Towards development of treat to target (T2T) in childhood-onset systemic lupus erythematosus: PReS-endorsed overarching principles and points-to-consider from an international task force



Smith, Eve Mary Dorothy, Aggarwal, Amita, Ainsworth, Jenny, Al-Abadi, Eslam, Avcin, Tadej, Bortey, Lynette, Burnham, Jon, Ciurtin, Coziana, Hedrich, Christian M ORCID: 0000-0002-1295-6179, Kamphuis, Sylvia
et al (show 13 more authors) (2023) Towards development of treat to target (T2T) in childhood-onset systemic lupus erythematosus: PReS-endorsed overarching principles and points-to-consider from an international task force. ANNALS OF THE RHEUMATIC DISEASES, 82 (6). pp. 788-798.

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Abstract

<h4>Objectives</h4>Application of 'treat-to-target' (T2T) in childhood-onset systemic lupus erythematosus (cSLE) may improve care and health outcomes. This initiative aimed to harmonise existing evidence and expert opinion regarding T2T for cSLE.<h4>Methods</h4>An international T2T Task Force was formed of specialists in paediatric rheumatology, paediatric nephrology, adult rheumatology, patient and parent representatives. A steering committee formulated a set of draft overarching principles and points-to-consider, based on evidence from systematic literature review. Two on-line preconsensus meeting Delphi surveys explored healthcare professionals' views on these provisional overarching principles and points-to-consider. A virtual consensus meeting employed a modified nominal group technique to discuss, modify and vote on each overarching principle/point-to-consider. Agreement of >80% of Task Force members was considered consensus.<h4>Results</h4>The Task Force agreed on four overarching principles and fourteen points-to-consider. It was agreed that both treatment targets and therapeutic strategies should be subject to shared decision making with the patient/caregivers, with full remission the preferred target, and low disease activity acceptable where remission cannot be achieved. Important elements of the points-to-consider included: aiming for prevention of flare and organ damage; glucocorticoid sparing; proactively addressing factors that impact health-related quality of life (fatigue, pain, mental health, educational challenges, medication side effects); and aiming for maintenance of the target over the long-term. An extensive research agenda was also formulated.<h4>Conclusions</h4>These international, consensus agreed overarching principles and points-to-consider for T2T in cSLE lay the foundation for future T2T approaches in cSLE, endorsed by the Paediatric Rheumatology European Society.

Item Type: Article
Uncontrolled Keywords: Lupus Erythematosus, Systemic, Health services research, Glucocorticoids, Autoimmune Diseases, Outcome and Process Assessment, Health Care
Divisions: Faculty of Health and Life Sciences
Faculty of Health and Life Sciences > Institute of Life Courses and Medical Sciences
Depositing User: Symplectic Admin
Date Deposited: 17 Jan 2023 15:44
Last Modified: 11 Aug 2023 12:01
DOI: 10.1136/ard-2022-223328
Open Access URL: http://dx.doi.org/10.1136/ard-2022-223328
Related URLs:
URI: https://livrepository.liverpool.ac.uk/id/eprint/3167103