Early Rituximab as an Add-On Therapy in Children With the Initial Episode of Nephrotic Syndrome

IntroductionThe approximately 70% 12-month relapse in children experiencing the initial episode of steroid-sensitive nephrotic syndrome (SSNS) is a significant concern, with over 50% developing frequent relapses or steroid-dependent nephrotic syndrome (FRNS/SDNS). There is a lack of strategies to reduce relapse after the onset, whether early administration of rituximab, which effectively reduces relapses in FRNS/SDNS, may be a solution has not been evaluated.MethodsA prospective, multicentre, open-label, single-arm trial was conducted in China, with a 12-month follow-up. Children aged 1 to 18 years with the first episode of NS were screened for eligibility. Proteinuria was evaluated daily using dipsticks. A dose of 375 mg/m2 of rituximab was intravenously infused within one week after achieving corticosteroid-induced remission. The main outcome was twelve-month relapse-free survival.ResultsOut of the initially screened 66 children, 44 were enrolled and received rituximab, with all but one participant completing the 12-month follow-up. The median age at diagnosis was 4.3 years (interquartile range 3.4-5.9), and 33 (77%) of the participants were male. In rituximab group, the 12-month relapse-free survival was significantly higher compared to historical controls (32/43 [74.4%] vs 10/33 [30.3%]; P <0.001; hazard ratio, 3.76; 95% confidence interval, 1.80-7.81). The post hoc analysis revealed a higher 24-month relapse-free survival and a lower incidence of FRNS/SDNS at the 12-month follow-up. Treatment with rituximab was well-tolerated.ConclusionOur findings support early administration of rituximab may be associated with a higher 12-month relapse-free survival and a reduced incidence of FRNS/SDNS in children experiencing the initial episode of SSNS.