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Blood advancesJul 2025 DOI:
10.1182/bloodadvances.2025016399

Long-term results from the AGILE study of azacitidine plus ivosidenib vs placebo in newly diagnosed IDH1-mutated AML

Montesinos, Pau; Marchione, Dylan M; Récher, Christian; Heuser, Michael; Vives, Susana; Zarzycka, Ewa; Wang, Jianxiang; Riva, Marta; Calado, Rodrigo T; Schuh, Andre C; Yeh, Su-Peng; Tron, Adriana E; Hui, Jianan; Gianolio, Diego A; Choe, Sung; Patel, Prapti A; De Botton, Stéphane; DiNardo, Courtney D; Döhner, Hartmut
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Abstract
In the phase 3 AGILE study, after a 12.4-month median follow-up, ivosidenib, a mutant isocitrate dehydrogenase 1 (IDH1) inhibitor, combined with azacitidine significantly improved event-free survival, overall survival (OS), and complete remission rates compared with placebo-azacitidine in patients with newly diagnosed IDH1-mutated acute myeloid leukemia who were unfit for intensive chemotherapy. This post hoc analysis reports long-term follow-up results from AGILE after a median follow-up of 28.6 months. Overall, 148 patients were randomized to receive ivosidenib-azacitidine (N=73) or placebo-azacitidine (N=75). Median OS was significantly longer with ivosidenib (29.3 months; 95% CI 13.2, not reached) than with placebo (7.9 months; 95% CI 4.1, 11.3; hazard ratio 0.42 [0.27, 0.65]; p
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