Adam M Ravelli, Angelo Beard, Laura Beresford, Michael W Wedderburn, Lucy R Pediatr Rheumatol Online J Research BACKGROUND: Juvenile dermatomyositis (JDM) is a rare but severe autoimmune inflammatory myositis of childhood. International collaboration is essential in order to undertake clinical trials, understand the disease and improve long-term outcome. The aim of this study was to propose from existing collaborative initiatives a preliminary minimal dataset for JDM. This will form the basis of the future development of an international consensus-approved minimum core dataset to be used both in clinical care and inform research, allowing integration of data between centres. METHODS: A working group of internationally-representative JDM experts was formed to develop a provisional minimal dataset. Clinical and laboratory variables contained within current national and international collaborative databases of patients with idiopathic inflammatory myopathies were scrutinised. Judgements were informed by published literature and a more detailed analysis of the Juvenile Dermatomyositis Cohort Biomarker Study and Repository, UK and Ireland. RESULTS: A provisional minimal JDM dataset has been produced, with an associated glossary of definitions. The provisional minimal dataset will request information at time of patient diagnosis and during on-going prospective follow up. At time of patient diagnosis, information will be requested on patient demographics, diagnostic criteria and treatments given prior to diagnosis. During on-going prospective follow-up, variables will include the presence of active muscle or skin disease, major organ involvement or constitutional symptoms, investigations, treatment, physician global assessments and patient reported outcome measures. CONCLUSIONS: An internationally agreed minimal dataset has the potential to significantly enhance collaboration, allow effective communication between groups, provide a minimal standard of care and enable analysis of the largest possible number of JDM patients to provide a greater understanding of this disease. This preliminary dataset can now be developed into a consensus-approved minimum core dataset and tested in a wider setting with the aim of achieving international agreement. BioMed Central 2014-07-21 /pmc/articles/PMC4113599/ /pubmed/25075205 http://dx.doi.org/10.1186/1546-0096-12-31 Text en Copyright © 2014 McCann et al.; licensee BioMed Central Ltd. http://creativecommons.org/licenses/by/4.0 This is an Open Access article distributed under the terms of the Creative Commons Attribution License (http://creativecommons.org/licenses/by/4.0), which permits unrestricted use, distribution, and reproduction in any medium, provided the original work is properly credited. The Creative Commons Public Domain Dedication waiver (http://creativecommons.org/publicdomain/zero/1.0/) applies to the data made available in this article, unless otherwise stated. |
spellingShingle | Research McCann, Liza J Arnold, Katie Pilkington, Clarissa A Huber, Adam M Ravelli, Angelo Beard, Laura Beresford, Michael W Wedderburn, Lucy R Developing a provisional, international Minimal Dataset for Juvenile Dermatomyositis: for use in clinical practice to inform research |
title | Developing a provisional, international Minimal Dataset for Juvenile Dermatomyositis: for use in clinical practice to inform research |
title_full | Developing a provisional, international Minimal Dataset for Juvenile Dermatomyositis: for use in clinical practice to inform research |
title_fullStr | Developing a provisional, international Minimal Dataset for Juvenile Dermatomyositis: for use in clinical practice to inform research |
title_full_unstemmed | Developing a provisional, international Minimal Dataset for Juvenile Dermatomyositis: for use in clinical practice to inform research |
title_short | Developing a provisional, international Minimal Dataset for Juvenile Dermatomyositis: for use in clinical practice to inform research |
title_sort | developing a provisional, international minimal dataset for juvenile dermatomyositis: for use in clinical practice to inform research |
topic | Research |
url | https://www.ncbi.nlm.nih.gov/pmc/articles/PMC4113599/ https://www.ncbi.nlm.nih.gov/pubmed/25075205 http://dx.doi.org/10.1186/1546-0096-12-31 |
work_keys_str_mv | AT mccannlizaj developingaprovisionalinternationalminimaldatasetforjuveniledermatomyositisforuseinclinicalpracticetoinformresearch AT arnoldkatie developingaprovisionalinternationalminimaldatasetforjuveniledermatomyositisforuseinclinicalpracticetoinformresearch AT pilkingtonclarissaa developingaprovisionalinternationalminimaldatasetforjuveniledermatomyositisforuseinclinicalpracticetoinformresearch AT huberadamm developingaprovisionalinternationalminimaldatasetforjuveniledermatomyositisforuseinclinicalpracticetoinformresearch AT ravelliangelo developingaprovisionalinternationalminimaldatasetforjuveniledermatomyositisforuseinclinicalpracticetoinformresearch AT beardlaura developingaprovisionalinternationalminimaldatasetforjuveniledermatomyositisforuseinclinicalpracticetoinformresearch AT beresfordmichaelw developingaprovisionalinternationalminimaldatasetforjuveniledermatomyositisforuseinclinicalpracticetoinformresearch AT wedderburnlucyr developingaprovisionalinternationalminimaldatasetforjuveniledermatomyositisforuseinclinicalpracticetoinformresearch |