PAtient STratification & Individualised trEatment: Systemic sclerosis

• Research type

  Research Study

• Full title

  PAtient STratification and Individualised trEatment in Systemic sclerosis - PASTIES

• IRAS ID

  319323

• Contact name

  Christian M. Hedrich

• Contact email

  chedrich@liverpool.ac.uk

• Sponsor organisation

  University of Liverpool

• Duration of Study in the UK

  2 years, 0 months, 2 days

• Research summary

  Systemic sclerosis (SSc) is a condition where an overactive immune system can cause inflammation, fibrosis (scar tissue) and blood vessel changes that cause symptoms in multiple parts of the body, impacting on patients’ lives. Children with SSc have told us they want a better understanding of why they have SSc, and better treatments. This study will increase our understanding of how to treat SSc across the world.

  Treatments for SSc have only partially been successful in trials in adults and, so far, have not involved children with this condition. Often patients must try different treatments before finding the right one. Being able to choose the right treatment for the right patient would enable switching off the disease and improving symptoms more rapidly, reducing damage and long-term complications from the disease.
  By analysing blood samples, we will look at the genetic code in people with this condition and investigate how important genes function in SSc. When we asked patients, they thought taking an extra blood sample along with routine bloods to do this study would be acceptable and of benefit to them and people like them with SSc. We will compare these findings with other information, such as how a patient is affected and which blood markers they have (antibodies against their own self), to find patterns which allow us to group patients who may be best treated with e.g., treatment A rather than B. These genetic studies may also help us to discover new treatments. We will work with our colleagues in the United States of America who have already identified certain genetic changes in children with SSc and collect samples on this rare condition from patients in the US, UK, The Netherland, Germany and Italy. Through working with our colleagues caring for adults, we can compare results from adults with SSc.

• REC name

  Wales REC 5

• REC reference

  23/WA/0088

• Date of REC Opinion

  23 Mar 2023

• REC opinion

  Favourable Opinion