OPTIC-2L: a Superiority Trial of Two Lower Doses of Ponatinib Versus Standard Dose Nilotinib in Second-Line Chronic Phase CML

Up to half of chronic-phase chronic myeloid leukemia (CP-CML) patients with first-line tyrosine kinase inhibitor (TKI) imatinib treatment become resistant or intolerant. A sizable portion of patients (38-49%) do not achieve major cytogenetic response (MCyR) with second-line treatment with dasatinib, nilotinib, or bosutinib. None of these agents have activity against all known kinase domain mutations and all are refractory to the T315I mutation. Ponatinib (45 mg daily), a potent pan-BCR-ABL TKI, demonstrated strong efficacy among a small cohort of second-line CP-CML patients, including those with the T315I mutation, in the pivotal PACE trial (NCT01207440).
Source: Clinical Lymphoma, Myeloma and Leukemia - Category: Hematology Authors: Source Type: research