A phase 2 trial of ponatinib in Philadelphia chromosome-positive leukemias.

ABSTRACT:

Background

Ponatinib is a potent oral tyrosine kinase inhibitor of unmutated and mutated BCR-ABL, including BCR-ABL with the tyrosine kinase inhibitor-refractory threonine-to-isoleucine mutation at position 315 (T315I). We conducted a phase 2 trial of ponatinib in patients with chronic myeloid leukemia (CML) or Philadelphia chromosome-positive acute lymphoblastic leukemia (Ph-positive ALL).

Methods

We enrolled 449 heavily pretreated patients who had CML or Ph-positive ALL with resistance to or unacceptable side effects from dasatinib or nilotinib or who had the BCR-ABL T315I mutation. Ponatinib was administered at an initial dose of 45 mg once daily. The median follow-up was 15 months.

Results

Among 267 patients with chronic-phase CML, 56% had a major cytogenetic resp

SUBMITTER: Cortes JE 

PROVIDER: S-EPMC3886799 | biostudies-literature | 2013 Nov

REPOSITORIES: biostudies-literature