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Methods of deriving EULAR/ACR recommendations on reporting disease activity in clinical trials of patients with rheumatoid arthritis
  1. T Karonitsch1,
  2. D Aletaha1,
  3. M Boers2,
  4. S Bombardieri3,
  5. B Combe4,
  6. M Dougados5,
  7. P Emery6,
  8. D Felson7,
  9. J Gomez-Reino8,
  10. E Keystone9,
  11. T K Kvien10,
  12. E Martin-Mola11,
  13. M Matucci-Cerinic12,
  14. P Richards13,
  15. P van Riel14,
  16. J Siegel15,
  17. J S Smolen1,16,
  18. T Sokka17,
  19. D van der Heijde18,
  20. R van Vollenhoven19,
  21. M Ward20,
  22. G Wells21,
  23. A Zink22,
  24. R Landewe23
  1. 1
    Medical University of Vienna, Vienna, Austria
  2. 2
    VU University Medical Center, Amsterdam, The Netherlands
  3. 3
    University of Pisa, Pisa, Italy
  4. 4
    CHU de Montpellier, Montpellier, France
  5. 5
    Paris-Descartes University, Paris, France
  6. 6
    University of Leeds, Leeds, UK
  7. 7
    Boston University School of Medicine, Boston, Massachusetts, USA
  8. 8
    University of Santiago de Compostela School of Medicine, Santiago, Spain
  9. 9
    University of Toronto, Toronto, Ontario, Canada
  10. 10
    Diakonhjemmet Hospital, Oslo, Norway
  11. 11
    Hospital Universitario La Paz, Madrid, Spain
  12. 12
    University of Florence, Florence, Italy
  13. 13
    University of Bristol, Bristol, UK
  14. 14
    Radboud University Nijmegen Medical Centre, Nijmegen, The Netherlands
  15. 15
    Food and Drug Administration, Rockville, Maryland, USA
  16. 16
    Hietzing Hospital, Vienna, Austria
  17. 17
    Jyvaskyla Central Hospital, Jyvaskyla, Finland
  18. 18
    Leiden University Medical Center, Leiden, The Netherlands
  19. 19
    Karolinska University Hospital, Stockholm, Sweden
  20. 20
    National Institutes of Health, Bethesda, Maryland, USA
  21. 21
    University of Ottawa, Ottawa, Ontario, Canada
  22. 22
    Charité Medical School, Berlin, Germany
  23. 23
    University Hospital Maastricht, Maastricht, The Netherlands
  1. D Aletaha, Division of Rheumatology, Medical University of Vienna, Waehringer Guertel 18–20, 1090 Vienna, Austria; daniel.aletaha{at}meduniwien.ac.at

Abstract

Objective: To use an evidence-based and consensus-based approach to elaborate recommendations on how to report disease activity in clinical trials of patients with rheumatoid arthritis (RA) endorsed by the European League Against Rheumatism (EULAR) and the American College of Rheumatology (ACR).

Methods: After an initial expert meeting, during which relevant research questions were identified, a systematic literature search was performed using Medline, Embase and the Cochrane Library as sources. To ensure literature retrieved was comprehensive, we emphasised search algorithms that were sensitive rather than specific. The results of the literature search were discussed by the expert panel, modified and expanded, and were used as the basis for the elaboration of the recommendation in the consensus process. Finally, an independent ACR panel approved these items with some minor modifications.

Results: The following pieces of evidence were obtained from the literature search: (1) timing and the sustaining of a response is relevant to achieve better outcomes; (2) composite disease activity indices have been used to define low disease activity and remission and these definitions have been validated as has the American Rheumatism Association (ARA) remission criteria. The “patient-reported symptom state” (PASS) is not yet well validated; (3) evidence was obtained to identify those measures, scales and patient-reported instruments, for which there is a documented association with relevant outcomes; (4) baseline disease activity is associated with disease activity levels at the end of follow-up; and (5) there was not sufficient evidence relating the added benefit of MRI or ultrasound over clinical assessments. Most data stemmed from observational studies rather than clinical trials and literature review was supplemented by input from experts. The results served as the basis for the elaboration of the seven recommendations by the experts.

Conclusions: The approach based on scientific evidence from the literature as well as on expert input provided sufficient information to derive recommendations on reporting disease activity in RA clinical trials. The methodology, results and conclusions of this project were endorsed by EULAR and the ACR.

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Footnotes

  • Funding: This project was fully funded by EULAR and the ACR.

  • Competing interests: None.

  • The views presented in this presentation do not necessarily reflect those of the Food and Drug Administration or the National Institutes of Health.

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