Oncology · Hematology · 29 Oct
Phase II ACT-GI Trial Finds Avatrombopag Improved Platelet Recovery in GI Cancer Patients
In a randomized, double-blind US phase II trial, 47 patients with gastrointestinal cancers and persistent chemotherapy-induced thrombocytopenia received avatrombopag or placebo. The primary endpoint was met by 70% of avatrombopag recipients and 17% of placebo recipients; enrollment stopped at a planned interim analysis after prespecified efficacy criteria were met.
- Primary endpoint: 70% with avatrombopag versus 17% with placebo
- Trial stopped at planned interim analysis for efficacy
- Phase II results require confirmation in larger studies
Persistent chemotherapy-induced thrombocytopenia can lead to chemotherapy delays or dose reductions, and the source notes that no widely available approved treatment exists. The multicenter, investigator-initiated US trial randomized patients with gastrointestinal cancers and platelet counts of 85 × 10⁹/L or lower at the start of a chemotherapy cycle to avatrombopag or placebo. The primary endpoint required platelet recovery to at least 100 × 10⁹/L, no chemotherapy dose modification or delay, and prevention of recurrent thrombocytopenia during the on-study cycle.
The trial enrolled 47 patients: 23 received avatrombopag and 24 placebo. The primary endpoint was reached by 16 patients (70%; 95% CI, 47–87) in the avatrombopag group and 4 (17%; 95% CI, 5–37) in the placebo group (P < .001). Median platelet counts at the end of the treatment period were 157 × 10⁹/L and 72 × 10⁹/L, respectively. Enrollment ended at the planned interim analysis after the data and safety monitoring board found that prespecified efficacy stopping criteria had been met.
Adverse events occurred in 78% versus 46% of patients, and serious adverse events in 17% versus 0%; none of the serious events was considered study-drug-related. These phase II findings are promising but come from a small trial stopped early. They do not establish that avatrombopag should be used routinely for chemotherapy-induced thrombocytopenia.
In practice
For patients with gastrointestinal cancers and persistent chemotherapy-induced thrombocytopenia, avatrombopag improved platelet recovery and reduced the composite of persistent correction without chemotherapy dose modification or delay in this randomized phase II trial. The result is promising, but it is based on only 47 patients and the trial stopped early at a planned interim analysis for efficacy. The findings do not establish routine use; larger confirmatory studies would be needed to define the benefit and safety more reliably.
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Journal of Clinical Oncology: Avatrombopag Versus Placebo for Persistent Chemotherapy-Induced Thrombocytopenia in GI Cancers: The Phase II ACT-GI Trial ↗This is an automated AI-condensed summary that has not yet been reviewed by an editor. Always consult the full item at the original source.