Hui Rex Wan-Hin, Mak Lung-Yi, Fung James, Seto Wai-Kay, Yuen Man-Fung
Department of Medicine, The University of Hong Kong, Hong Kong.
State Key Laboratory of Liver Research, The University of Hong Kong, Hong Kong.
Clin Mol Hepatol. 2025 Feb;31(Suppl):S165-181. doi: 10.3350/cmh.2024.0855. Epub 2024 Nov 14.
Functional cure, defined as sustained hepatitis B surface antigen (HBsAg) seroclearance with unquantifiable hepatitis B virus (HBV) DNA at 24 weeks off treatment, is a favorable treatment endpoint in chronic hepatitis B (CHB). Nonetheless, functional cure is rarely attained with the current treatment modalities of nucleos(t)ide analogues (NUCs) and pegylated interferon alpha. Multiple novel virus-targeting agents and immunomodulators are under development for HBV with functional cure as the treatment goal. Among virus-targeting agents, antisense oligonucleotides and small-interfering RNAs are the most advanced in the developmental pipeline, and can induce potent and sustainable HBsAg suppression. The other virus-targeting agents have varying effects on HBsAg and HBV DNA, depending on the drug mechanism. In contrast, immunomodulators have modest effects on HBsAg and have limited roles in monotherapy. Multiple combination regimens incorporating RNA interference agents with immunomodulators have been studied through many ongoing clinical trials. These combination strategies demonstrate synergistic effects in inducing functional cure, and will likely be the future direction of development. Despite the promising results, research is warranted to optimize treatment protocols and to establish criteria for NUC withdrawal after novel therapies. Functional cure is now an attainable target in CHB, and the emerging novel therapeutics will revolutionize CHB management.
功能性治愈定义为在停止治疗24周时乙肝表面抗原(HBsAg)血清学清除且乙肝病毒(HBV)DNA不可测,是慢性乙型肝炎(CHB)治疗的一个理想终点。尽管如此,目前使用核苷(酸)类似物(NUCs)和聚乙二醇化干扰素α的治疗方式很少能实现功能性治愈。多种新型病毒靶向药物和免疫调节剂正在研发中,目标是实现对HBV的功能性治愈。在病毒靶向药物中,反义寡核苷酸和小干扰RNA在研发进程中最为先进,能够有效且持续地抑制HBsAg。其他病毒靶向药物对HBsAg和HBV DNA的影响各不相同,这取决于药物作用机制。相比之下,免疫调节剂对HBsAg的作用较小,在单一疗法中的作用有限。通过许多正在进行的临床试验研究了多种将RNA干扰药物与免疫调节剂结合的联合治疗方案。这些联合策略在诱导功能性治愈方面显示出协同效应,很可能是未来的发展方向。尽管取得了令人鼓舞的结果,但仍有必要开展研究以优化治疗方案,并确定新型疗法后停用NUC的标准。功能性治愈现在是CHB中一个可实现的目标,新兴的新型疗法将彻底改变CHB的治疗方式。