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Development of CARRA/PReS-endorsed consensus Core and Expanded Datasets in childhood-onset systemic lupus erythematosus for international registry-based research
  1. Rebecca E Sadun1,
  2. Jennifer C Cooper2,
  3. Alexandre Belot3,
  4. Tadej Avcin4,
  5. Amita Aggarwal5,
  6. Jenny Ainsworth6,7,
  7. Alisha Akinsete8,
  8. Stacy P Ardoin9,
  9. Michael W Beresford6,7,
  10. Lynette Bortey10,
  11. Hermine I Brunner11,
  12. Joyce C Chang12,
  13. Coziana Ciurtin13,
  14. Ashley Daftary14,
  15. Barbara Eberhard15,
  16. Candace H Feldman16,
  17. Christian M Hedrich6,7,
  18. Aimee O Hersh17,
  19. Linda T Hiraki18,19,
  20. David A Isenberg13,
  21. Sylvia Kamphuis20,
  22. Andrea M Knight18,19,
  23. Lou Lambert10,
  24. Deborah M Levy18,19,
  25. Stephen D Marks21,22,
  26. Naomi Maxwell10,
  27. Angela Migowa23,
  28. Katharine Moore2,
  29. Seza Ozen24,
  30. Rosalind Ramsey-Goldman25,
  31. Angelo Ravelli26,
  32. Bryce B Reeve27,
  33. Tamar B Rubinstein28,
  34. Claudia Saad-Magalhaes29,
  35. Sujata Sawhney30,
  36. Laura E Schanberg31,
  37. Emily von Scheven32,
  38. Christiaan Scott33,34,
  39. Mary Beth Son12,
  40. Gladys Tony35,
  41. Elissa R Weitzman12,
  42. Scott E Wenderfer36,37,
  43. Alisha Woodside35,
  44. Laura B Lewandowski38,
  45. Eve MD Smith6,7
  46. the CARRA SLE Committee and PReS Lupus Working Party
  1. 1Departments of Medicine and Pediatrics, Duke University School of Medicine, Durham, North Carolina, USA
  2. 2Department of Pediatrics, University of Colorado Anschutz Medical Campus, Aurora, Colorado, USA
  3. 3Service de Pediatrie, Centre Hospitalier Universitaire de Lyon, Lyon, France
  4. 4Department of Allergology, Rheumatology and Clinical Immunology, University Medical Centre, Ljubljana, Slovenia
  5. 5Department of Clinical Immunology and Rheumatology, Sanjay Gandhi Post Graduate Institute of Medical Sciences, Lucknow, Uttar Pradesh, India
  6. 6Department of Women’s & Children’s Health, University of Liverpool, Liverpool, UK
  7. 7Department of Paediatric Rheumatology, Alder Hey Children's NHS Foundation Trust, Liverpool, UK
  8. 8Department of Pediatrics, Hospital for Special Surgery, New York, New York, USA
  9. 9Departments of Medicine and Pediatrics, The Ohio State University College of Medicine, Columbus, Ohio, USA
  10. 10TARGET LUPUS PPIE Group, University of Liverpool, Liverpool, UK
  11. 11Department of Pediatrics, University of Cincinnati, Cincinnati, Ohio, USA
  12. 12Department of Pediatrics, Harvard Medical School, Boston, Massachusetts, USA
  13. 13Centre for Rheumatology, University College London, London, UK
  14. 14University of Nevada Reno School of Social Work, Reno, Nevada, USA
  15. 15Department of Pediatrics, Hofstra Northwell School of Medicine at Hofstra University, Hempstead, New York, USA
  16. 16Division of Rheumatology, Inflammation and Immunity, Brigham and Women's Hospital, Boston, Massachusetts, USA
  17. 17The University of Utah School of Medicine, Salt Lake City, Utah, USA
  18. 18Division of Rheumatology and Department of Pediatrics, The Hospital for Sick Children, Toronto, Ontario, Canada
  19. 19Division of Rheumatology, University of Toronto Temerty Faculty of Medicine, Toronto, Ontario, Canada
  20. 20Erasmus MC University Medical Center, Rotterdam, The Netherlands
  21. 21NIHR Great Ormond Street Hospital Biomedical Research Centre, University College London, London, UK
  22. 22Institute of Child Health and Department of Paediatric Nephrology, Great Ormond Street Hospital for Children NHS Foundation Trust, London, UK
  23. 23Department of Pediatrics, The Aga Khan University Medical College East Africa, Nairobi, Kenya
  24. 24Department of Pediatrics, Hacettepe University, Ankara, Turkey
  25. 25Department of Medicine, Northwestern University Feinberg School of Medicine, Chicago, Illinois, USA
  26. 26Direzione Scientifica, IRCCS Istituto Giannina Gaslini and Dipartimento di Neuroscienze, Riabilitazione, Oftalmologia, Genetica e Scienze Materno-Infantili, Università degli Studi di Genova, Genova, Italy
  27. 27Departments of Population Health Sciences and Pediatrics, Duke University School of Medicine, Durham, North Carolina, USA
  28. 28Children's Hospital at Montefiore, New York, New York, USA
  29. 29Pediatric Rheumatology Division, Faculdade de Medicina de Botucatu, Universidade Estadual Paulista (UNESP), Botucatu, Brazil
  30. 30Institute of Child Health, Paediatric Rheumatology Division, Sir Ganga Ram Hospital, New Delhi, India
  31. 31Department of Pediatrics, Duke University School of Medicine, Durham, North Carolina, USA
  32. 32Department of Pediatrics, University of California San Francisco, San Francisco, California, USA
  33. 33Department of Child and Adolescent Health, University of Cape Town, Rondebosch, South Africa
  34. 34Division of Pediatric Rheumatology, University of Ottawa, Ottawa, Ontario, Canada
  35. 35Patient/Caregiver Representative, Childhood Arthritis and Rheumatology Research Alliance, Milwaukee, Wisconsin, USA
  36. 36Department of Pediatrics, The University of British Columbia, Vancouver, Columbia, Canada
  37. 37BC Children's Hospital, Vancouver, Columbia, Canada
  38. 38National Institutes of Health, National Institute of Arthritis and Musculoskeletal and Skin Diseases, Bethesda, Maryland, USA
  1. Correspondence to Dr Eve MD Smith; esmith8{at}liverpool.ac.uk

Abstract

Objectives Childhood-onset systemic lupus erythematosus (cSLE), representing 15%–20% of individuals with SLE, has been difficult to study globally due to differences between registries. This initiative, supported by Childhood Arthritis Rheumatology Research Alliance (CARRA) and Paediatric Rheumatology European Society (PReS), aims to create Core and Expanded cSLE Datasets to standardise and enhance research worldwide.

Methods 21 international cSLE experts and 4 patients participated in a Delphi process (questionnaires, 2 topic-specific focus groups and 3 virtual consensus meetings) to create 2 standardised cSLE datasets. The Core cSLE Dataset was designed to include data essential to meaningful clinical research across many settings. The Expanded cSLE Dataset was designed for centres able to consistently collect data to address broader research questions. Final data items for the Core and Expanded datasets were determined by consensus defined as >80% agreement) using an adapted nominal group technique and voting.

Results The resulting Core cSLE Dataset contains 46 items, including demographics, clinical features, laboratory results, medications and significant adverse events. The Expanded cSLE Dataset adds 26 additional items and includes patient-reported outcomes. Consensus was also achieved regarding the frequency and time points for data collection: baseline, quarterly follow-up visits, annually and flare visits.

Conclusion Standardised Core and Expanded cSLE Datasets for registry-based international cSLE research were defined through the consensus of global experts and patient/caregiver representatives, endorsed by CARRA and PReS. These datasets incorporate disease-specific and patient-specific features, optimised for diverse settings to facilitate international collaborative research for children and adolescents with SLE worldwide.

  • Child
  • Lupus Erythematosus, Systemic
  • Lupus Nephritis
  • Outcome Assessment, Health Care
  • Patient Reported Outcome Measures

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Footnotes

  • RES, JCC and AB are joint first authors.

  • LBL and EMS are joint senior authors.

  • Handling editor Josef S Smolen

  • X @AlexandreBelot, @Elissa_Weitzman, @PedsLupusRenal, @evemdsmith_eve

  • Contributors AB, EMDS, JCC, LBL and RS developed the Delphi surveys, organised and facilitated the consensus meeting and wrote the first version of the manuscript. AAggarwal, AH, AMK, AM, AR, CC, CMH, CS, CS-M, DL, DAI, EvS, GT, HIB, KM, LB, LTH, LL, LES, MWB, MBS, NM, SA, SK, SM, SO, SA, SW and TA participated as voting members of the consensus meetings, contributing to decisions regarding items to be included in the Core and Expanded datasets. AD, AAkinsete, AW, BE, BBR, CF, ERW, JC, RR-G and TBR participated in a task force meeting, making intellectual contributions to the decisions regarding which PROs to incorporate. All authors reviewed and approved the final draft of the manuscript. EMDS is the guarantor. EMDS and LBL are Co-last authors.

  • Funding This research was funded by the inaugural CARRA/PReS Collaborative Research Award (JXR30630). LBL was funded by the NIAMS Intramural Program.

  • Competing interests None declared.

  • Patient and public involvement statement Patients and parents from the USA and Europe actively participated in the design and conduct of this research. The cSLE Patient/Caregiver committee included two parents and three young adults with childhood-onset SLE, now over 18 years old, providing patient-centred perspectives. They attended all virtual consensus meetings with full, equal voting rights. They joined a focus group to identify key domains and assess the patient experience using PRO measures, voting on the most important domains to be captured and participated in a PRO expert focus group with cSLE consensus group experts and additional PRO researchers. Additionally, they were involved in discussions on efficiently capturing cSLE patients' experiences with discrimination. Patients and parents reviewed the manuscript reporting the Core and Expanded cSLE datasets and are appropriately included as named authors.

  • Provenance and peer review Not commissioned; externally peer reviewed.

  • Supplemental material This content has been supplied by the author(s). It has not been vetted by BMJ Publishing Group Limited (BMJ) and may not have been peer-reviewed. Any opinions or recommendations discussed are solely those of the author(s) and are not endorsed by BMJ. BMJ disclaims all liability and responsibility arising from any reliance placed on the content. Where the content includes any translated material, BMJ does not warrant the accuracy and reliability of the translations (including but not limited to local regulations, clinical guidelines, terminology, drug names and drug dosages), and is not responsible for any error and/or omissions arising from translation and adaptation or otherwise.