Franck E. NicoliniTamas MassziZhixiang ShenNeil J. GallagherSaengsuree JootarBayard L. PowellPedro Enrique Dorlhiac-LlacerMing ZhengTomasz SzczudloAnna TurkinaHopital Edouard HerriotSzent Laszlo HospitalRuijin HospitalNovartis International AGMahidol UniversityWake Forest UniversityUniversidade de Sao Paulo - USPNovartis Pharmaceuticals CorporationNational Research Center for Hematology2018-06-112018-06-112012-05-01Leukemia and Lymphoma. Vol.53, No.5 (2012), 907-91410292403104281942-s2.0-84859972409https://repository.li.mahidol.ac.th/handle/20.500.14594/13749Nilotinib has shown favorable safety in patients with Philadelphia chromosome-positive (Ph+) chronic myeloid leukemia (CML) in chronic (CML-CP) or accelerated phase (CML-AP) who failed prior imatinib, and superior efficacy over imatinib in newly diagnosed Ph+ patients with CML-CP. Reported here are the efficacy and safety data for patients in CML-AP (n = 181) or blast crisis (CML-BC) (n = 190; myeloid BC, 133; lymphoid BC, 50; unknown, seven) enrolled in an expanded access phase IIIb study. Non-hematologic adverse events were mostly mild to moderate. Drug-related myelosuppression was generally manageable with dose reductions or interruptions and infrequently led to discontinuation of nilotinib. Drug-related grade 3/4 elevations in serum bilirubin and lipase were infrequent. While an analysis of efficacy was not the primary objective of this study, significant hematologic and cytogenetic responses were observed. These results support the safety and efficacy of nilotinib in patients with advanced CML in AP and BC. © 2012 Informa UK, Ltd.Mahidol UniversityBiochemistry, Genetics and Molecular BiologyMedicineArticleSCOPUS10.3109/10428194.2011.627480