Preliminary experience with tacrolimus in managing Henoch-Schönlein purpura featuring extrarenal symptoms refractory to conventional therapy
Abstract Background Henoch-Schönlein purpura (HSP) is the commonest vasculitis of childhood, affecting multiple organ systems. The extrarenal manifestations are prominent in some HSP cases, for which no standard treatment is available. It is interesting to examine the value of tacrolimus in such cases. Findings Seventeen children received tacrolimus to manage their refractory extrarenal symptoms of HSP in an open-label prospective study. Tacrolimus was administered at daily dose of 0.05–0.15 mg/kg. Tacrolimus was effective in all cases after treatment for 7–21 days. Tacrolimus treatment resulted in complete resolution of extrarenal symptoms in 15 cases, and partial resolution in the remaining 2 cases. During follow-up, skin purpura reoccurred in 2 cases, abdominal pain and loss of follow-up in 1 case each. Drug-related adverse reaction was not observed in any case. Abnormal renal function test was identified in 7 patients by urinary screening tests before tacrolimus therapy, 4 of them received renal biopsy. Only one patient showed evidence of renal impairment during follow-up. Conclusions Tacrolimus works quickly without drug-related adverse reaction in managing the HSP patients featuring recurrent extrarenal symptoms. Our preliminary experience suggests good clinical efficacy and safety of tacrolimus in such off-label use.