艾滋病免疫功能重建不全者治疗药物临床试验设计的关键考量  

Key consideration on the clinical trial design of therapeutic drugs for immunological non-responder with AIDS

作　　者：何春俐 赵建中 HE Chunli;ZHAO Jianzhong(Center for Drug Evaluation,National Medical Products Administration,Beijing 100076,China)

机构地区：[1]国家药品监督管理局药品审评中心,北京100076

出　　处：《中国艾滋病性病》2024年第9期983-987,共5页Chinese Journal of Aids & STD

摘　　要：随着cART的广泛应用,大部分HIV感染者体内病毒得到控制,有效延缓了疾病进展,使HIV感染从一种致命性疾病变为可治疗的慢性疾病。但达到病毒学抑制后,部分患者CD4细胞恢复不佳,不能完全实现免疫重建,其机会性感染、非艾滋病并发症和肿瘤等的发病率和病死率均更高。针对艾滋病免疫功能重建不全的治疗,目前已有多个新药进入临床研发阶段,但国内外尚无相关临床试验技术指导原则。本文通过综述免疫功能重建不全者治疗药物的临床试验概况,对临床试验重点关注的要素进行讨论,包括试验设计、试验人群、主要疗效指标、治疗周期等,以期为药物临床研发提供参考。With the widespread application of combination antiretroviral therapy(cART),the majority of HIVinfected individuals have achieved viral control,effectively prevented disease progression.Nowadays,HIV infection has been transformed from a fatal disease into a chronic disease.However,following the attainment of virological suppression,a subset of patients exhibits inadequate recovery of CD4+T cells,and can not achieve immunological response,which results in higher incidences and mortality rates associated with opportunistic infections,non-AIDS-related complications,and malignancies.In response to the challenge of incomplete immune reconstitution,several novel therapeutic agents are currently undergoing clinical development.Nevertheless,there exists a lack of clinical trial guidances both domestically and internationally.This paper reviews the landscape of clinical trials evaluating therapies for immunological nonresponder,discussing key elements of clinical trial design,trail population,primary efficacy variable,treatment period,etc.,with the aim of providing insights to inform the clinical development of these drugs.

关 键 词：艾滋病病毒/艾滋病 免疫功能重建不全者/免疫无应答者 临床试验 

分 类 号：R512.91[医药卫生—内科学] R373.9[医药卫生—临床医学]