Long-term outcomes after early treatment with rituximab for Japanese children with cyclosporine- and steroid-resistant nephrotic syndrome

BackgroundAlthough rituximab (RTX) may be effective treatment in children with nephrotic syndrome who are resistant to cyclosporine A and steroid (CsA-SRNS), long-term outcomes after B cell depleting therapy remain unclear.Case-diagnosis/treatmentWe retrospectively reviewed the clinical courses (median follow-up, 5.1years) of six CsA-SRNS children (three boys; median age at RTX, 4.2years) unresponsive to oral cyclosporine combined with 2 courses of intravenous methylprednisolone pulses, who received RTX within 6months after disease onset (median 11weeks). After initial RTX treatment (median two doses of 375mg/m(2)) followed by retreatment with intravenous methylprednisolone pulses and/or high-dose prednisolone, all patients achieved complete remission at a median of 158days. Although 17 relapses occurred in five patients during follow-up, all but one patient became steroid sensitive. Severe neutropenia and hypogammaglobulinemia developed in two and four patients, respectively. However, no life-threatening infections were identified in the cohort. At last visit (median age, 11.3years), all patients maintained complete remission without renal insufficiency.ConclusionsAlthough late-onset adverse events should be considered, particularly for young patients, early RTX treatment may have positive outcomes in children with CsA-SRNS in the long term.