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

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 × 10 9 /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.

Authors

Institutions

Publication Details

Journal
Cancer
Published
2026-10-30
DOI
https://doi.org/10.1002/cncr.70669
Primary Topic
Acute Myeloid Leukemia Research
Type
article
Field-Weighted Citation Impact
0.00
Controls
|||
ALL TIME
JAN
FEB
MAR
APR
MAY
JUN
JUL
AUG
SEP
OCT
article

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

Arnaud Pigneux, Audrey Bidet, Christian Récher, Pierre‐Yves Dumas et al.
Cancer
Acute Myeloid Leukemia Research
article

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

Arnaud Pigneux, Audrey Bidet, Christian Récher, Pierre‐Yves Dumas, Pierre Péterlin, Romain Guièze, Sarah Bertoli, Thibaut Leguay, Yosr Hicheri, Norbert Vey, Célestine Simand, Marie C. Béné, Emmanuel Gyan, Ariane Mineur, Martin Carré, Yohan Desbrosses, Tony Marchand, Jean Vial, Éric Jourdan, Marc Bernard, Mario Ojeda, Anne Banos, Jean François Hamel, Gabrielle Roth Guepin, Aline SCHMIDT
article en

Abstract

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 × 10 9 /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.

CancerVol. 132(21)
Centre National de la Recherche Scientifique (FR), Centre Hospitalier Universitaire de Grenoble (FR), Centre Hospitalier Universitaire de Bordeaux (FR), Centre Hospitalier Universitaire de Nantes (FR), Centre Hospitalier Universitaire de Besançon (FR), Centre de Recherche en Cancérologie et Immunologie Intégrée Nantes Angers (FR), Centre Hospitalier Universitaire de Tours (FR), Institut universitaire du cancer de Toulouse Oncopole (FR), Centre Hospitalier Régional et Universitaire de Nancy (FR), Centre Hospitalier Universitaire de Nîmes (FR), Centre Hospitalier Universitaire d'Angers (FR), Centre Hospitalier de Mulhouse (FR), Centre Hospitalier de la Côte Basque (FR), Centre Hospitalier Universitaire de Clermont-Ferrand (FR), Hôpitaux Universitaires de Strasbourg (FR), Institut Paoli-Calmettes (FR), Centre Hospitalier Universitaire de Rennes (FR), BoRdeaux Institute of onCology (FR), Université de Nantes (FR), Université d'Angers (FR), Université Grenoble Alpes (FR), Nantes Université (FR)
Openalex Percentile: Top 11%
Acute Myeloid Leukemia Research
DOIOpen Access PDFOpenAlex
AI Navigator

Ask Laika to Summarize, Analyze, and Connect papers live on the map.

Summarize Papers & Methodologies

Extract key findings, datasets, and comparative methods across publications.

Benchmark Rankings & Visual Analytics

Rank top research institutions, authors, funders, topics, and journals by Field-Weighted Citation Impact (FWCI) and paper volume with instant charts.

Connect Distant Disciplines

Bridge topological clusters on the map to find hidden collaborative intersections.

Laika access coming soon