UCL Discovery
UCL home » Library Services » Electronic resources » UCL Discovery

Challenges in systemic sclerosis trial design

Denton, CP; (2019) Challenges in systemic sclerosis trial design. Seminars in Arthritis and Rheumatism , 49 (3) S3-S7. 10.1016/j.semarthrit.2019.09.019. Green open access

[thumbnail of Denton-SSc trials-ATT 2019 article-final text in press for UCL.pdf]
Preview
Text
Denton-SSc trials-ATT 2019 article-final text in press for UCL.pdf - Accepted Version

Download (386kB) | Preview

Abstract

Systemic sclerosis (scleroderma; SSc) is an autoimmune rheumatic disease with high clinical burden and unmet need due to connective tissue fibrosis and vascular damage. It has the highest case specific mortality of any rheumatic disease, with approximately half of patients diagnosed eventually dying as a direct result of SSc. There are no approved diseases modifying treatments. This is partly related to the difficulty of conducting clinical trials for regulatory approval. Traditionally skin thickness has been assessed using the modified Rodnan skin score (MRSS) that has been shown to correlate with survival and risk of complications in SSc. However recent trials have highlighted the limitations of MRSS which often improves over time, even on placebo. A new composite measure integrating changes in multiple domains of lung function, skin, patients and physician global and HAQ disability index has been developed, the CRISS (Composite Response Index for Systemic Sclerosis). This measure looks promising and has provisional acceptance by American College of Rheumatology (designated ACR CRISS) but is unlikely to be strongly persuasive to Health Authorities in isolation unless there are also clinically meaningful changes in relevant domains that reflect how patients feel, function or survive.

Type: Article
Title: Challenges in systemic sclerosis trial design
Location: United States
Open access status: An open access version is available from UCL Discovery
DOI: 10.1016/j.semarthrit.2019.09.019
Publisher version: https://doi.org/10.1016/j.semarthrit.2019.09.019
Language: English
Additional information: This version is the author accepted manuscript. For information on re-use, please refer to the publisher’s terms and conditions.
Keywords: Clinical trial, Fibrosis, Scleroderma, Systemic sclerosis
UCL classification: UCL
UCL > Provost and Vice Provost Offices > School of Life and Medical Sciences
UCL > Provost and Vice Provost Offices > School of Life and Medical Sciences > Faculty of Medical Sciences
UCL > Provost and Vice Provost Offices > School of Life and Medical Sciences > Faculty of Medical Sciences > Div of Medicine
UCL > Provost and Vice Provost Offices > School of Life and Medical Sciences > Faculty of Medical Sciences > Div of Medicine > Inflammation
URI: https://discovery.ucl.ac.uk/id/eprint/10087766
Downloads since deposit
287Downloads
Download activity - last month
Download activity - last 12 months
Downloads by country - last 12 months

Archive Staff Only

View Item View Item