Work

Clinical trials in systemic sclerosis: lessons learned and outcomes

Year: 2007

Type: conference-abstract

Abstract: 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.

Source: Arthritis Research & Therapy

DOI: 10.1186/ar2191

Authors: Marco Matucci-Cerinic, Virginia D Steen, Daniel E Furst, James R Seibold

Institutions: University of Florence, Georgetown University Medical Center, University of California, Los Angeles, University of Michigan

Language: English

Topic: Systemic Sclerosis and Related Diseases

Subfield: Pathology and Forensic Medicine

Field: Medicine

Domain: Health Sciences

SDG: Good health and well-being

Open Access status: gold

Estimated APC paid: $3,690