Hijiya N, Maschan A, Rizzari C, et al. - Researchers investigated the efficacy of nilotinib as an alternative treatment option to imatinib in pediatric patients with Philadelphia chromosome-positive (Ph+) chronic myeloid leukemia (CML) in chronic phase (CP), particularly considering patients who developed resistance/intolerance (R/I) to imatinib. In this phase 2 study, pediatric patients with either Ph+ CML-CP R/I to imatinib/dasatinib or newly diagnosed Ph+ CML-CP (R/I, n = 33; newly diagnosed, n = 25) were enrolled. From analyses with up to 24 cycles' minimum follow-up (1 cycle = 28 days), data were obtained. In pediatric patients, the nilotinib demonstrated safety profile generally comparable with the known safety profile in adults, although there were no cardiovascular events in this study and there were more frequent hepatic laboratory abnormalities; they observed no new safety signals. In summary, nilotinib was established as an efficacious option with a manageable safety profile in pediatric patients with Ph+ CML-CP.