海曲泊帕在中国：适应症拓展、比较定位与风险管理——一篇批判性叙述综述。
完整摘要 · 中文译文
目的：海曲泊帕是一种口服非肽类血小板生成素受体激动剂，仅在中国获批。我们批判性评价了其已获批及研究性适应症的证据，与其他血小板生成素受体激动剂进行了比较，并评估了临床相关安全性和风险管理。 方法：检索PubMed/MEDLINE、Cochrane图书馆、ClinicalTrials.gov、CNKI和万方数据库，检索时间自建库至2026年10月18日，并进行引文追溯及监管/指南审查。证据按研究设计和发表状态进行分级。 结果：一项中国III期成人免疫性血小板减少症试验报告，两种起始剂量第8周血小板反应率分别为58.9%和64.3%，而安慰剂组为5.9%。在难治性重型再生障碍性贫血中，一项单臂II期研究报告第18周至少一个系别血液学反应率为41.8%（95%置信区间28.7-55.9）。随机试验还支持了2026年中国批准用于年龄至少6岁的慢性免疫性血小板减少症患者及成人实体瘤治疗所致血小板减少症；然而，关键的III期实体瘤报告仍为会议摘要，国际验证尚待进行。尚无随机头对头试验确立其优于艾曲泊帕、阿伐曲泊帕或罗米司亭。安全性数据识别出成人免疫性血小板减少症中的肝脏实验室异常、罕见血栓形成和骨髓纤维化，以及重型再生障碍性贫血中的克隆演化。 讨论：终点异质性、申办方参与、仅摘要证据及有限的多民族数据限制了cr 单臂试验推断。食物显著降低暴露量；环孢素相互作用数据令人安心但不精确。 结论：使用应遵循当地说明书和疾病指南，以临床有意义的结果为目标而非血小板正常化，并包括针对适应证的肝脏、血栓、骨髓和停药监测。
完整摘要 · 英文原文
OBJECTIVES: Hetrombopag is an oral nonpeptide thrombopoietin receptor agonist approved only in China. We critically appraised evidence across approved and investigational indications, compared it with other thrombopoietin receptor agonists, and assessed clinically relevant safety and risk management. METHODS: PubMed/MEDLINE, the Cochrane Library, ClinicalTrials.gov, CNKI, and Wanfang were searched from inception through 18 October 2026, with citation tracing and regulatory/guideline review. Evidence was ranked by design and publication status. RESULTS: A Chinese phase III adult immune thrombocytopenia trial reported week-8 platelet responses of 58.9% and 64.3% with two starting doses versus 5.9% with placebo. In refractory severe aplastic anemia, a single-arm phase II study reported week-18 hematologic response in at least one lineage of 41.8% (95% confidence interval 28.7-55.9). Randomized trials also underpin 2026 Chinese approvals for chronic immune thrombocytopenia in patients aged at least 6 years and adult solid-tumor treatment-induced thrombocytopenia; however, the pivotal phase III solid-tumor report remained a conference abstract and international validation was pending. No randomized head-to-head trial establishes superiority over eltrombopag, avatrombopag, or romiplostim. Safety data identify hepatic laboratory abnormalities, rare thrombosis and marrow fibrosis in adult immune thrombocytopenia, and clonal evolution in severe aplastic anemia. DISCUSSION: Endpoint heterogeneity, sponsor involvement, abstract-only evidence, and limited multiethnic data constrain cross-trial inference. Food markedly reduces exposure; cyclosporine interaction data are reassuring but imprecise. CONCLUSION: Use should follow local labels and disease guidelines, target clinically meaningful outcomes rather than platelet normalization, and include indication-specific hepatic, thrombotic, marrow, and discontinuation surveillance.