Article
Five-year follow-up of OPTIC: long-term efficacy, safety, and mutation analyses of ponatinib in chronic-phase chronic myeloid leukemia from a randomized phase 2 trial.
Leukemia - 1 Sept 2026
Kantarjian Hagop, Deininger Michael, Apperley Jane F, Arthur Christopher Kevin, Chuah Charles, Hochhaus Andreas, de Lavallade Hugues, Lipton Jeffrey H, Lomaia Elza, McCloskey James, Maness Lori, Mauro Michael, Moiraghi Beatriz, Pavlovsky Carolina, Rosti Gianantonio, Rousselot Philippe, Chao Bo, Vorog Alexander, Reddick L Evan, Patel Niti, Hennessy Meliessa, Yeh Tammie, Cortes Jorge
Abstract excerpt
The primary analysis of the phase 2 OPTIC trial (NCT02467270) demonstrated optimal benefit:risk with response-based ponatinib dosing (45 mg once daily (QD) reduced to 15 mg QD) upon achieving ≤1% BCR::ABL1IS in patients with tyrosine kinase inhibitor-resistant or T315I-positive chronic-phase chronic myeloid leukemia (CP-CML). Here, we report 5-year long-term outcomes. Overall, 283 patients were randomized to...
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