International Cohort study of Juvenile Systemic Sclerosis
Research type
Research Study
Full title
International Cohort study of Juvenile Systemic Sclerosis
IRAS ID
256657
Contact name
Clare Pain
Contact email
Sponsor organisation
Alder Hey Childrens NHS Foundation Trust
Duration of Study in the UK
10 years, 0 months, 1 days
Research summary
Juvenile systemic sclerosis is an extremely rare condition in childhood with 4-5 new cases a year being diagnosed in the UK. There is very little published data on outcome in children.
Children and young people with juvenile systemic sclerosis are managed differently in different centres and countries. In particular, screening for heart and lung involvement is not standardised. This is important as heart and lung involvement may be silent and early detection can lead to effective treatments being started which may prevent death or improve quality of life.
This study has been running outside of the UK, since 2012 but has been modified to include all children (not just new cases) with juvenile systemic sclerosis AND to include those with possible early onset disease. The aims of the study are:
1) To assess patients in a standard way to help understand how children with this condition are affected for example, by heart or lung involvement, Raynaud's, fingertip ulcers or other organ involvement, how the condition affects function and quality of life.
2) To compare paediatric disease criteria with adult criteria, in particular whether newly developed adult criteria may lead to an earlier diagnosis.Patients and families have expressed that they want to know the prognosis of systemic sclerosis when they are diagnosed, as some children will have mild disease and others have life-threatening complications. We do not currently have any evidence to provide this information to families. We also do not know how best to treat or screen for complications. This study will help us understand this also.
The study will open in most tertiary Paediatric Rheumatology centres in the UK and will involve data commonly collected as part of routine clinical care plus some additional data such as quality of life questionnaires. Children will be followed up within their routine clinic appointments 6 monthly.
REC name
North West - Liverpool Central Research Ethics Committee
REC reference
19/NW/0429
Date of REC Opinion
17 Jul 2019
REC opinion
Favourable Opinion