A real-world assessment of mycophenolate mofetil for remission induction in eosinophilic granulomatosis with polyangiitis

Abstract

Eosinophilic granulomatosis with polyangiitis (EGPA) is a form of ANCA-associated vasculitis (AAV). Clinical trials demonstrating the efficacy of mycophenolate mofetil (MMF) for remission induction in AAV excluded patients with EGPA. Despite this, MMF is commonly used in these patients. The objective of this study was to evaluate, for the first time, the effectiveness and tolerance of MMF in EGPA remission induction. A retrospective, two-center, real-world study was conducted in patients with EGPA who received MMF in addition to prednisolone for newly diagnosed or relapsing disease between 2009 and 2019. Baseline, 3-, 6- and 12-month outcome data were extracted from electronic health records. The primary outcome was disease remission, defined as a Birmingham Vasculitis Activity Score of 0 at 6 months. Secondary outcomes included disease relapse, median prednisolone dose at 12 months and drug tolerance. In total, 15 patients (73% male, median age 57) with EGPA (11 newly diagnosed/4 relapsing) were identified. At 6 months, 67% had achieved disease remission. At 12 months, this was maintained (66.7%) and 4 patients had relapsed. All but one patient remained on MMF at study completion and all patients tolerated MMF. Our real-world data suggest that MMF is an effective and well-tolerated agent for achieving disease remission in EGPA. A future randomized controlled trial of MMF in this neglected orphan disease is now warranted.

Evidence-Based Guideline for the diagnosis and management of eosinophilic granulomatosis with polyangiitis

“…In patients whose disease does not respond to these approaches, different therapeutic options can be considered, including other anti-IL-5 agents (Statement 13), plasma exchange and intravenous immunoglobulin therapy; anti-IgE agents have also been tried but with unsatisfactory results 73,[79][80][81][82] . In selected patients, the use of IFNα 83 or mycophenolate mofetil can also be considered for remission induction 84 . However, no solid evidence supports their use as maintenance therapy.…”

Therapeutic advances in eosinophilic granulomatosis with polyangiitis

“…Despite paucity of data, MTX, MMF and AZA are still often employed clinically in addition to glucocorticoid in nonsevere disease in both induction and remission phases in the spirit of steroid-sparing, and these traditional DMARDs and glucocorticoid are favoured over glucocorticoid monotherapy in the recent 2021 ACR/VF guidelines [2 ▪▪ ]. Two recent small retrospective studies of traditional DMARD use for remission induction in EGPA have shown favourable results [28 ▪ ,29 ▪ ], including a study of MMF in 15 newly diagnosed EGPA patients in which two-third achieved remission at 6 months with median prednisone dose 7.5 mg/day [29 ▪ ]. Larger, controlled trials are certainly needed.…”

Eosinophilic granulomatosis with polyangiitis: case report and literature review

“…In a prospective trial of 28 patents with EGPA, methotrexate, when given alongside prednisolone, achieved remission in 72% but relapse occurred in 50% within 1 year [ 60 ]. A small observational study of 15 patients with newly diagnosed EGPA reported remission in 67% of patients who received MMF together with prednisolone at 12 months [ 61 ]. However, MMF was found to be less effective than azathioprine in non-EGPA AAVs when used for remission maintenance [ 62 ].…”