Efficacy of rituximab therapy in children with refractory nephrotic syndrome: a prospective observational study in
1Department of Nephrology and Rheumatology, Children's Hospital of Fudan University, Shanghai, 201102, China.
Insights
Rituximab (RTX) effectively treats refractory nephrotic syndrome in children, significantly reducing steroid use and relapses. The treatment showed a 91.67% efficacy rate, particularly benefiting minimal change disease patients.
Area of Science:
- Pediatric Nephrology
- Immunology
- Pharmacology
Background:
- Idiopathic nephrotic syndrome is a common childhood glomerular disease.
- Refractory cases pose significant treatment challenges.
- Rituximab (RTX) is explored for efficacy and safety in pediatric refractory nephrotic syndrome.
Purpose of the Study:
- To evaluate the efficacy and side-effects of rituximab (RTX) in children with refractory primary nephrotic syndrome.
- To assess RTX's impact on steroid dosage and relapse rates.
Main Methods:
- Twelve pediatric patients with steroid-dependent, frequently relapsing, or steroid-resistant nephrotic syndrome were enrolled.
- Rituximab was administered at 375 mg/m(2) body surface area, once or twice weekly.
- Patients' response, steroid dosage, and side-effects were monitored.
Main Results:
- Overall RTX efficacy was 91.67%, with steroid dosage reduction in 77.78% of patients.
- Significant decreases in mean steroid dosage (P=0.014) and recurrence rates (P<0.001) were observed post-RTX.
- Treatment outcomes were more favorable in minimal change disease (MCD) compared to focal segmental glomerulosclerosis (FSGS) (P=0.045).
- Common side-effects included skin rashes, hypotension, and fever; one patient had decreased immunoglobulin levels without serious infection.
Conclusions:
- Rituximab is an effective treatment for refractory nephrotic syndrome in children.
- RTX significantly reduces the need for steroids and other immunosuppressants.
- The drug demonstrates a favorable efficacy profile, especially in MCD cases.
Background:
Idiopathic nephrotic syndrome is the most common glomerular disease in children. This study was undertaken to observe the efficacy and side-effects of rituximab (RTX) in treating children with different types of refractory primary nephrotic syndrome.
Methods:
Twelve patients with steroid dependent nephrotic syndrome (SDNS), frequently relapsing nephritic syndrome (FRNS), and steroid resistant nephrotic syndrome (SRNS) were enrolled in our study. There were obvious drug side-effects, and proteinuria remained difficult to control. RTX was administered at a dose of 375 mg/m(2) body surface area, once or twice weekly.
Results:
The male to female ratio was 3:1, and the onset age was 1.6-8.9 years. There were 9 patients with steroid sensitive nephrotic syndrome (SDNS or FRNS), and 3 patients with SRNS. There were 7 patients with minimal change disease (MCD), 3 patients with focal segmental glomerular sclerosis (FSGS), 1 with focal proliferative glomerulonephritis, and 1 without renal biopsy. The total effective treatment rate of RTX was 91.67%, and for 77.78% of the patients, steroid dosage could be reduced. Six months before and after RTX infusion, the mean steroid dosage was significantly decreased (P=0.014) and the recurrence number was significantly reduced (P<0.001). The results were better in MCD patients than in FSGS patients (P=0.045). There was no significant difference between FRNS/SDNS and SRNS patients (P=0.175). During RTX administration, 3 patients developed skin rashes, 1 developed hypotension, and 1 developed a fever. One patient experienced a persistent decrease in serum immunoglobulin level but without serious infection.
Conclusion:
RTX was effective in the treatment of refractory nephrotic syndrome, and it could significantly reduce the use of steroid and immunosuppressants.
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