OP0286 DEVELOPMENT AND PRELIMINARY VALIDATION OF THE SJÖGREN’S TOOL FOR ASSESSING RESPONSE (STAR): A CONSENSUAL COMPOSITE SCORE FOR ASSESSING TREATMENT EFFECT IN PRIMARY SJÖGREN’S SYNDROME

Background Today, there are still no DMARDs licensed for primary Sjögren Syndrome (pSS) patients. Among the explanations, limitations of current outcome measures used as endpoints: e.g; high placebo response rate, evaluation either symptoms or systemic activity, and important features not being assessed. The NECESSITY consortium ( https://www.necessity-h2020.eu/ ), including pSS experts from academia, pharmaceutical industry patient groups formed to develop a new composite responder index, Sjögren’s Tool Assessing Response (STAR) that solve issues in is intended use clinical trials an efficacy endpoint. Objectives To index syndrome (pSS): STAR. Methods STAR, data-driven methods, based on 9 randomized controlled (RCTs), consensus techniques, involving 78 20 Based reanalysis rituximab (TRACTISS TEARS) literature review, Delphi panel identified core set domains include with their respective measures. STAR options combining these were designed proposed select improve them. For each option, sensitivity change was estimated by C-index (derived Effect size) all RCTs. rounds run selecting among options. also voted classify positive, negative “in between” regards but key secondary endpoints. remaining before final vote, meta-analyses RCTs performed separately positive together, trials. Results 5 (systemic symptoms, lachrymal gland function, salivary function biological parameters), 227 options, domains, selected be tested change. After two rounds, performed. candidate vote metrological properties relevance. In positive/in between trials, detected difference arms (OR 3.29, 95%-CI [2.07;5.22], whereas it did 1.53, [0.81;2.91]). Conclusion single tool main disease features, endpoint Its rigorous consensual development process ensures its face content validity. showed good specificity will prospectively validated dedicated three RCT evaluate combination synthetic (hydroxychloroquine + lefunomide hydroxychloroquine mycophenolate vs placebo). We encourage any ongoing future Table 1. Candidate Domain Point Definition Systemic activity 3 Decrease clinESSDAI ≥ Patient reported ESSPRI 1 point 15% Lachrymal Schirmer: If abnormal score at baseline: increase mm baseline normal Or Ocular Staining Score: decrease 2 points Salivary Unstimulated Whole Flow: > 0 25% Ultrasound: total Hocevar Biological Serum IgG levels: 10% Rheumatoid Factor ESSDAI: EULAR index; ESSPRI: IgG: Immunoglobulin G; Acknowledgements WP5 participants: Suzanne Arends (University Medical Center Groningen, Department Rheumatology Clinical Immunology, Groningen 9700 RB, Netherlands), Francesca Barone (Centre Translational Inflammation Research, Institute Ageing, University Birmingham, UK), Albin Björk (Division Rheumatology, Medicine, Karolinska Institutet, Stockholm, Sweden), Coralie Bouillot (Association Française du Gougerot et des Syndromes Secs, France), Guillermo Carvajal Alegria Brest, Inserm, CHU de LBAI, UMR1227, France; Service Rhumatologie, Centre Référence Maladies Autoimmunes Rares CERAINO, Cavale Blanche, Wen-Hung Chen (GlaxoSmithKline, Research Triangle Park, North Carolina, USA), Kenneth Clark (GlaxoSmithKline Medicines Centre, Gunnels Wood Road, Stevenage, Hertfordshire SG1 2NY, United Kingdom), Konstantina Delli (Department Oral Maxillofacial Surgery, (UMCG), Salvatore Vita (Rheumatology Clinic, Hospital Udine, Italy), Liseth Wolff Jennifer Evans (Novartis Pharmaceuticals corporation Stéphanie Galtier (Institut Recherches Internationales Servier (IRIS), Suresnes Cedex, Saviana Gandolfo area, ASUFC, 33100 Mickael Guedj Dewi Guellec (CHU CIC 1412, Safae Hamkour (Center Utrecht, Utrecht 3584 GA, Dominik Hartl Institutes BioMedical Basel, Switzerland), Malin Jonsson (Section Radiology, Dentistry, Faculty Medicine Bergen, Norway), Roland (Broegelmann Laboratory, Science, Haukeland Hospital, Frans Kroese Aike Albert Kruize Laurence Laigle Véronique Le Guern (AP-HP, Hôpital Cochin, référence maladies auto-immunes systémiques rares, service médecine interne, Paris, Wen-Lin Luo Biometrics Statistical Novartis Pharmaceuticals, East Hanover, New Jersey), Esther Mossel Wan-Fai Ng (Institute Cellular Newcastle University, upon Tyne NE2 4HH, Gaëtane Nocturne Université Paris-Saclay, INSERM U1184: Immunology Viral Infections Autoimmune Diseases, Assistance Publique-Hôpitaux Bicêtre, Kremlin Marleen Nys (Global Biometric Sciences, Bristol Myers Squibb, Braine L’Alleud, Belgium), Roald Omdal (Clinical Unit, Internal Stavanger PO Box 8100, 4068, Stavanger, Jacques-Olivier Pers (LBAI, France Maggy Pincemin Manel Ramos-Casals Clinic Barcelona Institut Medicinai Dermatologia, Barcelona, Catalunya, Spain), Philippe Ravaud d’Epidémiologie Clinique, Hôtel-Dieu, Neelanjana Ray Drug Development - Squibb Company, Princeton, Jersey, Alain Saraux (HU Univ Lymphocytes B Autoimmunité, LabEx IGO, Athanasios Tzioufas Gwenny Verstappen Arjan Vissink, Marie Wahren-Herlenius Sweden). thank following experts: Esen Karamursel Akpek, Alan Baer, Chiara Baldini, Elena Bartoloni, Marí-Alfonso Begona, Johan Brun, Vatinee Bunya, Laurent Chiche, Troy Daniels, Paul Emery, Robert Fox, Roberto Giacomelli, John Gonzales, Greenspan, Moots, Susumu Nishiyama, Elizabeth Price, Christophe Richez, Caroline Shiboski, Roser Solans Laque, Muthiah Srinivasan, Peter Olsson, Tsutomu Takeuchi, Frederick Vivino, Paraskevi Voulgari, Daniel Wallace, Ava Wu, Wen Zhang. anonymous patients Advisory Group Foundation valuable contribution process. EW StClair AN Baer who generated baminercept data made them publicly available. Disclosure Interests Raphaèle Seror Consultant of: GlaxoSmithKline, Boehringer, Janssen Novartis, Grant/research support from: GlaxoSmithKline Amgen, Gabriel Baron: None declared, Marine Camus: Divi Cornec Janssen, Pfizer Roche, Elodie Perrodeau: Simon J. Bowman Abbvie, Astra Zeneca, Galapagos Michele Bombardieri UCB, Amgen/Medimmune, Hendrika Bootsma: Jacques-Eric Gottenberg AbbVie, Eli Lilly, Galapagos, Gilead, Pfizer, Sanofi, MSD, CSL-Behring Genzyme, Benjamin Fisher Speakers bureau: Servier, Wolfgang Hueber Shareholder Pharma, Employee Joel van Roon: Valerie Devauchelle-Pensec: Gergely Xavier Mariette Ose Raphaël Porcher: declared