静脉血栓栓塞症（venous thromboembolism，VTE）在儿童中较成人罕见，然而近年来儿童VTE的发病率显著增加，临床上儿童患者对于有充分疗效和安全性证据的治疗方案存在需求。儿童VTE药物研发面临很多挑战，基于科学分析进行成人疗效数据外推，可以减少不必要的儿童人群临床研究，促进儿童药物研发和可及性。利伐沙班是口服凝血因子Xa抑制剂，基于成人VTE数据外推和儿童临床研究数据的支持，近期已在多国获批用于儿童VTE的治疗和预防复发。通过介绍成人数据外推的一般原则和国外相关指导原则，结合利伐沙班审评案例，对VTE适应症成人用药数据外推儿童的临床研发策略进行讨论，以期为该类药物的研发和评价提供参考。

Venous thromboembolism (VTE) is rarer in children than adults. However, the incidence of VTE in children has increased significantly in recent years. There is a need for treatment protocols with sufficient evidence of efficacy and safety in children. Since the development of drug for pediatric VTE faces many challenges, extrapolation of adult efficacy data to children based on scientific analysis could help to reduce unnecessary pediatric clinical trials and promote the development and accessibility of pediatric drugs. Rivaroxaban is an oral factor Xa inhibitor. Based on the extrapolation of adult data and the support of pediatric clinical data, it has recently been approved in many countries for the treatment and prevention of pediatric VTE. This article introduces the general principles of adult data extrapolation and related clinical guidelines abroad, and discusses the extrapolation strategy in pediatric VTE drug development, in combination with the review case of rivaroxaban, with a view to provide reference for research and evaluation.

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