Raphaèle Seror1, Hendrika Bootsma2, Alain Saraux3, Simon J Bowman4, Elke Theander5, Johan G Brun6, Gabriel Baron7, Véronique Le Guern8, Valérie Devauchelle-Pensec3, Manel Ramos-Casals9, Valeria Valim10, Thomas Dörner11, Athanasios Tzioufas12, Jacques-Eric Gottenberg13, Roser Solans Laqué14, Thomas Mandl5, Eric Hachulla15, Kathy L Sivils16, Wan-Fai Ng17, Anne-Laure Fauchais18, Stefano Bombardieri19, Roberta Priori20, Elena Bartoloni21, Vincent Goeb22, Sonja Praprotnik23, Takayuki Sumida24, Sumusu Nishiyama25, Roberto Caporali26, Aike A Kruize27, Cristina Vollenweider28, Philippe Ravaud7, Petra Meiners29, Pilar Brito-Zerón9, Claudio Vitali30, Xavier Mariette31. 1. Department of Rheumatology, Hôpitaux Universitaires Paris-Sud, Assistance Publique-Hopitaux de Paris (AP-HP), Université Paris-Sud, INSERM U1012, Le Kremlin Bicêtre, France Center of Clinical Epidemiology, Hôpital Hotel Dieu, Paris, France INSERM U738, Université Paris-René Descartes, Paris, France. 2. Department of Rheumatology and Clinical Immunology, University of Groningen, University Medical Center Groningen, Groningen, The Netherlands. 3. Department of Rheumatology, Hôpital Cavale Blanche, CHU Brest, and EA 2216, Université Bretagne occidentale, Brest, France. 4. Rheumatology Department, University Hospital Birmingham NHS Foundation Trust, Birmingham, UK. 5. Department of Rheumatology, Skane University Hospital Malmö, Lund University, Malmö, Sweden. 6. Department of Rheumatology, Haukeland University Hospital, Bergen, Norway. 7. Center of Clinical Epidemiology, Hôpital Hotel Dieu, Paris, France INSERM U738, Université Paris-René Descartes, Paris, France. 8. Department of Internal Medicine and National Referral Centre for Rare Systemic Auto-immune Diseases, Assistance Publique-Hopitaux de Paris, Hôpital Cochin, Paris Descartes University, Paris, France. 9. Laboratory of Autoimmune Diseases "Josep Font", IDIBAPS, ICMiD, Hospital Clinic, Barcelona, Spain. 10. Division of Rheumatology, Department of Medicine, Federal University of Espírito Santo, Espírito Santo, Brazil. 11. Rheumatology Department, Charité, University Hospital, Berlin, Germany. 12. Department of Pathophysiology, School of Medicine, University of Athens, Athens, Greece. 13. Department of Rheumatology, Centre National de Référence des Maladies Auto-Immunes Rares, INSERM UMRS_1109, Fédération de Médecine Translationnelle de Strasbourg (FMTS), Strasbourg university Hospital, Université de Strasbourg, Strasbourg, France. 14. Department of Autoimmune systemic Diseases, Vall d'Hebron University Hospital, Barcelona, Spain. 15. Department of Internal Medicine, Claude Huriez Hospital, Université Nord de France, Lille, France. 16. Department of Arthritis and Clinical Immunology, Oklahoma Medical research foundation, Oklahoma City, USA. 17. Musculoskeletal Research Group, Institute of Cellular Medicine, Newcastle University, Newcastle upon Tyne, UK. 18. Department of Rheumatology, University Hospital, Limoges, France. 19. Rheumatology Unit, Department of Internal Medicine, University of Pisa, Pisa, Italy. 20. Department of Medicina Interna e Specialità Mediche, Rheumatology Clinic, La Sapienza University of Rome, Rome, Italy. 21. Department of Rheumatology, Perugia University Hospital, Perugia, Italy. 22. Department of Rheumatology, Amiens university hospital, Amiens, France. 23. Department of Rheumatology, University Medical Centre Ljubljana, Ljubljana, Slovenia. 24. Department of Internal Medicine, University of Tsukuba, Tsukuba, Japan. 25. Rheumatic Disease Center, Kurashiki Medical Center, Kurashiki, Japan. 26. Department of Rheumatology, University of Pavia, IRCCS S. Matteo Foundation, Pavia, Italy. 27. Department of Rheumatology & Clinical Immunology, University Medical Center Utrecht (UMCU), Utrecht, The Netherlands. 28. Department of Rheumatology, German Hospital, Buenos-Aires, Argentina. 29. Department of Oral and Maxillofacial Surgery, University of Groningen, University Medical Center Groningen, Groningen, The Netherlands. 30. Sections of Rheumatology, Instituto San Giuseppe, Como and Casa di Cura di Lecco, Lecco, Italy. 31. Department of Rheumatology, Hôpitaux Universitaires Paris-Sud, Assistance Publique-Hopitaux de Paris (AP-HP), Université Paris-Sud, INSERM U1012, Le Kremlin Bicêtre, France.
Abstract
OBJECTIVES: To define disease activity levels, minimal clinically important improvement (MCII) and patient-acceptable symptom state (PASS) with the primary Sjögren's syndrome (SS) disease activity indexes: European League Against Rheumatism (EULAR) SS disease activity index (ESSDAI) and EULAR SS patient-reported index (ESSPRI). METHODS: For 790 patients from two large prospective cohorts, ESSDAI, physician evaluation of disease activity, ESSPRI and patients' satisfaction with their current health status were recorded. Receiver operating characteristic curve analyses and anchoring methods were used to estimate disease activity levels of ESSDAI and the PASS of ESSPRI. At follow-up visit, patients and physicians assessed, respectively, whether symptoms and disease activity have improved or not. An anchoring method based on this evaluation was used to estimate MCII of ESSDAI and ESSPRI. RESULTS: Low-activity (ESSDAI<5), moderate-activity (5≤ESSDAI≤13) and high-activity (ESSDAI≥14) levels were defined. MCII of ESSDAI was defined as an improvement of at least three points. The PASS estimate was defined as an ESSPRI<5 points and MCII as a decrease of at least one point or 15%. CONCLUSIONS: This study determined disease activity levels, PASS and MCII of ESSDAI and ESSPRI. These results will help designing future clinical trials in SS. For evaluating systemic complications, the proposal is to include patients with moderate activity (ESSDAI≥5) and define response to treatment as an improvement of ESSDAI at least three points. For addressing patient-reported outcomes, inclusion of patients with unsatisfactory symptom state (ESSPRI≥5) and defining response as an improvement of ESSPRI at least one point or 15% seems reasonable. Published by the BMJ Publishing Group Limited. For permission to use (where not already granted under a licence) please go to http://www.bmj.com/company/products-services/rights-and-licensing/
OBJECTIVES: To define disease activity levels, minimal clinically important improvement (MCII) and patient-acceptable symptom state (PASS) with the primary Sjögren's syndrome (SS) disease activity indexes: European League Against Rheumatism (EULAR) SS disease activity index (ESSDAI) and EULAR SS patient-reported index (ESSPRI). METHODS: For 790 patients from two large prospective cohorts, ESSDAI, physician evaluation of disease activity, ESSPRI and patients' satisfaction with their current health status were recorded. Receiver operating characteristic curve analyses and anchoring methods were used to estimate disease activity levels of ESSDAI and the PASS of ESSPRI. At follow-up visit, patients and physicians assessed, respectively, whether symptoms and disease activity have improved or not. An anchoring method based on this evaluation was used to estimate MCII of ESSDAI and ESSPRI. RESULTS: Low-activity (ESSDAI<5), moderate-activity (5≤ESSDAI≤13) and high-activity (ESSDAI≥14) levels were defined. MCII of ESSDAI was defined as an improvement of at least three points. The PASS estimate was defined as an ESSPRI<5 points and MCII as a decrease of at least one point or 15%. CONCLUSIONS: This study determined disease activity levels, PASS and MCII of ESSDAI and ESSPRI. These results will help designing future clinical trials in SS. For evaluating systemic complications, the proposal is to include patients with moderate activity (ESSDAI≥5) and define response to treatment as an improvement of ESSDAI at least three points. For addressing patient-reported outcomes, inclusion of patients with unsatisfactory symptom state (ESSPRI≥5) and defining response as an improvement of ESSPRI at least one point or 15% seems reasonable. Published by the BMJ Publishing Group Limited. For permission to use (where not already granted under a licence) please go to http://www.bmj.com/company/products-services/rights-and-licensing/
Authors: Peter Olsson; Iris L A Bodewes; Anna M Nilsson; Carl Turesson; Lennart T H Jacobsson; Elke Theander; Marjan A Versnel; Thomas Mandl Journal: Rheumatol Int Date: 2019-05-28 Impact factor: 2.631
Authors: Normann Steiner; Georg Göbel; Daniela Michaeler; Anna-Luise Platz; Wolfgang Prokop; Anna Maria Wolf; Dominik Wolf; Christina Duftner; Eberhard Gunsilius Journal: Blood Adv Date: 2021-03-23
Authors: Clare Oni; Sheryl Mitchell; Katherine James; Wan-Fai Ng; Bridget Griffiths; Victoria Hindmarsh; Elizabeth Price; Colin T Pease; Paul Emery; Peter Lanyon; Adrian Jones; Michele Bombardieri; Nurhan Sutcliffe; Costantino Pitzalis; John Hunter; Monica Gupta; John McLaren; Annie Cooper; Marian Regan; Ian Giles; David Isenberg; Vadivelu Saravanan; David Coady; Bhaskar Dasgupta; Neil McHugh; Steven Young-Min; Robert Moots; Nagui Gendi; Mohammed Akil; Francesca Barone; Ben Fisher; Saaeha Rauz; Andrea Richards; Simon J Bowman Journal: Rheumatology (Oxford) Date: 2015-10-27 Impact factor: 7.580