抗CD20单抗在儿童难治性肾病综合征中的临床应用进展
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篇名: 抗CD20单抗在儿童难治性肾病综合征中的临床应用进展
TITLE: Progress in clinical application of anti-CD20 monoclonal antibody in refractory nephrotic syndrome in children
摘要: 抗CD20单抗在儿童难治性肾病综合征(RNS)的治疗中具有有效性。第一代利妥昔单抗的临床应用最为广泛,其对RNS患儿的疗效确切,获得了指南推荐,尤其对微小病变性肾病的缓解率较高,可显著减少激素和免疫抑制剂的累积使用量;第二代奥法妥木单抗在利妥昔单抗不耐受或耐药患者中有替代治疗潜力;第三代奥妥珠单抗则在利妥昔单抗耐药、移植后复发等复杂病例中显现出较好的疗效。但关于利妥昔单抗的治疗剂量优化,以及奥法妥木单抗和奥妥珠单抗相较于利妥昔单抗治疗儿童RNS是否更具优势,仍存在一定争议。抗CD20单抗最常见的不良反应是输液反应,长期不良事件主要是持续免疫抑制和感染风险增加。利妥昔单抗在儿童RNS的治疗中具有显著的经济性优势,今后需开展基于中国医疗环境的药物经济学研究以评价奥法妥木单抗和奥妥珠单抗在儿童RNS中的经济性。由于目前奥法妥木单抗与奥妥珠单抗在该领域的使用仍属于超说明书用药,临床须在严格评估患者获益和风险后使用。
ABSTRACT: Anti-CD20 monoclonal antibodies for the treatment of refractory nephrotic syndrome (RNS) in children. The first- generation rituximab is the most widely used in clinical practice; it shows definite efficacy in children with RNS, is recommended by guidelines, particularly for achieving a high remission rate in minimal change nephrosis, and can significantly reduce the cumulative use of glucocorticoids and immunosuppressants. The second-generation ofatumumab has potential as an alternative treatment for patients who are intolerant or resistant to rituximab, while the third-generation obinutuzumab has shown efficacy in complex cases such as rituximab resistance or post-transplant recurrence. However, there is still controversy regarding the optimization of rituximab treatment dosage and whether ofatumumab and obinutuzumab offer greater advantages than rituximab for the treatment of RNS in children. The most common adverse reaction induced by anti-CD20 monoclonal antibodies is infusion reactions, and long-term adverse events mainly include increased risks of sustained immunosuppression and infections. Rituximab has significant economic advantages for the treatment of RNS, but additional pharmacoeconomic research based on China’s healthcare environment is needed to evaluate the cost-effectiveness of ofatumumab and obinutuzumab in this population. Given that the current use of ofatumumab and obinutuzumab in this field is considered off-label use, clinical application should only proceed after a rigorous evaluation of the patient’s benefits and risks.
期刊: 2025年第36卷第16期
作者: 向金波;沈官国;李政;胡小燕;曹婷婷;徐自然;丁婷;李静波
AUTHORS: XIANG Jinbo,SHEN Guanguo,LI Zheng,HU Xiaoyan,CAO Tingting,XU Ziran,DING Ting,LI Jingbo
关键字: 难治性肾病综合征;儿童;抗CD20单抗;利妥昔单抗;奥法妥木单抗;奥妥珠单抗
KEYWORDS: refractory nephrotic syndrome; children; anti-CD20 monoclonal antibody; rituximab; ofatumumab; obinutuzumab
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