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

Eve Mary Dorothy Smith*, Amita Aggarwal, Jenny Ainsworth, Eslam Al-Abadi, Tadej Avcin, Lynette Bortey, Jon Burnham, Coziana Ciurtin, Christian M. Hedrich, Sylvia Kamphuis, Deborah M. Levy, Laura B. Lewandowski, Naomi Maxwell, Eric F. Morand, Seza Ozen, Clare E. Pain, Angelo Ravelli, Claudia Saad Magalhaes, Clarissa A. Pilkington, Dieneke Schonenberg-MeinemaChristiaan Scott, Kjell Tullus, Michael William Beresford

*Corresponding author for this work

Research output: Contribution to journalArticleAcademicpeer-review

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Abstract

Objectives 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. Methods 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. Results 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. Conclusions 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.

Original languageEnglish
Pages (from-to)788-798
Number of pages11
JournalAnnals of the Rheumatic Diseases
Volume82
Issue number6
Early online date11 May 2023
DOIs
Publication statusPublished - 2023

Bibliographical note

Funding Information:
This work was supported by the Wellcome Trust through a Wellcome Trust Institutional Strategic Support Fund (204822z16z), Equality and Diversity grant, awarded to EMDS by the Faculty of Health and Life Sciences, University of Liverpool’. It was also supported through an NIHR CRN: Children/Versus Arthritis Paediatric Rheumatology Clinical Studies Group grant, awarded to EMDS. The study took place as part of the UK’s ‘Experimental Arthritis Treatment Centre for Children’ supported by Versus Arthritis (grant number ARUK-20621), the University of Liverpool, Alder Hey Children’s NHS Foundation Trust and the Alder Hey Charity, and based at the University of Liverpool and Alder Hey Children’s NHS Foundation Trust. LBL is supported by the NIAMS Intramural Research Program.

Publisher Copyright:
© Author(s) (or their employer(s)) 2023. Re-use permitted under CC BY-NC. No commercial re-use. See rights and permissions. Published by BMJ.

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