Cancer. 2026 Nov 1;132(21):e70669. doi: 10.1002/cncr.70669.
ABSTRACT
BACKGROUND: Elderly patients with acute myeloid leukemia (AML) treated with intensive chemotherapy are at risk of developing thrombopenia, which requires platelet transfusions. It would be interesting to provide them with thrombopoietin receptor agonists such as eltrombopag.
METHODS: The EPAG-2015 multicenter phase 2 trial (18 French hospitals from October 2018 to June 2021) blindly randomized elderly patients (aged ≥60 years) with placebo or eltrombopag administered on Day 11 after induction chemotherapy and continued until AML response evaluation (Day 45) or a platelet count of >100 × 109/L. A cohort of 110 patients with a median age of 70 years was included and randomized (55 per arm) to receive placebo or eltrombopag.
RESULTS: After induction, 90 patients (81.8%) achieved complete response (CR) or CR with incomplete reconstitution (CRi) (45 in each group), including 74 patients with CR (38 with eltrombopag and 36 with placebo). With a median follow-up for alive patients of 20.9 months the primary objective was not reached, given that there was no difference in overall survival (hazard ratio, 1.59; 95% CI, 0.65-3.89; p = .31). Eltrombopag, however, did not lead to increased grade 3/4 adverse events, and was associated with a lower median number of platelet transfusions (eight; interquartile range [IQR], 6-14; vs. 10; IQR, 7-14; i.e., a 20% benefit).
CONCLUSIONS: This study failed to demonstrate a survival advantage after eltrombopag addition, at variance with previous reports, but shows that initiating eltrombopag after chemotherapy washout is feasible and safe, and resulted in a decreased need for platelet transfusions.
PMID:42963911 | DOI:10.1002/cncr.70669
