Tacrolimus in Children With Henoch-Schönlein Purpura Nephritis

Henoch-Schönlein purpura (HSP) is the most common vasculitis in children, with an incidence of approximately 10:100 000 children and a slight male predominance (male-to-female ratio of 1.5:1). Henoch-Schönlein purpura nephritis (HSPN) is the principal cause of morbidity for HSP and 1%-7% of HSPN patients may progress to renal failure or end-stage renal disease. Immunosuppressive therapy has become the standard treatment in children with HSPN, however the use of these drugs are still mainly in an off-label manner in clinical practice. Tacrolimus, a calcineurin inhibitor, has been recently suggested in the treatment of HSPN in children. However, the evidence-based clinical data are still limited. Given the potential benefits and unmet need in clinical practice, the purposes of this pilot study were to assess effectiveness and safety of tacrolimus in HSPN children and evaluate the potential impact of CYP3A5.