Single-arm trial finds preliminary hetrombopag activity in gynecologic cancer

A single-arm exploratory trial found that hetrombopag was associated with platelet recovery in 37 Chinese patients with gynecologic malignancies who had thrombocytopenia while receiving concurrent chemoradiotherapy. Thirty patients, or 81.1%, reached a platelet count of at least 100 × 10⁹/L within 14 days, and 19 patients, or 51.4%, reached that threshold within seven days. The median time to reach the threshold was 6.0 days, with a 95% confidence interval of 6.0 to 15.0 days.

Only one patient required a platelet transfusion. Thirty patients met the composite endpoint. Mean platelet counts also showed an upward trend from baseline during the early treatment phase.

Treatment-emergent adverse events occurred in 34 of the 37 patients, or 91.9%, although the researchers reported no serious adverse events. The safety findings came from the same small cohort as the efficacy results. The results provide preliminary evidence of clinical activity and a manageable safety profile for hetrombopag in this population, but they do not establish comparative efficacy.

The sample was small, restricted to Chinese patients, and used different starting doses according to thrombocytopenia grade, which limits the strength and generalizability of the conclusions. The investigators said the findings warrant validation in randomized controlled trials. The study is registered as NCT05160857 and was published as open-access research in BMC Cancer.

Subject of Research: Preliminary evaluation of hetrombopag for thrombocytopenia during concurrent chemoradiotherapy in gynecologic malignancies

Article Title: The efficacy and safety of hetrombopag for chemoradiotherapy-induced thrombocytopenia in patients with gynecologic malignancies: a single-arm exploratory trial

Article References: Wu, J., Song, X., Wang, N., Yuan, Q., Pei, Z., & Wu, W. (2026). The efficacy and safety of hetrombopag for chemoradiotherapy-induced thrombocytopenia in patients with gynecologic malignancies: a single-arm exploratory trial. BMC Cancer. https://doi.org/10.1186/s12885-026-16764-y

Image Credits: AI Generated

DOI: 10.1186/s12885-026-16764-y

Keywords: hetrombopag, thrombocytopenia, gynecologic malignancies, chemoradiotherapy, platelet count, single-arm trial, clinical activity, treatment safety, efficacy, safety, chemoradiotherapy-induced, patients