Updated review of nilotinib as frontline treament for newly diagnosed Philadelphia chromosome-positive chronic myeloid leukemia

Author(s): Massimo Breccia, Matteo Molica & Giulina Alimena

Nilotinib, a second-generation tyrosine kinase, is approved for the treatment of newly diagnosed chronic phase chronic myeloid leukemia patients. The drug showed impressive rates of cytogenetic and molecular responses with only few cases of disease progression in Phase II trials. Five years follow-up of Phase III randomized ENESTnd study demonstrated deep molecular responses, lower rate of transformation in the core treatment compared with imatinib (0.7% for nilotinib 300 mg twice daily [BID], 1.1% for nilotinib 400 mg BID and 4.2% with imatinib). ENEST1st study confirmed the activity of nilotinib in first line at the dose of 300 mg BID. Aim of this review is to provide reported evidences on the efficacy and safety of nilotinib when used for the management of early chronic phase chronic myeloid leukemia patients and to discuss about future possible discontinuation.