We sought to explore patient and parental views on treatment targets, outcome measures and study designs being considered for a future juvenile-onset systemic lupus erythematosus (JSLE) treat to target (T2T) study.Topic guided, semi-structured interviews with JSLE patients/parents. Analysis of audio recorded interviews using thematic approaches.Patients and parents differed regarding symptoms they felt would be tolerable, representing 'low disease activity'. Patients often classed symptoms that they had previously experienced, were 'invisible' or had minimal disruption on their life as signs of low disease activity. Parents were more accepting of visible signs, but were concerned about potential organ involvement and symptom severity. Overall, patients and parents preferred that children were entirely asymptomatic, with no on-going treatment side-effects. They regarded fatigue as particularly challenging, requiring proper monitoring using a fatigue patient reported outcome measure. Most families felt that reducing corticosteroids would also be a good treatment target. Overall, families liked the concept of T2T, commenting that it could help to improve disease control, structure treatment, improve communication with clinicians and treatment compliance. They were concerned that T2T might increase the frequency of hospital visits, thus impacting upon schooling, parental employment, and finances. Families made suggestions on how to modify the future trial design to mitigate such effects.This study provides guidance from patients/parents on T2T targets and study designs. Complementary quantitative studies assessing the achievability and impact of different targets (e.g. LLDAS or remission) are now warranted, to inform an international consensus process to agree treatment targets.
Eve M D Smith, Sarah L Gorst, Eslam Al-Abadi, Daniel P Hawley, Valentina Leone, Clarissa Pilkington, Athimalaipet V Ramanan, Satyapal Rangaraj, Arani Sridhar, Michael W Beresford, Bridget Young