Session Information
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.
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 .« Back to ACR Convergence 2024
ACR Meeting Abstracts - https://acrabstracts.org/abstract/glucocorticoids-versus-glucocorticoids-plus-cyclophosphamide-in-eosinophilic-granulomatosis-with-polyangiitis-with-poor-prognosis-factors-a-target-trial-emulation-study/