Winner of the 2019 PReS/PRINTO call for grants, the study aims to formally evaluate the psychometric properties of the LoSQI in a European sample of patients with JLS.
Collaboration between PRINTO centres, CARRA and PRES scleroderma working party members in partnership with patients and families will facilitate shared aims and this will be the first multi-national study of a disease-specific patient-reported outcome (PRO) in JLS.
In order to combine datasets and results with the overarching aim to embed PRO in routine clinical practice, the study will allow cohorts from multiple countries.
BACKGROUND and RATIONALE
Juvenile localised scleroderma (JLS) is characterised by chronic inflammation within the skin and tissues leading to fibrosis. It is associated with significant complications including joint contractures, limb length discrepancy and facial atrophy that impact quality of life. Patient reported outcomes (PRO) are not well established within research settings and are not part of routine clinical care in many centres.
Several studies have measured health-related quality of life (HRQoL) in JLS, most commonly using the Children’s Dermatology Life Quality Index. This measure only captures impact of skin involvement and not HRQoL from extra-cutaneous manifestations. Psychometric analysis shows that it incompletely measures important concepts of HRQoL that are unique to JLS. The Localised Scleroderma Quality of Life Instrument (LoSQI) has been developed in partnership with patients and families to capture aspects of disease which may not be well defined within generic QoL measures. The initial development and validation process was iterative, patient-centred, and consistent with best practices in PRO development. Currently, the LoSQI is the only JLS-specific PRO, and the only PRO that includes both qualitative and quantitative validity evidence. It is currently being utilized within two large American scleroderma registries but will require cross-cultural adaptation for international use.
The study is formed by two Workstreams, the Cross-cultural adaptation of LoSQI via methods previously described by Guillemin et al, with pre-testing in selected study population (WS1) and the validation of LoSQI via a multicentre prospective cohort study of 100 patients (WS2).
Study PI: Dr Clare Pain