Type I osteogenesis imperfecta is a genetic disease associated with mutations in the pro-alpha1 chains of type 1 collagen encoder gene COL1A1. It has an autosomal dominant pattern of inheritance, and leads to reduction in the amount of structurally normal collagen and subsequently, disorders of the extracellular membrane of skin, tendons and bone.
Objectives: Current guidelines suggest treating poor-prognosis eosinophilic granulomatosis with polyangiitis (EGPA) with a combination of glucocorticoids (GCs) plus cyclophosphamide (CYC). However, there is little data to support the need for the addition of CYC. The objective of this study was to compare GCs plus CYC to GCs alone as induction therapy in poor-prognosis EGPA. Methods: We emulated a target trial ...