Hetrombopag in China: expanding indications, comparative positioning, and risk management - a critical narrative review.

Authors

Fan Chao, Rongwei Li

Published in

Hematology (Amsterdam, Netherlands). Volume 31. Issue 1. Pages 2755117. Dec 31, 2026. Epub Nov 02, 2026.

Abstract

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.
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.
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.
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.
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.

PMID:
42970480
Bibliographic data and abstract were imported from PubMed on 03 Nov 2026.

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