Michele Iudici1, Christian Pagnoux2, Delphine S Courvoisier3, Pascal Cohen4, Mohamed Hamidou5, Achille Aouba6, François Lifermann7, Marc Ruivard8, Olivier Aumaître8, Bernard Bonnotte9, François Maurier10, Olivier Decaux11, Eric Hachulla12, Alexandre Karras13, Chahéra Khouatra14, Noémie Jourde-Chiche15, Jean-François Viallard16, Claire Blanchard-Delaunay17, Pascal Godmer18, Alain Le Quellec19, Thomas Quéméneur20, Claire de Moreuil21, Alexis Régent4, Benjamin Terrier4, Luc Mouthon4, Loïc Guillevin4, Xavier Puéchal22. 1. National Referral Center for Rare Systemic Autoimmune Diseases, Université Paris Descartes, Hôpital Cochin, Assistance Publique-Hôpitaux de Paris (APHP), 27, rue du faubourg Saint-Jacques, Paris, Cedex 14 75679, France; Division of Rheumatology, Department of Internal Medicine Specialties, Geneva University Hospitals, Switzerland. 2. National Referral Center for Rare Systemic Autoimmune Diseases, Université Paris Descartes, Hôpital Cochin, Assistance Publique-Hôpitaux de Paris (APHP), 27, rue du faubourg Saint-Jacques, Paris, Cedex 14 75679, France; Present address: Vasculitis Clinic, Mount Sinai Hospital, University of Toronto, Toronto, Canada. 3. Division of Rheumatology, Department of Internal Medicine Specialties, Geneva University Hospitals, Switzerland. 4. National Referral Center for Rare Systemic Autoimmune Diseases, Université Paris Descartes, Hôpital Cochin, Assistance Publique-Hôpitaux de Paris (APHP), 27, rue du faubourg Saint-Jacques, Paris, Cedex 14 75679, France. 5. Department of Internal Medicine, Centre Hospitalier Universitaire (CHU) Hôtel-Dieu, Nantes, France. 6. Department of Internal Medicine, CHU Côte-de-Nacre, Caen, France. 7. Department of Internal Medicine, CH Côte-d'Argent, Dax, France. 8. Department of Internal Medicine, CHU, Clermont-Ferrand, France. 9. Department of Internal Medicine, CHU du Bocage, Dijon, France. 10. Department of Internal Medicine, HP, Metz, France. 11. Department of Internal Medicine, CHU Sud, Rennes, France. 12. National Referral Center for Rare Systemic Autoimmune Diseases, Department of Internal Medicine and Clinical Immunology, Claude-Huriez Hospital, University of Lille, Lille, France. 13. Department of Nephrology, Hôpital Européen Georges-Pompidou, APHP, Paris, France. 14. Department of Respiratory Medicine, CHU Louis-Pradel and UMR754, Université Claude-Bernard Lyon 1, Lyon, France. 15. Aix-Marseille Univ, C2VN, INSERM 1263, INRAE 1260, AP-HM, Department of Nephrology, CHU de la Conception, Marseille, France. 16. Department of Internal Medicine, CHU Haut-Lévêque, Bordeaux, France. 17. Department of Internal Medicine, Centre Hospitalier, Niort, France. 18. Department of Internal Medicine, Centre Hospitalier Bretagne Atlantique, Vannes, France. 19. Department of Internal Medicine, CHU Saint-Eloi, Montpellier, France. 20. Department of Nephrology and Internal Medicine, Centre Hospitalier, Valenciennes, France. 21. Department of Internal Medicine, CHU La Cavale Blanche, Brest, France. 22. National Referral Center for Rare Systemic Autoimmune Diseases, Université Paris Descartes, Hôpital Cochin, Assistance Publique-Hôpitaux de Paris (APHP), 27, rue du faubourg Saint-Jacques, Paris, Cedex 14 75679, France. Electronic address: xavier.puechal@aphp.fr.
Abstract
OBJECTIVE: To describe the characteristics and long-term outcomes of patients with granulomatosis with polyangiitis (GPA) from the French Vasculitis Study Group database. METHODS: Patients' clinical and laboratory characteristics, Birmingham Vasculitis Activity Score (BVAS)-assessed disease activity, malignancies, opportunistic infections, and vital status were collected at diagnosis and each visit. Estimated probabilities and predictors of overall (OS) and relapse-free survival (RFS) were analyzed by Cox regression. RESULTS: We enrolled 795 newly diagnosed patients, followed for a median of 3.5 years. Initial clinical manifestations involved ear, nose & throat (ENT; 80%), lungs (68%) and kidneys (56%). Among the 728 available ELISA results, 75.0% were PR3-ANCA-positive, 16.5% MPO-ANCA-positive and 62 (8.5%) ANCA-negative. Relapses occurred in 394 (50%) patients, involving ≥1 organ(s) affected at onset in 179 (46%), mainly ENT, lungs and kidneys, with mean BVAS 10.2 points below that at diagnosis (p<0.001). Five- and 10-year RFS rates were 37% and 17%, respectively. PR3-ANCA-positivity independently predicted relapse (p = 0.05) and prolonged survival (p = 0.038). OS-but not RFS-improved significantly over time (p<0.001); 10-year OS reached 88.2% (95% CI 83.9 to 92.7) for the 660 patients diagnosed after 2000. Infections were the main causes of death. Malignancy or opportunistic infection each occurred in ≤5% of the patients. CONCLUSION: Survival has improved dramatically over the last decades but the high relapse rate remains a major concern for GPA patients, once again stressing the need for therapeutic strategy optimization to lower it. PR3-ANCA-positivity was associated with increased probability of relapse and survival.
OBJECTIVE: To describe the characteristics and long-term outcomes of patients with granulomatosis with polyangiitis (GPA) from the French Vasculitis Study Group database. METHODS:Patients' clinical and laboratory characteristics, Birmingham Vasculitis Activity Score (BVAS)-assessed disease activity, malignancies, opportunistic infections, and vital status were collected at diagnosis and each visit. Estimated probabilities and predictors of overall (OS) and relapse-free survival (RFS) were analyzed by Cox regression. RESULTS: We enrolled 795 newly diagnosed patients, followed for a median of 3.5 years. Initial clinical manifestations involved ear, nose & throat (ENT; 80%), lungs (68%) and kidneys (56%). Among the 728 available ELISA results, 75.0% were PR3-ANCA-positive, 16.5% MPO-ANCA-positive and 62 (8.5%) ANCA-negative. Relapses occurred in 394 (50%) patients, involving ≥1 organ(s) affected at onset in 179 (46%), mainly ENT, lungs and kidneys, with mean BVAS 10.2 points below that at diagnosis (p<0.001). Five- and 10-year RFS rates were 37% and 17%, respectively. PR3-ANCA-positivity independently predicted relapse (p = 0.05) and prolonged survival (p = 0.038). OS-but not RFS-improved significantly over time (p<0.001); 10-year OS reached 88.2% (95% CI 83.9 to 92.7) for the 660 patients diagnosed after 2000. Infections were the main causes of death. Malignancy or opportunistic infection each occurred in ≤5% of the patients. CONCLUSION: Survival has improved dramatically over the last decades but the high relapse rate remains a major concern for GPA patients, once again stressing the need for therapeutic strategy optimization to lower it. PR3-ANCA-positivity was associated with increased probability of relapse and survival.
Authors: Xavier Puéchal; Michele Iudici; Christian Pagnoux; Pascal Cohen; Mohamed Hamidou; Achille Aouba; François Lifermann; Marc Ruivard; Olivier Aumaître; Bernard Bonnotte; Francois Maurier; Thomas Le Gallou; Eric Hachulla; Alexandre Karras; Chahéra Khouatra; Noémie Jourde-Chiche; Jean-François Viallard; Claire Blanchard-Delaunay; Pascal Godmer; Alain Le Quellec; Thomas Quéméneur; Claire de Moreuil; Luc Mouthon; Benjamin Terrier; Loïc Guillevin Journal: RMD Open Date: 2022-03