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.
19 affiliations
- 1Hematology Clinic and Cellular Therapy, Bordeaux University Hospital, Pessac, France.
- 2BRIC UMR 1312, National Institute of Health and Medical Research, Bordeaux University, Bordeaux, France.
- 3Hematology Clinic, Rennes University Hospital, Rennes, France.
- 4Hematology Clinic, Paoli-Calmettes Cancer Institute, Marseilles, France.
- 5Hematology Biology, Bordeaux University Hospital, Pessac, France.
- 6Hematology Clinic, Angers University Hospital, FHU Goal, Université d'Angers, Université de Nantes, CHU Angers, National Institute of Health and Medical Research, CNRS, CRCI2NA, SFR ICAT, Angers, France.
- 7Hematology Clinic, Grenoble Alpes University Hospital, La Tronche, France.
- 8Hematology Clinic, Mulhouse Hospital, Mulhouse, France.
- 9Institut Universitaire du Cancer de Toulouse Oncopole, Toulouse University Hospital, Toulouse University, Toulouse, France.
- 10Hematology Clinic, Bayonne Hospital, Bayonne, France.
- 11Hematology Clinic, Nantes University Hospital, Nantes, France.
- 12Hematology Clinic, Clermont Ferrand University Hospital, Clermont Ferrand, France.
- 13Hematology Clinic, Besançon University Hospital, Besançon, France.
- 14Hematology Clinic, Nancy University Hospital, Nancy, France.
- 15Hematology Clinic, Strasbourg University Hospital, Strasbourg, France.
- 16Hematology Clinic and Cellular Therapy, Tours University Hospital, Tours, France.
- 17Hematology Clinic, Nîmes University Hospital, Nîmes, France.
- 18Hematology Biology, Nantes University, Nantes, France.
- 19Clinical Research Center, Angers University Hospital, Angers, France.
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.
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BibTeX
@article{pigneux2026eltrombopag,
title = {{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.}},
author = {Arnaud Pigneux and Pierre-Yves Dumas and Marc Bernard and Norbert Vey and Audrey Bidet and Ariane Mineur and Aline Schmidt and Martin Carré and Mario Ojeda and Sarah Bertoli and Anne Banos and Pierre Peterlin and Romain Guièze and Yohan Desbrosses and Gabrielle Roth Guepin and Célestine Simand and Thibaut Leguay and Emmanuel Gyan and Eric Jourdan and Jean-Philippe Vial and Tony Marchand and Yosr Hicheri and Marie C Béné and Jean Francois Hamel and Christian Récher},
journal = {Cancer},
year = {2026},
month = oct,
doi = {10.1002/cncr.70669},
url = {https://europepmc.org/article/MED/42963911},
note = {CC BY},
}
RIS
TY - JOUR
TI - 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.
AU - Arnaud Pigneux
AU - Pierre-Yves Dumas
AU - Marc Bernard
AU - Norbert Vey
AU - Audrey Bidet
AU - Ariane Mineur
AU - Aline Schmidt
AU - Martin Carré
AU - Mario Ojeda
AU - Sarah Bertoli
AU - Anne Banos
AU - Pierre Peterlin
AU - Romain Guièze
AU - Yohan Desbrosses
AU - Gabrielle Roth Guepin
AU - Célestine Simand
AU - Thibaut Leguay
AU - Emmanuel Gyan
AU - Eric Jourdan
AU - Jean-Philippe Vial
AU - Tony Marchand
AU - Yosr Hicheri
AU - Marie C Béné
AU - Jean Francois Hamel
AU - Christian Récher
PY - 2026
DA - 2026/10/30
JO - Cancer
DO - 10.1002/cncr.70669
UR - https://europepmc.org/article/MED/42963911
AB - 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.
SN - 0008-543X
SN - 1097-0142
ER -