All-case Japanese post-marketing surveillance of the real-world safety and efficacy of rituximab treatment in patients with refractory nephrotic syndrome

<jats:title>Abstract</jats:title><jats:sec> <jats:title>Background</jats:title> <jats:p>Rituximab is conditionally approved in Japan for use in patients with refractory nephrotic syndrome. To meet the conditions of approval, an all-case post-marketing surveillance study was conducted to confirm the real-world safety and efficacy of rituximab in patients of all ages with refractory nephrotic syndrome.</jats:p> </jats:sec><jats:sec> <jats:title>Methods</jats:title> <jats:p>All patients scheduled to receive rituximab treatment for refractory nephrotic syndrome were eligible to register (registration: August 29, 2014 through April 15, 2016); the planned observation period was 2 years from the initiation of rituximab treatment (intravenous infusion, 375 mg/m<jats:sup>2</jats:sup> once weekly for four doses). The study was conducted at 227 hospitals throughout Japan. Adverse drug reactions (ADRs) were collected for safety outcomes. The efficacy outcomes were relapse-free period and the degree of growth in pediatric (< 15 years) patients.</jats:p> </jats:sec><jats:sec> <jats:title>Results</jats:title> <jats:p>In total, 997 (447 pediatric) patients were registered; 981 (445) were included in the safety analysis set; 852 (402) completed the 2-year observation period; and 810 (429) were included in the efficacy analysis set. Refractory nephrotic syndrome had developed in childhood for 85.0% of patients, and 54.6% were aged ≥15 years. ADRs were observed in 527 (53.7%) patients, treatment-related infection/infestation in 235 (24.0%) patients, and infusion reactions in 313 (31.9%) patients. The relapse-free period was 580 days (95% confidence interval, 511–664). There was a significant change in height standard deviation score (pediatric patients; mean change, 0.093; standard deviation, 0.637; <jats:italic>P</jats:italic> = 0.009).</jats:p> </jats:sec><jats:sec> <jats:title>Conclusion</jats:title> <jats:p>The safety and efficacy of rituximab treatment in patients with refractory nephrotic syndrome were confirmed in the real-world setting.</jats:p> </jats:sec><jats:sec> <jats:title>Clinical trial registration</jats:title> <jats:p>UMIN000014997.</jats:p> </jats:sec>