eprintid: 10087766
rev_number: 18
eprint_status: archive
userid: 608
dir: disk0/10/08/77/66
datestamp: 2019-12-11 11:27:13
lastmod: 2021-09-26 23:13:04
status_changed: 2019-12-11 11:27:13
type: article
metadata_visibility: show
creators_name: Denton, CP
title: Challenges in systemic sclerosis trial design
ispublished: pub
subjects: RFH
divisions: UCL
divisions: B02
divisions: C10
divisions: D17
divisions: G90
keywords: Clinical trial, Fibrosis, Scleroderma, Systemic sclerosis
note: This version is the author accepted manuscript. For information on re-use, please refer to the publisher’s terms and conditions.
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.
date: 2019-12
date_type: published
official_url: https://doi.org/10.1016/j.semarthrit.2019.09.019
oa_status: green
full_text_type: other
language: eng
primo: open
primo_central: open_green
verified: verified_manual
elements_id: 1724861
doi: 10.1016/j.semarthrit.2019.09.019
pii: S0049-0172(19)30654-7
lyricists_name: Denton, Christopher
lyricists_id: CPDEN87
actors_name: Denton, Christopher
actors_id: CPDEN87
actors_role: owner
full_text_status: public
publication: Seminars in Arthritis and Rheumatism
volume: 49
number: 3
pagerange: S3-S7
event_location: United States
citation:        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 <https://doi.org/10.1016/j.semarthrit.2019.09.019>.       Green open access   
 
document_url: https://discovery.ucl.ac.uk/id/eprint/10087766/1/Denton-SSc%20trials-ATT%202019%20article-final%20text%20in%20press%20for%20UCL.pdf