Cony-Makhoul P, et al. - This prospective, observational study was performed to investigate the safety and efficiency of nilotinib after imatinib first-line therapy in a cohort of patients with chronic myeloid leukaemia (CML) in chronic phase (CP). As per findings, 66·3% patients achieved major molecular response (MMR) and 44·2% deep molecular response (DMR) within a median of 5·7 and 6·24 months, respectively. The outcomes added valuable information to the body of evidence on the efficiency and safety of nilotinib in the treatment of patients with CP-CML.

Methods

• The researchers enrolled 150 adult patients with CML in CP treated with nilotinib as second-line after imatinib, in a real life setting in France.

Results

• Due to lack of imatinib efficacy, 2/3^rd of patients switched to nilotinib treatment.
• Sixteen patients (11·0%) (95% confidence interval: 6·4-17·2%) achieved uMR among 146 evaluable patients, defined as undetectable molecular disease in cDNA with MR sensitivity (≥10,000 ABL1 transcripts) at 18 months and confirmed at 24 months (primary endpoint).
• In this study, 66·3% patients achieved MMR and 44·2% DMR within a median of 5·7 and 6·24 months, respectively, among patients without major molecular response (MMR) or deep molecular response (DMR) at study entry.
• Before 24 months of follow-up, 53 patients (36·3%) prematurely terminated the study, primarily due to nilotinib treatment discontinuation (n = 43; 29·5%), mainly motivated by treatment intolerance (n = 27; 18·5%) and inefficacy (n = 10; 6·8%).
• Pruritus (16·4%), asthenia (13·7%) and dry skin (13·0%) were the most frequently reported extra-haematological adverse events (AEs) related to treatment with nilotinib.
• In 18 patients (12·3%), ischaemic cardiovascular AEs were reported.