Ponatinib efficacy and safety in Philadelphia chromosome-positive leukemia: final 5-year results of the phase 2 PACE trial.

ABSTRACT: Ponatinib has potent activity against native and mutant BCR-ABL1, including BCR-ABL1^T315I The pivotal phase 2 Ponatinib Ph⁺ ALL and CML Evaluation (PACE) trial evaluated efficacy and safety of ponatinib at a starting dose of 45 mg once daily in 449 patients with chronic myeloid leukemia (CML) or Philadelphia chromosome-positive acute lymphoblastic leukemia (ALL) resistant/intolerant to dasatinib or nilotinib, or with BCR-ABL1^T315I This analysis focuses on chronic-phase CML (CP-CML) patients (n = 270) with 56.8-month median follow-up. Among 267 evaluable patients, 60%, 40%, and 24% achieved major cytogenetic response (MCyR), major molecular response (MMR), and 4.5-log molecular response, respectively. The probability of maintaining MCyR for 5 years was 82% a

SUBMITTER: Cortes JE 

PROVIDER: S-EPMC6071555 | biostudies-literature | 2018 Jul

REPOSITORIES: biostudies-literature