Nilotinib after imatinib first‐line: a real‐life longitudinal cohort of patients with chronic myeloid leukaemia in chronic phase. (19th December 2017)

• Record Type: Journal Article
• Authors: Cony‐Makhoul, Pascale; Gardembas, Martine; Coiteux, Valérie; Carpentier, Nathalie; Pommier, Cécile; Violet, Isabelle; Quittet, Philippe; Berger, Marc G.
• Is Part Of: British journal of haematology. Volume 180:Number 3(2018)
• Page Start: 356
• Page End: 364
• Publication Date: 2017-12-19
• Subjects: chronic myeloid leukaemia -- real‐life practice -- nilotinib -- second line treatment -- BCR‐ABL1; Hematology -- Periodicals; Blood -- Diseases -- Periodicals; 616.15
• DOI: 10.1111/bjh.15042
• Languages: English
• ISSNs: 0007-1048

Abstract

Summary: This prospective, observational study enrolled 150 adult patients with chronic myeloid leukaemia (CML) in chronic phase (CP) treated with nilotinib as second‐line after imatinib, in a real life setting in France. Two‐thirds of patients switched to nilotinib treatment due to lack of imatinib efficacy. Of 146 evaluable patients, 16 (11·0%) (95% confidence interval: 6·4–17·2%) achieved uMR 4, defined as undetectable molecular disease in cDNA with MR 4 sensitivity (≥10 000 ABL1 transcripts) at 18 months and confirmed at 24 months (primary endpoint). Among patients without major molecular response (MMR) or deep molecular response (DMR) at study entry, 66·3% achieved MMR and 44·2% DMR within a median of 5·7 and 6·24 months, respectively. Fifty‐three patients (36·3%) have prematurely terminated the study before 24 months of follow‐up, 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%). The most frequent extra‐haematological adverse events (AEs) reported as related to treatment with nilotinib were pruritus (16·4%), asthenia (13·7%) and dry skin (13·0%). Ischaemic cardiovascular AEs were reported in 18 patients (12·3%). This French nationwide large cohort adds valuable information to the body of evidence on the efficiency and safety of nilotinib in the treatment of patients with CP‐CML.