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Abstract Number: 1599

Glucocorticoids versus Glucocorticoids Plus Cyclophosphamide in Eosinophilic Granulomatosis with Polyangiitis with Poor-Prognosis Factors: A Target Trial Emulation Study

Boris Sorin1, Matthias Papo1, Renato Alberto Sinico2, Vítor Silvestre Teixeira3, Nils Venhoff4, Maria-Letizia Urban5, Michele Iudici6, Juliane Mahrhold7, Francesco Locatelli8, Giulia Cassone9, Franco Schiavon10, Benjamin Seeliger11, Thomas Neumann12, Claudia Feder13, Claus Kroegel14, Matthieu Groh15, Chiara Marvisi16, Maxime Samson17, Thomas Barba18, David Jayne19, Arianna Troilo20, Jens Thiel20, Bernhard Hellmich21, Sara Monti22, Carlomaurizio Montecucco23, Carlo Salvarani24, Jean-Emmanuel Kahn25, Bernard Bonnotte26, Cécile-Audrey Durel27, Xavier Puéchal28, Luc Mouthon29, Loïc Guillevin30, Giacomo Emmi31, Augusto Vaglio32, Raphael Porcher33 and Benjamin Terrier34, and the French Vasculitis Study Group (FVSG) and the European EGPA Study Group (EESG), 1Department of Internal Medicine, Cochin Hospital, National Referral Center for Rare Systemic Autoimmune and Autoinflammatory Diseases of Ile de France, East and West, Paris Cité University, Assistance Publique – Hôpitaux de Paris, Paris, France, 2Nephrology and Dialysis Unit, IRCCS Humanitas Research Hospital, Milan, Italy, 3Department of Rheumatology, Faro Hospital, Algarve, Portugal, 4Medical Center - University of Freiburg, Internal Medicine, Department of Rheumatology and Clinical Immunology, Freiburg, Germany, 5Department of Experimental and Clinical Medicine, University of Florence, Florence, Italy, 6Department of Internal Medicine, Cochin Hospital, National Referral Center for Rare Systemic Autoimmune and Autoinflammatory Diseases of Ile de France, East and West, Paris Cité University, Assistance Publique – Hôpitaux de Paris and Division of rheumatology, Geneva University Hospitals and University of Geneva, Geneva, Switzerland, Paris, France, 7Department of Internal Medicine, Rheumatology, Pulmonology, Nephrology and Diabtologyy and Immunology, ERN-RITA Reference Center, Vasculitis-Center Tübingen-Kirchheim, Medius Kliniken Kirchheim, University of Tübingen, Kirchheim-Teck, Germany, 8Department of Rheumatology, IRCCS Policlinico S. Matteo Foundation, University of Pavia, Pavia, Italy, 9Clinical and Experimental Medicine PhD Program, Azienda USL-IRCCS di Reggio Emilia and Universita di Modena and Reggio Emilia, Modena, Italy, 10Department of Medicine DIMED, Division of Rheumatology, University of Padua, Padua, Italy, 11Department of Respiratory Medicine, Hannover Medical School, Hannover, Germany, 12Department of Rheumatology, Clinic of Internal Medicine III, Jena University Hospital, Jena, Germany and Division of Rheumatology, Immunology and Rehabilitation, Kantonsspital St Gallen, St Gallen, Switzerland, Jena, Germany, 13Department of Rheumatology, Clinic of Internal Medicine III, Jena University Hospital, Jena, Germany, 14Department of Pneumology and Allergology, Clinic of Internal Medicine I, Jena University Hospital, Jena, Germany, 15Foch, Suresnes, France, 16Rheumatology Unit Universita di Modena and Reggio Emilia, Modena, Italy, 17Department of Internal Medicine and Clinical Immunology, François-Mitterrand Teaching Hospital, University of Bourgogne-Franche-Comté, Dijon, France, 18Department of Internal Medicine, Hôpital Edouard Herriot, Lyon, France, 19University of Cambridge, Cambridge, United Kingdom, 20Department of Rheumatology and Clinical Immunology, Medical Center – University of Freiburg, Faculty of Medicine, Freiburg, Germany, 21Klinik für Innere Medizin, Rheumatologie, Pneumologie, Nephrologie und Diabetologie, Medius Kliniken, Akademisches Lehrkrankenhaus der Universität Tübingen, Kirchheim unter Teck, Germany, 22IRCCS Istituto Auxologico Italiano, Milan, Italy, 23IRCCS policlinico S. Matteo foundation, University of Pavia, Pavia, Italy, 24Azienda USL-IRCCS di Reggio Emilia and University of Modena and Reggio Emilia, Reggio Emilia, Italy, Reggio Emilia, Italy, 25Department of Internal Medicine, Ambroise Paré Hospital, Assistance Publique - Hôpitaux de Paris, Université de Versailles Saint-Quentin-en-Yvelines, Boulogne-Billancourt, France, Boulogne Billancourt, France, 26Department of Internal Medicine and Clinical Immunology, Dijon University Hospital, Dijon, France., Dijon, France, 27Edouard Herriot University Hospital, Lyon, France, 28National Referral Center For Rare Systemic Autoimmune Diseases, Paris, France, 29Service de Médecine Interne, Centre de Référence Maladies Systémiques Autoimmunes et Autoinflammatoires Rares d'Ile de France de l'Est et de l'Ouest, Hôpital Cochin, Assistance Publique-Hôpitaux de Paris (AP-HP), Paris, France, 30National Referral Center For Rare Systemic Autoimmune Diseases, Paris, Ile-de-France, France, 31Department of Medical, Surgery and Health Sciences, University of Trieste, and Clinical Medicine and Rheumatology Unit, Cattinara University Hospital, Trieste, Italy, 32Department of Biomedical, Experimental and Clinical Sciences "Mario Serio", University of Firenze, Florence, Italy, 33Department of Rheumatology, Bicêtre AP-HP Hôpital, Université Paris-Saclay, Paris, Ile-de-France, France, 34Service de Médecine interne, Hôpital Cochin, AP-HP, Paris, Ile-de-France, France

Meeting: ACR Convergence 2024

Keywords: ANCA associated vasculitis, cytokines, Eosinophilic Granulomatosus with Polyangiitis (Churg-Strauss)

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Session Information

Date: Sunday, November 17, 2024

Title: Vasculitis – ANCA-Associated Poster II

Session Type: Poster Session B

Session Time: 10:30AM-12:30PM

Background/Purpose: Eosinophilic granulomatosis with polyangiitis (EGPA) is a small-vessel necrotizing vasculitis characterized by asthma, blood and tissue eosinophilia and vasculitis affecting multiple organs. According to the European and French recommendations, patients with EGPA with poor-prognosis factors (1996 Five Factor Score [FFS] ≥1) are treated with the combination of glucocorticoids (GCs) and cyclophosphamide (CYC). However, the original data on which these recommendations are based are weak, which justifies the need to obtain data of good methodological quality. In the present study, using a large retrospective European database, we compared the efficacy of GCs plus CYC versus GCs alone for the treatment of EGPA with poor-prognosis in a large population of unselected patients.

Methods: We emulated a target trial using observational data from a European multicenter retrospective database. We included patients with newly diagnosed EGPA with a 1996 Five Factor Score (FFS) of at least 1, treated with GCs or GCs plus CYC between June 1985 and November 2018. Outcomes were all-severity relapse, major relapse, death at 12 months and GC-dependent asthma and/or ear nose and throat (ENT) manifestations at 24 months. Propensity score analysis was used to adjust for potential confounders.

Results: A total of 209 patients were included: 99 (47%) were male and the mean age at diagnosis was 52 (± 16 years); 54 (26%) were treated with GCs alone and 154 (74%) with GCs plus CYC. After weighting, the variables included in the PS estimation were balanced in the two groups and the risk of any relapse at 12 months (hazard ratio [HR]: 0.24, 95% CI [0.08-0.67], p =0.007), major relapse at 12 months (HR: 0.24, 95% CI [0.07-0.85], p =0.026) and the proportion of GC-dependent asthma and/or ENT manifestations at 24 months (odds ratio: 0.30, 95% CI [0.14-0.66], p =0.003) were lower in the GCs plus CYC group compared with the GCs alone group. At 24 months, 3 (8%) patients in the GCs group and 1 (1%) in the GCs plus CYC group had a severe infection and no patient developed cancer.

Conclusion: This target trial emulation study shows that the addition of CYC to GCs reduces the rate of vasculitis relapse and the rate of GCs-dependent asthma and/or ENT manifestations in patients with poor-prognosis EGPA.


Disclosures: B. Sorin: None; M. Papo: None; R. Sinico: None; V. Teixeira: None; N. Venhoff: AbbVie/Abbott, 1, 5, 6, AstraZeneca, 1, 6, Boehringer-Ingelheim, 1, 6, Bristol-Myers Squibb(BMS), 6, Celgene, 6, Chugai, 6, GlaxoSmithKlein(GSK), 1, 6, Janssen, 1, 6, Medac, 5, Novartis, 1, 5, 6, Pfizer, 1, 5, 6, Roche, 1, 6, UCB, 1, 6, Vifor, 1, 6; M. Urban: None; M. Iudici: None; J. Mahrhold: None; F. Locatelli: None; G. Cassone: None; F. Schiavon: None; B. Seeliger: None; T. Neumann: None; C. Feder: None; C. Kroegel: None; M. Groh: None; C. Marvisi: None; M. Samson: None; T. Barba: None; D. Jayne: Amgen, 2, 6, AstraZeneca, 2, 6, Aurinia, 4, Boehringer Ingelheim, 2, 6, Bristol-Myers Squibb(BMS), 2, 6, ChemoCentryx, 2, 6, Chinook, 1, CSL Vifor, 2, GSK, 1, 2, 6, Novartis, 2, 6, Roche, 2, 6, Takeda, 1, 2, 6, Vifor Pharma, 2, 6; A. Troilo: None; J. Thiel: None; B. Hellmich: AbbVie, 2, 6, Amgen, 2, 6, AstraZeneca, 2, 6, Boehringer-Ingelheim, 2, 6, Bristol Myers Squibb, 2, 6, Chugai, 2, 6, GlaxoSmithKline, 2, 6, InflaRx, 2, 6, Janssen, 2, 6, MSD, 2, 6, Novartis, 2, 6, Pfizer, 2, 6, Phadia, 2, 6, Roche, 2, 6, Vifor Pharma, 2, 6; S. Monti: AstraZeneca, 6, GlaxoSmithKlein(GSK), 6; C. Montecucco: None; C. Salvarani: None; J. Kahn: None; B. Bonnotte: None; C. Durel: None; X. Puéchal: None; L. Mouthon: None; L. Guillevin: None; G. Emmi: None; A. Vaglio: None; R. Porcher: None; B. Terrier: AstraZeneca, 2, GlaxoSmithKline, 2, Novartis, 2, Vifor Pharma, 2.

To cite this abstract in AMA style:

Sorin B, Papo M, Sinico R, Teixeira V, Venhoff N, Urban M, Iudici M, Mahrhold J, Locatelli F, Cassone G, Schiavon F, Seeliger B, Neumann T, Feder C, Kroegel C, Groh M, Marvisi C, Samson M, Barba T, Jayne D, Troilo A, Thiel J, Hellmich B, Monti S, Montecucco C, Salvarani C, Kahn J, Bonnotte B, Durel C, Puéchal X, Mouthon L, Guillevin L, Emmi G, Vaglio A, Porcher R, Terrier B. Glucocorticoids versus Glucocorticoids Plus Cyclophosphamide in Eosinophilic Granulomatosis with Polyangiitis with Poor-Prognosis Factors: A Target Trial Emulation Study [abstract]. Arthritis Rheumatol. 2024; 76 (suppl 9). https://acrabstracts.org/abstract/glucocorticoids-versus-glucocorticoids-plus-cyclophosphamide-in-eosinophilic-granulomatosis-with-polyangiitis-with-poor-prognosis-factors-a-target-trial-emulation-study/. Accessed .
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