ASCO 2026
Oral drug may help patients stay on chemotherapy despite low platelets
Clinical Takeaway: Avatrombopag may offer a new oral treatment approach for chemotherapy-induced thrombocytopenia in patients with gastrointestinal cancers. By improving platelet recovery and reducing chemotherapy delays or dose reductions, the drug could help preserve planned treatment intensity in patients at risk for compromised cancer outcomes.
Chemotherapy-induced thrombocytopenia (CIT) can force changes to cancer treatment plans and may compromise oncologic outcomes. Although avatrombopag, an oral thrombopoietin receptor agonist, is already FDA approved for thrombocytopenia in patients with chronic liver disease, its effectiveness in CIT has remained uncertain. To date, no thrombopoietin receptor agonist has been approved for CIT, although several have shown promise in clinical trials.
Phase 2 data presented at the 2026 American Society of Clinical Oncology annual meeting suggest avatrombopag may help address this unmet need in patients with gastrointestinal malignancies and persistent CIT.
The randomized placebo-controlled trial enrolled patients with gastrointestinal cancers whose platelet counts were unlikely to recover adequately between chemotherapy cycles. Investigators evaluated whether avatrombopag could both restore platelet counts within two weeks and maintain platelet recovery through the subsequent chemotherapy cycle.
Among patients receiving avatrombopag, 65% achieved both treatment goals compared with 17% receiving placebo. The study was stopped early at interim analysis after enrolling 23 patients because of the magnitude of benefit observed.
The results suggest avatrombopag may help more patients maintain planned chemotherapy schedules and avoid treatment modifications associated with poorer cancer outcomes. An oral thrombopoietin receptor agonist offers a practical advantage over injectable agents by reducing clinic visits and logistical burden for patients already undergoing intensive cancer care.
Patients with many other tumor types also experience CIT and treatment delays, suggesting the approach may have broader applicability beyond gastrointestinal malignancies.
“There is pretty clear evidence that dose reduction or delay has an impact on cancer outcomes, and CIT is a common reason that doses would be delayed or reduced,” said Gerald A. Soff, MD, chief of classical hematology at Sylvester Comprehensive Cancer Center. “Our goal here is to avoid compromising the cancer treatment.”
Source: Soff GA. ASCO 2026 Annual Meeting Abstract 3580. Avatrombopag for chemotherapy-induced thrombocytopenia in gastrointestinal malignancies (ACT-GI): A multicenter, U.S., randomized, double-blind, placebo-controlled clinical trial