Parental perspectives on the effectiveness and safety of rituximab in treating pediatric nephrotic syndrome: a nationwide multi-center survey study.

BACKGROUND

Rituximab is increasingly used for steroid-dependent/frequently relapsing nephrotic syndrome, yet parental perspectives on its real-world impact remain understudied. This study evaluated parental perceptions of rituximab's effectiveness, safety, and socioeconomic effects.

METHODS

A nationwide cross-sectional survey was conducted across eight pediatric centers in China (Feb 2025). Validated questionnaires were administered to 570 parents of children with nephrotic syndrome, assessing rituximab efficacy, safety, financial burden, caregiver quality of life (SF-36), and treatment barriers. Multivariable regression models analyzed predictors of treatment satisfaction and parental well-being.

RESULTS

Among 489 parents analyzed, 232 children (47.4%) received rituximab. Most parents (79.3%) reported good disease control, and 90.5% perceived overall improvement, with 10.3% achieving sustained complete remission without relapse after rituximab treatment. Regarding safety, 86.2% of parents observed no increase in respiratory infections, and 98.3% reported improved child quality of life. Financial burden influenced perceptions; parents from households earning < $3000 annually were less satisfied with rituximab efficacy (OR = 0.22, 95% CI: 0.08-0.60, P = 0.004). Higher SF-36 scores were reported by parents of children who discontinued steroids or had significant quality-of-life improvements. Immune suppression concerns (80.6%) and high costs remained major barriers.

CONCLUSIONS

This study demonstrates favorable parental perceptions of rituximab regarding efficacy, safety, and caregiver quality of life in patients with pediatric nephrotic syndrome. However, financial challenges and safety concerns limit its broader use. Policy interventions should prioritize financial subsidies, expand insurance coverage, and implement multidisciplinary support programs to address caregivers' psychosocial needs. Enhanced pre-treatment counseling and steroid-sparing protocols may further optimize parental satisfaction and treatment outcomes.