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Current Rheumatology Reports
Erschienen in: Current Rheumatology Reports 6/2016

01.06.2016 | Scleroderma (J Varga, Section Editor)

Clinical Trial Design Issues in Systemic Sclerosis: an Update

verfasst von: Jessica K. Gordon, Robyn T. Domsic

Erschienen in: Current Rheumatology Reports | Ausgabe 6/2016

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Abstract

Systemic sclerosis (scleroderma, SSc) is a multisystem disease characterized by vasculopathy, autoimmunity, and fibrosis. SSc has the highest disease-related mortality rate among the rheumatologic illnesses. In the USA, there remains no FDA-approved therapy. As our understanding of SSc pathogenesis improves, targeted therapies interrupting key pathways and mediators will be studied in clinical trials. However, clinical trials in SSc are fraught with challenges. Validated clinical outcome measures do not exist for all disease manifestations. It can be difficult to discern disease activity from damage. SSc is highly heterogeneous, with multiple different phenotypes, and predicting who will have progressive disease is not currently well understood. Biomarkers are in early stages of development and do not represent surrogate outcomes at this time. Given that SSc is uncommon, studies of similar disease aspects or populations can lead to competition for patients. This review will focus on current issues in SSc clinical trial design.
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Metadaten
Titel
Clinical Trial Design Issues in Systemic Sclerosis: an Update
verfasst von
Jessica K. Gordon
Robyn T. Domsic
Publikationsdatum
01.06.2016
Verlag
Springer US
Erschienen in
Current Rheumatology Reports / Ausgabe 6/2016
Print ISSN: 1523-3774
Elektronische ISSN: 1534-6307
DOI
https://doi.org/10.1007/s11926-016-0582-z

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