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. 2023 Jun;82(6):788-798.
doi: 10.1136/ard-2022-223328. Epub 2023 Jan 10.

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  1   2 , Amita Aggarwal  3 , Jenny Ainsworth  4   2 , Eslam Al-Abadi  5 , Tadej Avcin  6 , Lynette Bortey  7 , Jon Burnham  8 , Coziana Ciurtin  9 , Christian M Hedrich  4   2 , Sylvia Kamphuis  10 , Deborah M Levy  11   12 , Laura B Lewandowski  13 , Naomi Maxwell  7 , Eric F Morand  14 , Seza Ozen  15 , Clare E Pain  4   2 , Angelo Ravelli  16   17 , Claudia Saad Magalhaes  18 , Clarissa A Pilkington  19 , Dieneke Schonenberg-Meinema  20   21 , Christiaan Scott  22 , Kjell Tullus  23 , Michael William Beresford  4   2 ; cSLE T2T International Task Force
Collaborators, Affiliations

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 et al. Ann Rheum Dis. 2023 Jun.

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.

Keywords: Autoimmune Diseases; Glucocorticoids; Health services research; Lupus Erythematosus, Systemic; Outcome and Process Assessment, Health Care.

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Conflict of interest statement

Competing interests: None declared.

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