Abstract
BackgroundRituximab 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.MethodsAll 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/m2 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.ResultsIn 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; P = 0.009).ConclusionThe safety and efficacy of rituximab treatment in patients with refractory nephrotic syndrome were confirmed in the real-world setting.Clinical trial registrationUMIN000014997.
Highlights
Nephrotic syndrome is a kidney disorder in which protein leaks from the blood to the urine
Our post-marketing surveillance study of the real-world safety and efficacy of rituximab in patients with refractory nephrotic syndrome found that the incidence and severity of Adverse drug reactions (ADRs) of infections and infestations was manageable, with Grade ≥ 3 ADRs of infections and infestations being reported in 4.9% of patients over the 2-year study period
The incidence and severity of ADRs of infection/infestation were lower in our study (24.0%, 235 of 981 patients; Grade ≥ 3: 4.9%, 48 of 981 patients) compared with that reported for previous clinical trials (90.7%, 49 of 54 patients; Grade ≥ 3: 9.3%; 5 of 54 patients; data calculated using the results of RCRNS-01 and RCRNS-02)
Summary
Nephrotic syndrome is a kidney disorder in which protein leaks from the blood (through the glomeruli) to the urine. In Japan, approximately 1300 new cases of nephrotic syndrome are reported annually to the Research Project for the Treatment of Chronic Pediatric Diseases, suggesting that 5 out of 100,000 children will develop nephrotic syndrome each year [3]. 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. Results 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. Conclusion The safety and efficacy of rituximab treatment in patients with refractory nephrotic syndrome were confirmed in the real-world setting.
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