eprintid: 10178243
rev_number: 8
eprint_status: archive
userid: 699
dir: disk0/10/17/82/43
datestamp: 2023-10-06 11:56:16
lastmod: 2024-11-02 07:10:06
status_changed: 2023-10-06 11:56:16
type: article
metadata_visibility: show
sword_depositor: 699
creators_name: Herrick, Ariane L
creators_name: Denton, Christopher P
title: Enrichment strategies for clinical trials targeting skin fibrosis and interstitial lung disease in systemic sclerosis
ispublished: pub
divisions: UCL
divisions: B02
divisions: C10
divisions: D17
divisions: G90
note: This version is the author accepted manuscript. For information on re-use, please refer to the publisher’s terms and conditions.
abstract: PURPOSE OF REVIEW: This review gives an update on enrichment strategies for clinical trials in patients with systemic sclerosis (SSc) in two contexts - skin fibrosis in early diffuse cutaneous disease, and SSc-related interstitial lung disease (ILD) - focusing on reports from the last 18 months. Lessons have been learnt from recent studies, making this review timely. RECENT FINDINGS: Recent trials have highlighted how patients included into trials must be carefully selected to include 'progressors', that is, those most likely to benefit from treatment, and how drug mechanism action of action will influence trial design. For skin fibrosis, current enrichment strategies are mainly on clinical grounds (including disease duration, extent of skin thickening, tendon friction rubs and anti-RNA polymerase III positivity). Gene expression signatures may play a role in the future. For ILD, current enrichment strategies (degree of lung involvement as assessed by pulmonary function and high-resolution computed tomography) may help to recruit the most informative patients, but should avoid being too stringent to be feasible or for findings to be generalizable. SUMMARY: Both skin fibrosis and ILD trials are challenging in SSc. Ongoing work on enrichment strategies should help to differentiate effective new treatments from placebo with smaller sample sizes than have been included in recent studies.
date: 2023-11-01
date_type: published
publisher: Ovid Technologies (Wolters Kluwer Health)
official_url: http://doi.org/10.1097/BOR.0000000000000976
oa_status: green
full_text_type: other
language: eng
primo: open
primo_central: open_green
verified: verified_manual
elements_id: 2093546
doi: 10.1097/BOR.0000000000000976
medium: Print-Electronic
pii: 00002281-990000000-00084
lyricists_name: Denton, Christopher
lyricists_id: CPDEN87
actors_name: Denton, Christopher
actors_id: CPDEN87
actors_role: owner
full_text_status: public
publication: Current Opinion in Rheumatology
volume: 35
number: 6
pagerange: 349-355
event_location: United States
issn: 1040-8711
citation:        Herrick, Ariane L;    Denton, Christopher P;      (2023)    Enrichment strategies for clinical trials targeting skin fibrosis and interstitial lung disease in systemic sclerosis.                   Current Opinion in Rheumatology , 35  (6)   pp. 349-355.    10.1097/BOR.0000000000000976 <https://doi.org/10.1097/BOR.0000000000000976>.       Green open access   
 
document_url: https://discovery.ucl.ac.uk/id/eprint/10178243/1/CurrOp_Skin-ILD-Herrick%20Denton-for%20UCL%20upload.pdf