title: Defining disease activity states and clinically meaningful improvement in primary Sjögren's syndrome with EULAR primary Sjögren's syndrome disease activity (ESSDAI) and patient-reported indexes (ESSPRI).
abstract: OBJECTIVE
  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.
authors.affiliation: 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.
authors.forename: Raphaèle
authors.initials: R
authors.lastname: Seror
authors.affiliation: Department of Rheumatology and Clinical Immunology, University of Groningen, University Medical Center Groningen, Groningen, The Netherlands.
authors.forename: Hendrika
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authors.lastname: Bootsma
authors.affiliation: Department of Rheumatology, Hôpital Cavale Blanche, CHU Brest, and EA 2216, Université Bretagne occidentale, Brest, France.
authors.forename: Alain
authors.initials: A
authors.lastname: Saraux
authors.affiliation: Rheumatology Department, University Hospital Birmingham NHS Foundation Trust, Birmingham, UK.
authors.forename: Simon J
authors.initials: SJ
authors.lastname: Bowman
authors.affiliation: Department of Rheumatology, Skane University Hospital Malmö, Lund University, Malmö, Sweden.
authors.forename: Elke
authors.initials: E
authors.lastname: Theander
authors.affiliation: Department of Rheumatology, Haukeland University Hospital, Bergen, Norway.
authors.forename: Johan G
authors.identifier: http://orcid.org/0000-0002-6004-3702
authors.initials: JG
authors.lastname: Brun
authors.affiliation: Center of Clinical Epidemiology, Hôpital Hotel Dieu, Paris, France INSERM U738, Université Paris-René Descartes, Paris, France.
authors.forename: Gabriel
authors.initials: G
authors.lastname: Baron
authors.affiliation: 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.
authors.forename: Véronique
authors.initials: V
authors.lastname: Le Guern
authors.affiliation: Department of Rheumatology, Hôpital Cavale Blanche, CHU Brest, and EA 2216, Université Bretagne occidentale, Brest, France.
authors.forename: Valérie
authors.initials: V
authors.lastname: Devauchelle-Pensec
authors.affiliation: Laboratory of Autoimmune Diseases "Josep Font", IDIBAPS, ICMiD, Hospital Clinic, Barcelona, Spain.
authors.forename: Manel
authors.identifier: http://orcid.org/0000-0001-5709-6734
authors.initials: M
authors.lastname: Ramos-Casals
authors.affiliation: Division of Rheumatology, Department of Medicine, Federal University of Espírito Santo, Espírito Santo, Brazil.
authors.forename: Valeria
authors.initials: V
authors.lastname: Valim
authors.affiliation: Rheumatology Department, Charité, University Hospital, Berlin, Germany.
authors.forename: Thomas
authors.initials: T
authors.lastname: Dörner
authors.affiliation: Department of Pathophysiology, School of Medicine, University of Athens, Athens, Greece.
authors.forename: Athanasios
authors.initials: A
authors.lastname: Tzioufas
authors.affiliation: 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.
authors.forename: Jacques-Eric
authors.initials: JE
authors.lastname: Gottenberg
authors.affiliation: Department of Autoimmune systemic Diseases, Vall d'Hebron University Hospital, Barcelona, Spain.
authors.forename: Roser
authors.initials: R
authors.lastname: Solans Laqué
authors.affiliation: Department of Rheumatology, Skane University Hospital Malmö, Lund University, Malmö, Sweden.
authors.forename: Thomas
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authors.lastname: Mandl
authors.affiliation: Department of Internal Medicine, Claude Huriez Hospital, Université Nord de France, Lille, France.
authors.forename: Eric
authors.initials: E
authors.lastname: Hachulla
authors.affiliation: Department of Arthritis and Clinical Immunology, Oklahoma Medical research foundation, Oklahoma City, USA.
authors.forename: Kathy L
authors.initials: KL
authors.lastname: Sivils
authors.affiliation: Musculoskeletal Research Group, Institute of Cellular Medicine, Newcastle University, Newcastle upon Tyne, UK.
authors.forename: Wan-Fai
authors.initials: WF
authors.lastname: Ng
authors.affiliation: Department of Rheumatology, University Hospital, Limoges, France.
authors.forename: Anne-Laure
authors.initials: AL
authors.lastname: Fauchais
authors.affiliation: Rheumatology Unit, Department of Internal Medicine, University of Pisa, Pisa, Italy.
authors.forename: Stefano
authors.initials: S
authors.lastname: Bombardieri
authors.affiliation: Department of Medicina Interna e Specialità Mediche, Rheumatology Clinic, La Sapienza University of Rome, Rome, Italy.
authors.forename: Roberta
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authors.lastname: Priori
authors.affiliation: Department of Rheumatology, Perugia University Hospital, Perugia, Italy.
authors.forename: Elena
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authors.lastname: Bartoloni
authors.affiliation: Department of Rheumatology, Amiens university hospital, Amiens, France.
authors.forename: Vincent
authors.initials: V
authors.lastname: Goeb
authors.affiliation: Department of Rheumatology, University Medical Centre Ljubljana, Ljubljana, Slovenia.
authors.forename: Sonja
authors.initials: S
authors.lastname: Praprotnik
authors.affiliation: Department of Internal Medicine, University of Tsukuba, Tsukuba, Japan.
authors.forename: Takayuki
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authors.lastname: Sumida
authors.affiliation: Rheumatic Disease Center, Kurashiki Medical Center, Kurashiki, Japan.
authors.forename: Sumusu
authors.initials: S
authors.lastname: Nishiyama
authors.affiliation: Department of Rheumatology, University of Pavia, IRCCS S. Matteo Foundation, Pavia, Italy.
authors.forename: Roberto
authors.initials: R
authors.lastname: Caporali
authors.affiliation: Department of Rheumatology & Clinical Immunology, University Medical Center Utrecht (UMCU), Utrecht, The Netherlands.
authors.forename: Aike A
authors.initials: AA
authors.lastname: Kruize
authors.affiliation: Department of Rheumatology, German Hospital, Buenos-Aires, Argentina.
authors.forename: Cristina
authors.initials: C
authors.lastname: Vollenweider
authors.affiliation: Center of Clinical Epidemiology, Hôpital Hotel Dieu, Paris, France INSERM U738, Université Paris-René Descartes, Paris, France.
authors.forename: Philippe
authors.initials: P
authors.lastname: Ravaud
authors.affiliation: Department of Oral and Maxillofacial Surgery, University of Groningen, University Medical Center Groningen, Groningen, The Netherlands.
authors.forename: Petra
authors.initials: P
authors.lastname: Meiners
authors.affiliation: Laboratory of Autoimmune Diseases "Josep Font", IDIBAPS, ICMiD, Hospital Clinic, Barcelona, Spain.
authors.forename: Pilar
authors.initials: P
authors.lastname: Brito-Zerón
authors.affiliation: Sections of Rheumatology, Instituto San Giuseppe, Como and Casa di Cura di Lecco, Lecco, Italy.
authors.forename: Claudio
authors.initials: C
authors.lastname: Vitali
authors.affiliation: Department of Rheumatology, Hôpitaux Universitaires Paris-Sud, Assistance Publique-Hopitaux de Paris (AP-HP), Université Paris-Sud, INSERM U1012, Le Kremlin Bicêtre, France.
authors.forename: Xavier
authors.initials: X
authors.lastname: Mariette
mesh_terms: D000368:Aged; D059026:Diagnostic Self Evaluation; D018450:Disease Progression; D005260:Female; D006304:Health Status; D006801:Humans; D008297:Male; D008875:Middle Aged; D017060:Patient Satisfaction; D011446:Prospective Studies; D012372:ROC Curve; D012720:Severity of Illness Index; D012859:Sjogren's Syndrome / Q000175:diagnosis* / Q000523:psychology; D063189:Symptom Assessment / Q000379:methods* / Q000523:psychology
keywords: Outcomes research; Patient perspective; Sjøgren's Syndrome