Rituximab in children with steroid-dependent nephrotic syndrome: experience of a tertiary center and review of the literature

Abstract

Objectives: Rituximab (RTX) is a new treatment option in children with difficult-to-treat steroid-dependent nephrotic syndrome (SDNS). We evaluated the experience of our tertiary center and reviewed the current literature.

Methods: This is a retrospective single-center study evaluating the efficacy and safety of RTX in children with difficult-to-treat SDNS. Age at diagnosis, type and duration of immunosuppression, age at administration, dose of RTX, possible adverse events, number of relapses, duration of remission, and B-cell count after administration of RTX were analyzed.

Results: Nine children with a median age at diagnosis of nephrotic syndrome of 4.75 (range 1.33-11.33) years and a median age at administration of RTX of 16.08 (range 3.33-19.25) years were included. Before administration of RTX they had a median number of relapses per year of 1.70 (range 0.82-4.80). At last follow-up (median 2.75 years, range 0.58-3.92), a reduction in the number of relapses per year to 0.26 (range 0-2.18) was noted, despite cessation or lowering the dose of immunosuppressive therapy. Four patients achieved complete remission after the first administration of RTX, four more patients after subsequent doses of RTX. No severe adverse events were noted.

Conclusion: RTX was an effective and safe therapeutic option in our cohort of children with difficult-to-treat SDNS, resulting in a significant reduction of yearly relapses in the absence of severe adverse events and facilitating the reduction of other immunosuppressive medication.

Keywords: Children; Rituximab; Steroid-dependent nephrotic syndrome.