Nilotinib efficacy, safety, adherence and impact on quality of life in newly diagnosed patients with chronic myeloid leukaemia in chronic phase: a prospective observational study in daily clinical practice

This observational, prospective study assessed, in a daily clinical practice, the molecular response, safety, quality of life (QoL) and treatment adherence in 183 patients with chronic myeloid leukaemia in chronic phase (CML-CP), receiving nilotinib as first-line treatment. Premature study termination before 24 months of follow-up occurred in 61 patients (33.3%), and was essentially due to nilotinib treatment discontinuation (n = 53; 29%), motivated by treatment intolerance (n = 29; 15.8%) and inefficacy (n = 19; 10.4%). After 24 months of treatment, 112/122 patients (91.8%) had a molecular assessment, 95.5% of whom achieved a major molecular response (MMR), 32.1% achieved uMR(4) , defined as an undetectable molecular disease with 4-log molecular response sensitivity (>/=10 000 ABL1 transcripts). The Morisky Green Levine Medication Adherence Scale was completed by 94/122 patients (77.0%), and 89.4% of these patients obtained a satisfactory level of treatment adherence, defined as a score >/=3. Patients' QoL was good at baseline and stable during the follow-up period. The two most common nilotinib-related adverse events (AEs) were pruritus (14.8%) and asthenia (13.7%). Seven patients (3.8%) experienced at least one cardiovascular ischaemic AE. This French nationwide cohort study provides relevant information in daily clinical practice indicating that nilotinib is a valuable first-line treatment option for CML-CP patients.