Eltrombopag during induction chemotherapy in older patients with newly diagnosed acute myeloid leukemia is safe and reduces platelet transfusion requirements: The EPAG-2015 French Innovative Leukemia Organization phase 2 randomized trial
In brief
Older patients on eltrombopag needed 20% fewer platelet transfusions during leukemia treatment
In a randomized phase 2 trial of 110 older adults receiving intensive chemotherapy for acute myeloid leukemia, eltrombopag lowered the median number of platelet transfusions from 10 to eight, with no increase in severe adverse events. It did not improve overall survival, so the practical value is fewer transfusions; whether that benefit holds in a larger trial remains unknown.
- Journal
- Cancer (Q1)
- Published
- 1 November 2026
- Study design
- Phase 2 randomized trial (exploratory)
- Evidence level
- Level 2, Moderate (CEBM 2b)
- Authors
- Arnaud Pigneux, Pierre-Yves Dumas, Marc Bernard, Norbert Vey, Audrey Bidet, Ariane Mineur, et al.
- PMID
- 42963911
- DOI
- 10.1002/cncr.70669
Why clinicians should know about it
- Picked for Hematology (paper of the day, 2 November 2026): Randomized AML trial addresses thrombocytopenia and platelet transfusion needs
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.
Abstract as published, via PubMed.
For healthcare professionals. The summary is generated by AI from the published abstract, and the evidence level is assigned automatically from the study design on the Oxford CEBM hierarchy. Neither is medical advice. Read the full paper before changing practice.