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

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

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

^*Corresponding author for this work

Pediatrics

University of Liverpool
Alder Hey Children's NHS Foundation Trust
Sanjay Gandhi Postgraduate Institute of Medical Sciences
Birmingham Women's and Children's NHS Foundation Trust
Ljubljanski Univerzitetni Klinicni Center
Children's Hospital of Philadelphia
University College London
The Hospital for Sick Children
University of Toronto
National Institutes of Health
Monash University
Hacettepe University
IRCCS Istituto Giannina Gaslini - Genova
University of Genoa
Universidade de São Paulo
Great Ormond Street Hospital for Children NHS Foundation Trust
University of Amsterdam
University of Cape Town

Research output: Contribution to journal › Article › Academic › peer-review

44 Citations (Scopus)

51 Downloads (Pure)

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 language	English
Pages (from-to)	788-798
Number of pages	11
Journal	Annals of the Rheumatic Diseases
Volume	82
Issue number	6
Early online date	11 May 2023
DOIs	https://doi.org/10.1136/ard-2022-223328
Publication status	Published - 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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SDG 3 Good Health and Well-being

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Cite this

Smith, E. M. D., Aggarwal, A., Ainsworth, J., Al-Abadi, E., Avcin, T., Bortey, L., Burnham, J., Ciurtin, C., Hedrich, C. M., Kamphuis, S., Levy, D. M., Lewandowski, L. B., Maxwell, N., Morand, E. F., Ozen, S., Pain, C. E., Ravelli, A., Saad Magalhaes, C., Pilkington, C. A., ... Beresford, M. W. (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), 788-798. https://doi.org/10.1136/ard-2022-223328

@article{8a60b7a9ca76436ca28c169aec62a539,

title = "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",

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.",

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

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{\textquoteright}. 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{\textquoteright}s {\textquoteleft}Experimental Arthritis Treatment Centre for Children{\textquoteright} supported by Versus Arthritis (grant number ARUK-20621), the University of Liverpool, Alder Hey Children{\textquoteright}s NHS Foundation Trust and the Alder Hey Charity, and based at the University of Liverpool and Alder Hey Children{\textquoteright}s NHS Foundation Trust. LBL is supported by the NIAMS Intramural Research Program. Publisher Copyright: {\textcopyright} 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.",

year = "2023",

doi = "10.1136/ard-2022-223328",

language = "English",

volume = "82",

pages = "788--798",

journal = "Annals of the Rheumatic Diseases",

issn = "0003-4967",

publisher = "Elsevier",

number = "6",

}

Smith, EMD, Aggarwal, A, Ainsworth, J, Al-Abadi, E, Avcin, T, Bortey, L, Burnham, J, Ciurtin, C, Hedrich, CM, Kamphuis, S, Levy, DM, Lewandowski, LB, Maxwell, N, Morand, EF, Ozen, S, Pain, CE, Ravelli, A, Saad Magalhaes, C, Pilkington, CA, Schonenberg-Meinema, D, Scott, C, Tullus, K & Beresford, MW 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, vol. 82, no. 6, pp. 788-798. https://doi.org/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. / Smith, Eve Mary Dorothy; Aggarwal, Amita; Ainsworth, Jenny et al.
In: Annals of the Rheumatic Diseases, Vol. 82, No. 6, 2023, p. 788-798.

Research output: Contribution to journal › Article › Academic › peer-review

TY - JOUR

T1 - Towards development of treat to target (T2T) in childhood-onset systemic lupus erythematosus

T2 - PReS-endorsed overarching principles and points-to-consider from an international task force

AU - Smith, Eve Mary Dorothy

AU - Aggarwal, Amita

AU - Ainsworth, Jenny

AU - Al-Abadi, Eslam

AU - Avcin, Tadej

AU - Bortey, Lynette

AU - Burnham, Jon

AU - Ciurtin, Coziana

AU - Hedrich, Christian M.

AU - Kamphuis, Sylvia

AU - Levy, Deborah M.

AU - Lewandowski, Laura B.

AU - Maxwell, Naomi

AU - Morand, Eric F.

AU - Ozen, Seza

AU - Pain, Clare E.

AU - Ravelli, Angelo

AU - Saad Magalhaes, Claudia

AU - Pilkington, Clarissa A.

AU - Schonenberg-Meinema, Dieneke

AU - Scott, Christiaan

AU - Tullus, Kjell

AU - Beresford, Michael William

N1 - 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.

PY - 2023

Y1 - 2023

N2 - 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.

AB - 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.

UR - https://www.scopus.com/pages/publications/85152199008

U2 - 10.1136/ard-2022-223328

DO - 10.1136/ard-2022-223328

M3 - Article

C2 - 36627168

AN - SCOPUS:85152199008

SN - 0003-4967

VL - 82

SP - 788

EP - 798

JO - Annals of the Rheumatic Diseases

JF - Annals of the Rheumatic Diseases

IS - 6

ER -

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

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