Response and Adverse Effects of Nilotinib in Imatinib-resistant Chronic Myeloid Leukemia Patients: Data From a Developing Country

Document Type



Pathology and Laboratory Medicine


PURPOSE: The purpose of this study was to determine the frequency of major cytogenetic response (MCyR) and adverse events with nilotinib in adults with imatinib-resistant Philadelphia chromosome-positive chronic myeloid leukemia (CML).Methods: This is a descriptive cross-sectional study conducted at The Aga Khan University Hospital in Karachi from October 17, 2010, to October 17, 2011. A cytogenetic assessment using fluorescent in situ hybridization was performed on peripheral blood before initiation of treatment and 6 months after treatment with nilotinib. The frequency of adverse effects was assessed at 6 months, and the patient overall survival was calculated after 3 years.FINDINGS: This study enrolled 82 imatinib-resistant patients. The mean (SD) patient age was 38.9 (12.2) years. There were 62 patients (75.6%) in chronic phase, 15 patients (18.3%) in accelerated phase, and 5 patients (6%) in blast crisis phase. At 6 months cytogenetic studies were available for 40 (52%) of 77 patients, and MCyR was observed in 31 (77.5%) of 77 patients. The patients in chronic phase had the highest frequency of MCyR (n = 27 [87%] of 31). We observed 6 deaths (7.3%), and the overall survival at 3 years was 92.7%. There was isolated thrombocytopenia in 12 patients (15.6%). The most frequent nonhematologic adverse events were myalgia and headache.IMPLICATIONS: The nilotinib response rates were higher in chronic phase patients, and the most common adverse events were thrombocytopenia, myalgia, and headache.

Publication (Name of Journal)

Clinical Therapeutics