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

Detalhes bibliográficos
Autor(a) principal: Smith, Eve Mary Dorothy
Data de Publicação: 2023
Outros Autores: Aggarwal, Amita, Ainsworth, Jenny, Al-Abadi, Eslam, Avcin, Tadej, Bortey, Lynette, Burnham, Jon, Ciurtin, Coziana, Hedrich, Christian M, Kamphuis, Sylvia, Levy, Deborah M, Lewandowski, Laura B, Maxwell, Naomi, Morand, Eric F, Ozen, Seza, Pain, Clare E, Ravelli, Angelo, Saad Magalhaes, Claudia [UNESP], Pilkington, Clarissa A, Schonenberg-Meinema, Dieneke, Scott, Christiaan, Tullus, Kjell, Beresford, Michael William
Tipo de documento: Artigo
Idioma: eng
Título da fonte: Repositório Institucional da UNESP
Texto Completo: http://dx.doi.org/10.1136/ard-2022-223328
http://hdl.handle.net/11449/248668
Resumo: 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.
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spelling 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 forceAutoimmune DiseasesGlucocorticoidsHealth services researchLupus Erythematosus, SystemicOutcome and Process Assessment, Health CareObjectives 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.Department of Women's & Children's Health Institute of Life Course and Medical Sciences University of Liverpool Faculty of Health and Life SciencesDepartment of Paediatric Rheumatology Alder Hey Children's Nhs Foundation TrustDepartment of Clinical Immunology & Rheumatology Sanjay Gandhi Post Graduate Institute of Medical Sciences, Uttar PradeshDepartment of Paediatric Rheumatology Birmingham Children's Hospital Nhs Foundation TrustDepartment of Pediatric Rheumatology and Clinical Immunology University Medical Centre Ljubljana Division of PaediatricsTarget Lupus Public Patient Involvement and Engagement Group University of Liverpool Faculty of Health and Life SciencesDepartment of Pediatric Rheumatology The Children's Hospital of PhiladelphiaCentre for Adolescent Rheumatology Versus Arthritis Division of Medicine University College LondonDepartment of Paediatric Rheumatology Erasmus Medical CenterDepartment of Pediatric Rheumatology The Hospital for Sick ChildrenDepartment of Pediatric Rheumatology University of TorontoNational Institutes of Health National Institute of Arthritis and Musculoskeletal and Skin Diseases Systemic Autoimmunity BranchDepartment of Rheumatology Monash UniversityDepartment of Pediatrics Division of Rheumatology Hacettepe University Faculty of MedicineDirezione Scientifica Istituto Giannina Gaslini Istituto Pediatrico di Ricovero e Cura A Carattere Scientifico, LiguriaDipto. di Neuroscienze Riabilitazione Oftalmologia Genetica e Scienze Materno Infantili (DINIGMI) Università Degli Studi di GenovaDepartment of Pediatric Rheumatology Botucatu Medical School Sao Paulo University Faculty of MedicineDepartment of Paediatric Rheumatology Great Ormond Street Hospital for ChildrenDepartment of Pediatric Immunology Rheumatology and Infectious Diseases University of AmsterdamEmma Children's Hospital Amsterdam University Medical CentresDepartment of Paediatric Rheumatology University of Cape Town, Western CapeDepartment of Paediatric Nephrology Great Ormond Street Hospital for ChildrenDepartment of Pediatric Rheumatology Botucatu Medical School Sao Paulo University Faculty of MedicineUniversity of Liverpool Faculty of Health and Life SciencesAlder Hey Children's Nhs Foundation TrustSanjay Gandhi Post Graduate Institute of Medical SciencesBirmingham Children's Hospital Nhs Foundation TrustDivision of PaediatricsThe Children's Hospital of PhiladelphiaUniversity College LondonErasmus Medical CenterThe Hospital for Sick ChildrenUniversity of TorontoNational Institute of Arthritis and Musculoskeletal and Skin Diseases Systemic Autoimmunity BranchMonash UniversityHacettepe University Faculty of MedicineIstituto Giannina Gaslini Istituto Pediatrico di Ricovero e Cura A Carattere ScientificoUniversità Degli Studi di GenovaUniversidade Estadual Paulista (UNESP)Great Ormond Street Hospital for ChildrenUniversity of AmsterdamAmsterdam University Medical CentresUniversity of Cape TownSmith, Eve Mary DorothyAggarwal, AmitaAinsworth, JennyAl-Abadi, EslamAvcin, TadejBortey, LynetteBurnham, JonCiurtin, CozianaHedrich, Christian MKamphuis, SylviaLevy, Deborah MLewandowski, Laura BMaxwell, NaomiMorand, Eric FOzen, SezaPain, Clare ERavelli, AngeloSaad Magalhaes, Claudia [UNESP]Pilkington, Clarissa ASchonenberg-Meinema, DienekeScott, ChristiaanTullus, KjellBeresford, Michael William2023-07-29T13:50:17Z2023-07-29T13:50:17Z2023-06-01info:eu-repo/semantics/publishedVersioninfo:eu-repo/semantics/article788-798http://dx.doi.org/10.1136/ard-2022-223328Annals of the Rheumatic Diseases, v. 82, n. 6, p. 788-798, 2023.1468-20600003-4967http://hdl.handle.net/11449/24866810.1136/ard-2022-2233282-s2.0-85152199008Scopusreponame:Repositório Institucional da UNESPinstname:Universidade Estadual Paulista (UNESP)instacron:UNESPengAnnals of the Rheumatic Diseasesinfo:eu-repo/semantics/openAccess2024-09-03T13:47:04Zoai:repositorio.unesp.br:11449/248668Repositório InstitucionalPUBhttp://repositorio.unesp.br/oai/requestrepositoriounesp@unesp.bropendoar:29462024-09-03T13:47:04Repositório Institucional da UNESP - Universidade Estadual Paulista (UNESP)false
dc.title.none.fl_str_mv 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
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
spellingShingle 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
Autoimmune Diseases
Glucocorticoids
Health services research
Lupus Erythematosus, Systemic
Outcome and Process Assessment, Health Care
title_short 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
title_full 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
title_fullStr 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
title_full_unstemmed 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
title_sort 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
author Smith, Eve Mary Dorothy
author_facet Smith, Eve Mary Dorothy
Aggarwal, Amita
Ainsworth, Jenny
Al-Abadi, Eslam
Avcin, Tadej
Bortey, Lynette
Burnham, Jon
Ciurtin, Coziana
Hedrich, Christian M
Kamphuis, Sylvia
Levy, Deborah M
Lewandowski, Laura B
Maxwell, Naomi
Morand, Eric F
Ozen, Seza
Pain, Clare E
Ravelli, Angelo
Saad Magalhaes, Claudia [UNESP]
Pilkington, Clarissa A
Schonenberg-Meinema, Dieneke
Scott, Christiaan
Tullus, Kjell
Beresford, Michael William
author_role author
author2 Aggarwal, Amita
Ainsworth, Jenny
Al-Abadi, Eslam
Avcin, Tadej
Bortey, Lynette
Burnham, Jon
Ciurtin, Coziana
Hedrich, Christian M
Kamphuis, Sylvia
Levy, Deborah M
Lewandowski, Laura B
Maxwell, Naomi
Morand, Eric F
Ozen, Seza
Pain, Clare E
Ravelli, Angelo
Saad Magalhaes, Claudia [UNESP]
Pilkington, Clarissa A
Schonenberg-Meinema, Dieneke
Scott, Christiaan
Tullus, Kjell
Beresford, Michael William
author2_role author
author
author
author
author
author
author
author
author
author
author
author
author
author
author
author
author
author
author
author
author
author
dc.contributor.none.fl_str_mv University of Liverpool Faculty of Health and Life Sciences
Alder Hey Children's Nhs Foundation Trust
Sanjay Gandhi Post Graduate Institute of Medical Sciences
Birmingham Children's Hospital Nhs Foundation Trust
Division of Paediatrics
The Children's Hospital of Philadelphia
University College London
Erasmus Medical Center
The Hospital for Sick Children
University of Toronto
National Institute of Arthritis and Musculoskeletal and Skin Diseases Systemic Autoimmunity Branch
Monash University
Hacettepe University Faculty of Medicine
Istituto Giannina Gaslini Istituto Pediatrico di Ricovero e Cura A Carattere Scientifico
Università Degli Studi di Genova
Universidade Estadual Paulista (UNESP)
Great Ormond Street Hospital for Children
University of Amsterdam
Amsterdam University Medical Centres
University of Cape Town
dc.contributor.author.fl_str_mv Smith, Eve Mary Dorothy
Aggarwal, Amita
Ainsworth, Jenny
Al-Abadi, Eslam
Avcin, Tadej
Bortey, Lynette
Burnham, Jon
Ciurtin, Coziana
Hedrich, Christian M
Kamphuis, Sylvia
Levy, Deborah M
Lewandowski, Laura B
Maxwell, Naomi
Morand, Eric F
Ozen, Seza
Pain, Clare E
Ravelli, Angelo
Saad Magalhaes, Claudia [UNESP]
Pilkington, Clarissa A
Schonenberg-Meinema, Dieneke
Scott, Christiaan
Tullus, Kjell
Beresford, Michael William
dc.subject.por.fl_str_mv Autoimmune Diseases
Glucocorticoids
Health services research
Lupus Erythematosus, Systemic
Outcome and Process Assessment, Health Care
topic Autoimmune Diseases
Glucocorticoids
Health services research
Lupus Erythematosus, Systemic
Outcome and Process Assessment, Health Care
description 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.
publishDate 2023
dc.date.none.fl_str_mv 2023-07-29T13:50:17Z
2023-07-29T13:50:17Z
2023-06-01
dc.type.status.fl_str_mv info:eu-repo/semantics/publishedVersion
dc.type.driver.fl_str_mv info:eu-repo/semantics/article
format article
status_str publishedVersion
dc.identifier.uri.fl_str_mv http://dx.doi.org/10.1136/ard-2022-223328
Annals of the Rheumatic Diseases, v. 82, n. 6, p. 788-798, 2023.
1468-2060
0003-4967
http://hdl.handle.net/11449/248668
10.1136/ard-2022-223328
2-s2.0-85152199008
url http://dx.doi.org/10.1136/ard-2022-223328
http://hdl.handle.net/11449/248668
identifier_str_mv Annals of the Rheumatic Diseases, v. 82, n. 6, p. 788-798, 2023.
1468-2060
0003-4967
10.1136/ard-2022-223328
2-s2.0-85152199008
dc.language.iso.fl_str_mv eng
language eng
dc.relation.none.fl_str_mv Annals of the Rheumatic Diseases
dc.rights.driver.fl_str_mv info:eu-repo/semantics/openAccess
eu_rights_str_mv openAccess
dc.format.none.fl_str_mv 788-798
dc.source.none.fl_str_mv Scopus
reponame:Repositório Institucional da UNESP
instname:Universidade Estadual Paulista (UNESP)
instacron:UNESP
instname_str Universidade Estadual Paulista (UNESP)
instacron_str UNESP
institution UNESP
reponame_str Repositório Institucional da UNESP
collection Repositório Institucional da UNESP
repository.name.fl_str_mv Repositório Institucional da UNESP - Universidade Estadual Paulista (UNESP)
repository.mail.fl_str_mv repositoriounesp@unesp.br
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