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-Meinema; Christiaan Scott; Kjell Tullus; Michael William Beresford; cSLE T2T International Task Force

doi:10.1136/ard-2022-223328

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

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

Authors

Eve Mary Dorothy Smith^{1

2}, Amita Aggarwal³, Jenny Ainsworth^{4

2}, Eslam Al-Abadi⁵, Tadej Avcin⁶, Lynette Bortey⁷, Jon Burnham⁸, Coziana Ciurtin⁹, Christian M Hedrich^{4

2}, Sylvia Kamphuis¹⁰, Deborah M Levy^{11

12}, Laura B Lewandowski¹³, Naomi Maxwell⁷, Eric F Morand¹⁴, Seza Ozen¹⁵, Clare E Pain^{4

2}, Angelo Ravelli^{16

17}, Claudia Saad Magalhaes¹⁸, Clarissa A Pilkington¹⁹, Dieneke Schonenberg-Meinema^{20

21}, Christiaan Scott²², Kjell Tullus²³, Michael William Beresford^{4

2}; cSLE T2T International Task Force

Collaborators

cSLE T2T International Task Force:
Beatrice Goilav, Natasha Goss, Louise Oni, Stephen Marks

Affiliations

¹ Department of Women's & Children's Health, Institute of Life Course and Medical Sciences, University of Liverpool Faculty of Health and Life Sciences, Liverpool, UK esmith8@liverpool.ac.uk.
² Department of Paediatric Rheumatology, Alder Hey Children's NHS Foundation Trust, Liverpool, UK.
³ Department of Clinical Immunology & Rheumatology, Sanjay Gandhi Post Graduate Institute of Medical Sciences, Lucknow, Uttar Pradesh, India.
⁴ Department of Women's & Children's Health, Institute of Life Course and Medical Sciences, University of Liverpool Faculty of Health and Life Sciences, Liverpool, UK.
⁵ Department of Paediatric Rheumatology, Birmingham Children's Hospital NHS Foundation Trust, Birmingham, UK.
⁶ Department of Pediatric Rheumatology and Clinical Immunology, University Medical Centre Ljubljana Division of Paediatrics, Ljubljana, Slovenia.
⁷ TARGET Lupus Public Patient Involvement and Engagement Group, University of Liverpool Faculty of Health and Life Sciences, Liverpool, UK.
⁸ Department of Pediatric Rheumatology, The Children's Hospital of Philadelphia, Philadelphia, Pennsylvania, USA.
⁹ Centre for Adolescent Rheumatology Versus Arthritis, Division of Medicine, University College London, London, UK.
¹⁰ Department of Paediatric Rheumatology, Erasmus Medical Center, Rotterdam, The Netherlands.
¹¹ Department of Pediatric Rheumatology, The Hospital for Sick Children, Toronto, Ontario, Canada.
¹² Department of Pediatric Rheumatology, University of Toronto, Toronto, Ontario, Canada.
¹³ National Institutes of Health, National Institute of Arthritis and Musculoskeletal and Skin Diseases Systemic Autoimmunity Branch, Bethesda, Maryland, USA.
¹⁴ Department of Rheumatology, Monash University, Clayton, Victoria, Australia.
¹⁵ Department of Pediatrics, Division of Rheumatology, Hacettepe University Faculty of Medicine, Ankara, Turkey.
¹⁶ Direzione Scientifica, Istituto Giannina Gaslini Istituto Pediatrico di Ricovero e Cura a Carattere Scientifico, Genova, Liguria, Italy.
¹⁷ Dipartimento di Neuroscienze, Riabilitazione, Oftalmologia, Genetica e Scienze Materno Infantili (DINIGMI), Università degli Studi di Genova, Genova, Italy.
¹⁸ Department of Pediatric Rheumatology, Botucatu Medical School, Sao Paulo University Faculty of Medicine, Sao Paulo, Brazil.
¹⁹ Department of Paediatric Rheumatology, Great Ormond Street Hospital for Children, London, UK.
²⁰ Department of Pediatric Immunology, Rheumatology and Infectious Diseases, University of Amsterdam, Amsterdam, The Netherlands.
²¹ Emma Children's Hospital, Amsterdam University Medical Centres, Duivendrecht, The Netherlands.
²² Department of Paediatric Rheumatology, University of Cape Town, Rondebosch, Western Cape, South Africa.
²³ Department of Paediatric Nephrology, Great Ormond Street Hospital for Children, London, UK.

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.

Publication types

Systematic Review
Research Support, Non-U.S. Gov't

MeSH terms

Adult
Advisory Committees
Child
Humans
Lupus Erythematosus, Systemic* / drug therapy
Quality of Life*
Remission Induction
Surveys and Questionnaires

Abstract

Publication types

MeSH terms

Grants and funding