靜脈血栓栓塞癥（venous thromboembolism，VTE）在兒童中較成人罕見，然而近年來兒童VTE的發(fā)病率顯著增加，臨床上兒童患者對(duì)于有充分療效和安全性證據(jù)的治療方案存在需求。兒童VTE藥物研發(fā)面臨很多挑戰(zhàn)，基于科學(xué)分析進(jìn)行成人療效數(shù)據(jù)外推，可以減少不必要的兒童人群臨床研究，促進(jìn)兒童藥物研發(fā)和可及性。利伐沙班是口服凝血因子Xa抑制劑，基于成人VTE數(shù)據(jù)外推和兒童臨床研究數(shù)據(jù)的支持，近期已在多國(guó)獲批用于兒童VTE的治療和預(yù)防復(fù)發(fā)。通過介紹成人數(shù)據(jù)外推的一般原則和國(guó)外相關(guān)指導(dǎo)原則，結(jié)合利伐沙班審評(píng)案例，對(duì)VTE適應(yīng)癥成人用藥數(shù)據(jù)外推兒童的臨床研發(fā)策略進(jìn)行討論，以期為該類藥物的研發(fā)和評(píng)價(jià)提供參考。

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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