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Clinical trials in systemic sclerosis: lessons learned and outcomes
The pathogenesis of systemic sclerosis (SSc) is complex and largely unclear. The clinical heterogeneity of the disease and its progression over a number of years makes the choice of endpoints in the design of clinical trials difficult. The overwhelming need in this disease is to diagnose it early an...
Autores principales: | , , , |
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Formato: | Texto |
Lenguaje: | English |
Publicado: |
BioMed Central|1
2007
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Materias: | |
Acceso en línea: | https://www.ncbi.nlm.nih.gov/pmc/articles/PMC2072887/ https://www.ncbi.nlm.nih.gov/pubmed/17767745 http://dx.doi.org/10.1186/ar2191 |
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author | Matucci-Cerinic, Marco Steen, Virginia D Furst, Daniel E Seibold, James R |
author_facet | Matucci-Cerinic, Marco Steen, Virginia D Furst, Daniel E Seibold, James R |
author_sort | Matucci-Cerinic, Marco |
collection | PubMed |
description | The pathogenesis of systemic sclerosis (SSc) is complex and largely unclear. The clinical heterogeneity of the disease and its progression over a number of years makes the choice of endpoints in the design of clinical trials difficult. The overwhelming need in this disease is to diagnose it early and identify those patients who will benefit most from early, aggressive treatment that potentially can alter the clinical disease course. To achieve this, innumerable challenges must be overcome. This article reviews data from recent clinical trials and the lessons derived from retrospective observational studies, databases, and patient registries. Taken together, these observations will help to improve our understanding of the diverse clinical course of SSc and permit refinement of existing outcome measures for the design of future clinical trials, in which the likelihood of observing a positive treatment effect with the drugs at our disposal will be maximized. |
format | Text |
id | pubmed-2072887 |
institution | National Center for Biotechnology Information |
language | English |
publishDate | 2007 |
publisher | BioMed Central|1 |
record_format | MEDLINE/PubMed |
spelling | pubmed-20728872007-11-10 Clinical trials in systemic sclerosis: lessons learned and outcomes Matucci-Cerinic, Marco Steen, Virginia D Furst, Daniel E Seibold, James R Arthritis Res Ther Review The pathogenesis of systemic sclerosis (SSc) is complex and largely unclear. The clinical heterogeneity of the disease and its progression over a number of years makes the choice of endpoints in the design of clinical trials difficult. The overwhelming need in this disease is to diagnose it early and identify those patients who will benefit most from early, aggressive treatment that potentially can alter the clinical disease course. To achieve this, innumerable challenges must be overcome. This article reviews data from recent clinical trials and the lessons derived from retrospective observational studies, databases, and patient registries. Taken together, these observations will help to improve our understanding of the diverse clinical course of SSc and permit refinement of existing outcome measures for the design of future clinical trials, in which the likelihood of observing a positive treatment effect with the drugs at our disposal will be maximized. BioMed Central|1 2007 2007-08-15 /pmc/articles/PMC2072887/ /pubmed/17767745 http://dx.doi.org/10.1186/ar2191 Text en Copyright © 2007 BioMed Central Ltd |
spellingShingle | Review Matucci-Cerinic, Marco Steen, Virginia D Furst, Daniel E Seibold, James R Clinical trials in systemic sclerosis: lessons learned and outcomes |
title | Clinical trials in systemic sclerosis: lessons learned and outcomes |
title_full | Clinical trials in systemic sclerosis: lessons learned and outcomes |
title_fullStr | Clinical trials in systemic sclerosis: lessons learned and outcomes |
title_full_unstemmed | Clinical trials in systemic sclerosis: lessons learned and outcomes |
title_short | Clinical trials in systemic sclerosis: lessons learned and outcomes |
title_sort | clinical trials in systemic sclerosis: lessons learned and outcomes |
topic | Review |
url | https://www.ncbi.nlm.nih.gov/pmc/articles/PMC2072887/ https://www.ncbi.nlm.nih.gov/pubmed/17767745 http://dx.doi.org/10.1186/ar2191 |
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