Pharmacological therapy in a rare disease : challenges in the long-term management of granulomatosis with polyangiitis

Abstract

Granulomatosis with polyangiitis (GPA) is an anti-neutrophil cytoplasmic antibody (ANCA)-associated vasculitis with an annual global incidence between 1 in 84,000 to 475,000 persons. This notably wide epidemiologic estimate may be due to the different methods used to obtain single estimates as well the diversity of the estimates themselves in published literature. GPA is managed pharmacologically in an initial phase aimed at inducing remission and a later phase aimed at maintaining remission. However, most drug use in GPA is off-label. Real-world data (RWD) is valuable in providing detailed, large-scale information on drug use and safety, addressing existing information gaps. The aim of the present editorial is to critically review the challenges of long-term GPA treatment and identify potential solutions.