家长对利妥昔单抗治疗儿童肾病综合征有效性和安全性的看法：一项全国性多中心调查研究
Parental perspectives on the effectiveness and safety of rituximab in treating pediatric nephrotic syndrome: a nationwide multi-center survey study.
作者信息
Lan Ning, Zhao Chengguang, Wang Li, Tang Hanyun, Zhou Weiran, Zhu Hongtao, Li Xiaoyan, Li Yuhong, Xu Ximing, Zhang Gaofu
机构信息
Department of Nephrology, Children's Hospital of Chongqing Medical University; National Clinical Research Center for Child Health and Disorders; Ministry of Education Key Laboratory of Child Development and Disorders; China International Science and Technology Cooperation Base of Child Development and Critical Disorders, Chongqing Key Laboratory of Pediatric Metabolism and Inflammatory Diseases, Chongqing, 400014, China.
Department of Pediatric Nephrology, Shengjing Hospital of China Medical University, Shenyang, 110004, Liaoning, China.
出版信息
Pediatr Nephrol. 2025 Jun 25. doi: 10.1007/s00467-025-06862-7.
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.
背景
利妥昔单抗越来越多地用于治疗依赖类固醇/频繁复发的肾病综合征，但家长们对其在现实世界中的影响的看法仍未得到充分研究。本研究评估了家长对利妥昔单抗的有效性、安全性和社会经济影响的看法。
方法
2025年2月在中国的八个儿科中心进行了一项全国性横断面调查。对570名肾病综合征患儿的家长发放了经过验证的问卷，评估利妥昔单抗的疗效、安全性、经济负担、照顾者生活质量（SF-36）和治疗障碍。多变量回归模型分析了治疗满意度和家长幸福感的预测因素。
结果
在分析的489名家长中，232名儿童（47.4%）接受了利妥昔单抗治疗。大多数家长（79.3%）报告疾病得到良好控制，90.5%的家长认为整体有改善，10.3%的患儿在接受利妥昔单抗治疗后实现持续完全缓解且无复发。关于安全性，86.2%的家长观察到呼吸道感染没有增加，98.3%的家长报告孩子的生活质量有所改善。经济负担影响了看法；家庭年收入低于3000美元的家长对利妥昔单抗疗效的满意度较低（OR = 0.22，95% CI：0.08 - 0.60，P = 0.004）。停用类固醇或生活质量有显著改善的患儿家长报告的SF-36得分更高。对免疫抑制的担忧（80.6%）和高成本仍然是主要障碍。
结论
本研究表明家长对利妥昔单抗在儿童肾病综合征患者的疗效、安全性和照顾者生活质量方面持积极看法。然而，经济挑战和安全担忧限制了其更广泛的应用。政策干预应优先考虑财政补贴、扩大保险覆盖范围，并实施多学科支持计划以满足照顾者的心理社会需求。加强治疗前咨询和减少类固醇使用的方案可能会进一步优化家长满意度和治疗效果。
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