Tofacitinib in combination with glucocorticoids in the treatment of eosinophilic granulomatosis with polyangiitis: a pilot study of 11 cases

Objective Current treatment for eosinophilic granulomatosis with polyangiitis (EGPA) is not always satisfactory due to refractory disease, relapse and drug toxicity. This study aims to investigate the efficacy and safety of tofacitinib, a Janus kinase (JAK) inhibitor, in the treatment of EGPA. Methods Eleven active patients receiving oral tofacitinib 5 mg twice daily from a prospective single-center cohort of EGPA were enrolled. Patients were followed up for 3–48 months. Treatment response, inflammatory parameters, peripheral eosinophils count and dose of glucocorticoids (GCs) were evaluated at 3, 6 and 9 months. The relapse rate and adverse events were recorded throughout the follow-up. Results Of these 11 EGPA patients, five were newly diagnosed and the other six were refractory to GCs treatment in combination with immunosuppressants previously. Three months after tofacitinib treatment, most patients (10/11) responded to tofacitinib with a rapid relief of clinical symptoms. The response rate (including complete remission (CR) and partial remission (PR)) was 100% at 6 and 9 months. The CR rate at 6 and 9 months was 60% and 80%, respectively. After treatment for 6 months, both the erythrocyte sedimentation rate (ESR) and C-reactive protein (CRP) levels had normalized, eosinophils count had significantly decreased, and the GCs dose had been tapered in all patients. There was no relapse during the median follow-up period of 15 months. Conclusions Tofacitinib, which can induce remission, prevent recurrence, and taper the GC dosage, was well tolerated and may be an effective agent for the treatment of EGPA.