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| 基于循证证据与区域医疗机构真实世界研究的利伐沙班治疗儿童静
脉血栓栓塞症临床综合评价 |
| 佟欣1,李璐娟1,许佳1,张博研1,李中1,王晓玲2 |
| (1. 大连市妇女儿童医疗中心(集团),辽宁大连 116000;2. 国家儿童医
学中心,首都医科大学附属北京儿童医院,北京 100045]) |
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| 摘要: |
| 目的:以利伐沙班为核心开展抗凝药物应用于儿童静脉血栓栓塞症(VTE)治疗的临床综合评价,促进儿童VTE 治疗的
规范化。方法:系统检索国内外相关指南、文献及临床试验数据,从有效性、安全性、经济性、适宜性、可及性、创新性6 个维度对
利伐沙班进行评价。结果:有效性,利伐沙班治疗下症状复发性VTE 发生率低于传统抗凝治疗,血栓完全再通率及血栓负荷改
善效果更优,且在复合终点获益显著,获多部临床指南/ 共识推荐。安全性,利伐沙班与传统抗凝治疗相比,大出血发生率降低,
整体安全性相当,不良反应以1~2 级(轻度)为主,仅对新生儿或6 月龄以下、严重肝肾功能不全患儿存在使用限制。经济性,
仿制片剂日均费用0. 88 元,因无需常规监测,总费用低于华法林。适宜性,利伐沙班为国内唯一获批儿童VTE 适应证的口服
抗凝药物,拥有片剂、颗粒剂、干混悬剂等儿童适宜剂型,可解决低龄患儿给药难题,用药依从性优于肝素类及华法林。可及性,
利伐沙班已纳入国家医保及基本药品目录,被调研医疗机构中配备片剂,均未配备颗粒剂、干混悬剂,华法林基层医疗机构的配
备率偏低;仿制片剂可负担性最优,原研制剂经济负担较高。创新性,利伐沙班为高选择性直接Xa 因子抑制剂,全合成化合物
制剂性能均一,儿童专用干混悬剂为新生儿VTE 唯一获批口服抗凝剂型。结论:基于现有证据,利伐沙班治疗儿童VTE 的有效
性优于传统抗凝药物,安全性相当,仿制制剂经济性突出,且剂型适配性、依从性高。但其儿童适宜剂型覆盖率低、医保支付未
覆盖儿童适应证,且国内真实世界证据不足,限制了临床可及性。 |
| 关键词: 利伐沙班 静脉血栓栓塞症 儿童 抗凝药物 临床综合评价 |
| DOI：doi:10.13407/j.cnki.jpp.1672-108X.2026.08.013 |
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| 基金项目: |
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| Clinical Comprehensive Evaluation of Rivaroxaban in the Treatment of Venous Thromboembolism in ChildrenBased on Evidence-Based Research and Real-World Studies of Regional Medical Institutions |
| Tong Xin1, Li Lujuan1, Xu Jia1, Zhang Boyan1, Li Zhong1, Wang Xiaoling2 |
| (1. Dalian Women and Children’s Medical
Center (Group), Liaoling Dalian 116000, China; 2. Beijing Children’s Hospital, Capital Medical University, Beijing 100045, China) |
| Abstract: |
| Objective: To perform comprehensive clinical evaluation of anticoagulant drugs, with rivaroxaban as the core, in the
treatment of venous thromboembolism (VTE) in children, and to promote the standardization of VTE treatment in children. Methods:
Domestic and foreign guidelines, literature and clinical trial data were systematically retrieved, rivaroxaban was evaluated from 6
dimensions: effectiveness, safety, economy, suitability, accessibility and innovation. Results: In terms of effectiveness, compared with
traditional anticoagulants, rivaroxaban significantly reduced the incidence of recurrent VTE in children. It achieved higher rates of
complete thrombus recanalization and greater reduction in thrombotic burden, demonstrating significant benefits across composite
endpoints, which was recommended by multiple clinical guidelines/ consensus. In terms of safety, compared with traditional
anticoagulants, rivaroxaban had lower incidence of major bleeding. The overall safety is comparable. Adverse events were predominantly
grade 1 to 2 (mild) in severity. Application was restricted in neonates or infants aged <6 months and in children with severe hepatic or
renal impairment. In terms of economy, the daily cost of generic rivaroxaban tablets was 0. 88 RMB, the total treatment costs were lower
than those of warfarin owing to the elimination of routine monitoring requirements. In terms of suitability, rivaroxaban was the only oral
anticoagulant approved for pediatric VTE in China, its pediatric-specific formulations (tablets, granules, and oral suspensions) could
effectively address dosing challenges in young children, and the medication compliance was better than that of heparin and warfarin. In
terms of accessibility, rivaroxaban was included in the National Medical Insurance and Essential Medicines List of China. In the
surveyed medical institutions, only tablets were available, while granules and dry suspensions were not. Warfarin availability in primary
care settings was relatively limited. Generic tablets demonstrated optimal affordability, whereas originator brands imposed a higher
economic burden. In terms of innovation, rivaroxaban was a highly selective direct factor Xa inhibitor, and the performance of the fully
synthetic compound formulation was uniform. Notably, its dry powder for oral suspension was the only oral anticoagulant formulation
approved for neonatal VTE. Conclusion: Based on current evidence, rivaroxaban is more effective than traditional anticoagulants in the
treatment of VTE in children, with similar safety, outstanding economy of generics, and high dosage form adaptation and compliance.
However, the low coverage of appropriate dosage forms for children, the lack of coverage of children’s indications by medical insurance
payments, and the lack of real-world evidence in China restrict the clinical accessibility. |
| Key words: rivaroxaban venous thromboembolism chirdren anticoagulants comprehensive clinical evaluation |
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