1. 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
- Author
-
Raphaele, Seror, Gabriel, Baron, Marine, Camus, Divi, Cornec, Elodie, Perrodeau, Simon J, Bowman, Michele, Bombardieri, Hendrika, Bootsma, Jacques-Eric, Gottenberg, Benjamin, Fisher, Wolfgang, Hueber, Joel A, van Roon, Valérie, Devauchelle-Pensec, Peter, Gergely, Xavier, Mariette, Raphael, Porcher, Marie, Wahren-Herlenius, Piatrova, Alena, NEw Clinical Endpoints in primary Sjögren’s Syndrome: an Interventional Trial based on stratifYing patients - NECESSITY - 0000-00-00 - 0000-00-00 - 806975 - VALID, Translational Immunology Groningen (TRIGR), Immunologie des Maladies Virales et Autoimmunes (IMVA - U1184), Université Paris-Sud - Paris 11 (UP11)-Commissariat à l'énergie atomique et aux énergies alternatives (CEA)-Institut National de la Santé et de la Recherche Médicale (INSERM), Hôpital Bicêtre, Hôpital Hôtel-Dieu [Paris], Assistance publique - Hôpitaux de Paris (AP-HP) (AP-HP), Centre de Recherche Épidémiologie et Statistique Sorbonne Paris Cité (CRESS (U1153 / UMR_A_1125 / UMR_S_1153)), Conservatoire National des Arts et Métiers [CNAM] (CNAM), HESAM Université - Communauté d'universités et d'établissements Hautes écoles Sorbonne Arts et métiers université (HESAM)-HESAM Université - Communauté d'universités et d'établissements Hautes écoles Sorbonne Arts et métiers université (HESAM)-Université Sorbonne Paris Cité (USPC)-Institut National de la Santé et de la Recherche Médicale (INSERM)-Université Paris Cité (UPCité)-Institut National de Recherche pour l’Agriculture, l’Alimentation et l’Environnement (INRAE), Lymphocytes B, Autoimmunité et Immunothérapies (LBAI), Université de Brest (UBO)-Institut National de la Santé et de la Recherche Médicale (INSERM)-LabEX IGO Immunothérapie Grand Ouest, Nantes Université (Nantes Univ)-Nantes Université (Nantes Univ)-Institut Brestois Santé Agro Matière (IBSAM), Université de Brest (UBO), CHRU Brest - Service de Rhumatologie (CHU - BREST - Rhumato), Centre Hospitalier Régional Universitaire de Brest (CHRU Brest), University Hospitals Birmingham [Birmingham, Royaume-Uni], University of Birmingham [Birmingham], Queen Mary University of London (QMUL), University of Groningen [Groningen], CHU Strasbourg, Institut de biologie moléculaire et cellulaire (IBMC), Université de Strasbourg (UNISTRA)-Centre National de la Recherche Scientifique (CNRS), Novartis Pharma AG, Utrecht University [Utrecht], Novartis Institutes for BioMedical Research (NIBR), NECESSITY WP5- STAR development working group: Suzanne Arends, Francesca Barone, Albin Björk, Coralie Bouillot, Guillermo Carvajal Alegria, Wen-Hung Chen, Kenneth Clark, Konstantina Delli, Liseth de Wolff, Jennifer Evans, Stéphanie Galtier, Saviana Gandolfo, Mickael Guedj, Dewi Guellec, Safae Hamkour, Dominik Hartl, Malin V Jonsson, Roland Jonsson, Frans G M Kroese, Aike Albert Kruize, Laurence Laigle, Véronique Le Guern, Wen-Lin Luo, Esther Mossel, Wan-Fai Ng, Gaëtane Nocturne, Marleen Nys, Roald Omdal, Jacques-Olivier Pers, Maggy Pincemin, Manel Ramos-Casals, Philippe Ravaud, Neelanjana Ray, Alain Saraux, Athanasios Tzioufas, Gwenny Verstappen, Arjan Vissink, Marie Wahren-Herlenius, seror, raphaele, Service de Rhumatologie [CHU Bicêtre], Assistance publique - Hôpitaux de Paris (AP-HP) (AP-HP)-Hôpital Bicêtre, Projet NECESSITY (PN), Hôpitaux Universitaires Paris Sud [AP-HP] (HUPS)-Immunologie des maladies virales, auto-immunes, hématologiques et bactériennes (IMVA-HB), Commissariat à l'énergie atomique et aux énergies alternatives (CEA)-Institut National de la Santé et de la Recherche Médicale (INSERM)-Université Paris-Saclay-Commissariat à l'énergie atomique et aux énergies alternatives (CEA)-Institut National de la Santé et de la Recherche Médicale (INSERM)-Université Paris-Saclay, Equipe 2 : ECSTRA - Epidémiologie Clinique, STatistique, pour la Recherche en Santé (CRESS - U1153), HESAM Université - Communauté d'universités et d'établissements Hautes écoles Sorbonne Arts et métiers université (HESAM)-HESAM Université - Communauté d'universités et d'établissements Hautes écoles Sorbonne Arts et métiers université (HESAM)-Université Sorbonne Paris Cité (USPC)-Institut National de la Santé et de la Recherche Médicale (INSERM)-Université Paris Cité (UPCité)-Institut National de Recherche pour l’Agriculture, l’Alimentation et l’Environnement (INRAE)-Conservatoire National des Arts et Métiers [CNAM] (CNAM), CHRU Brest - Service de Nephrologie (CHU - BREST - Nephrologie), The Open University [Milton Keynes] (OU), William Harvey Research Institute, Barts and the London Medical School, University Medical Center Groningen [Groningen] (UMCG), Service de rhumatologie [Strasbourg], CHU Strasbourg-Hôpital de Hautepierre [Strasbourg], Immunologie, Immunopathologie et Chimie Thérapeutique (I2CT), Université de Strasbourg (UNISTRA)-Centre National de la Recherche Scientifique (CNRS)-Université de Strasbourg (UNISTRA)-Centre National de la Recherche Scientifique (CNRS)-Université de Strasbourg (UNISTRA)-Centre National de la Recherche Scientifique (CNRS), NIHR Biomedical Research Centre [London], Guy's and St Thomas' NHS Foundation Trust-King‘s College London, University Medical Center [Utrecht], NECESSITY WP5- STAR development working group : Suzanne Arends, Francesca Barone, Albin Björk, Coralie Bouillot, Guillermo Carvajal Alegria, Wen-Hung Chen, Kenneth Clark, Konstantina Delli, Liseth de Wolff, Jennifer Evans, Stéphanie Galtier, Saviana Gandolfo, Mickael Guedj, Dewi Guellec, Safae Hamkour, Dominik Hartl, Malin V Jonsson, Roland Jonsson, Frans G M Kroese, Aike Albert Kruize, Laurence Laigle, Véronique Le Guern, Wen-Lin Luo, Esther Mossel, Wan-Fai Ng, Gaëtane Nocturne, Marleen Nys, Roald Omdal, Jacques-Olivier Pers, Maggy Pincemin, Manel Ramos-Casals, Philippe Ravaud, Neelanjana Ray, Alain Saraux, Athanasios Tzioufas, Gwenny Verstappen, Arjan Vissink, Marie Wahren-Herlenius., and European Project: 806975,NECESSITY
- Subjects
[SDV.MHEP] Life Sciences [q-bio]/Human health and pathology ,Consensus ,[SDV]Life Sciences [q-bio] ,Immunology ,health care ,General Biochemistry, Genetics and Molecular Biology ,[SDV] Life Sciences [q-bio] ,Rheumatology ,patient reported outcome measures ,Sjogren's syndrome ,Outcome Assessment, Health Care ,Immunology and Allergy ,Humans ,Rituximab ,outcome assessment ,[SDV.MHEP]Life Sciences [q-bio]/Human health and pathology - Abstract
ObjectiveTo develop a composite responder index in primary Sjögren’s syndrome (pSS): the Sjögren’s Tool for Assessing Response (STAR).MethodsTo develop STAR, the NECESSITY (New clinical endpoints in primary Sjögren’s syndrome: an interventional trial based on stratifying patients) consortium used data-driven methods based on nine randomised controlled trials (RCTs) and consensus techniques involving 78 experts and 20 patients. Based on reanalysis of rituximab trials and the literature, the Delphi panel identified a core set of domains with their respective outcome measures. STAR options combining these domains were proposed to the panel for selection and improvement. For each STAR option, sensitivity to change was estimated by the C-index in nine RCTs. Delphi rounds were run for selecting STAR. For the options remaining before the final vote, a meta-analysis of the RCTs was performed.ResultsThe Delphi panel identified five core domains (systemic activity, patient symptoms, lachrymal gland function, salivary gland function and biological parameters), and 227 STAR options combining these domains were selected to be tested for sensitivity to change. After two Delphi rounds, a meta-analysis of the 20 remaining options was performed. The candidate STAR was then selected by a final vote based on metrological properties and clinical relevance.ConclusionThe candidate STAR is a composite responder index that includes all main disease features in a single tool and is designed for use as a primary endpoint in pSS RCTs. The rigorous and consensual development process ensures its face and content validity. The candidate STAR showed good sensitivity to change and will be prospectively validated by the NECESSITY consortium in a dedicated RCT.
- Published
- 2022