Nilotinib 300 mg twice daily: an academic single-arm study of newly diagnosed chronic phase chronic myeloid leukemia patients.

Fausto Castagnetti, Massimo Breccia, Gabriele Gugliotta, Bruno Martino, Mariella D'Adda, Fabio Stagno, Angelo Michele Carella, Paolo Avanzini, Mario Tiribelli, Elena Trabacchi, Giuseppe Visani, Marco Gobbi, Marzia Salvucci, Luciano Levato, Gianni Binotto, Silvana Franca Capalbo, Maria Teresa Bochicchio, Simona Soverini, Michele Cavo, Giovanni Martinelli, Giuliana Alimena, Fabrizio Pane, Giuseppe Saglio, Gianantonio Rosti, Michele Baccarani

Journal: Haematologica 2017;101(10):1200-1207

PMID: 27470600

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Abstract

The introduction and the extended clinical use of nilotinib in the first-line treatment of chronic myeloid leukemia have been based on company-sponsored trials. Independent confirmations are extremely important. We report an investigator-sponsored study of nilotinib 300 mg twice daily in 130 chronic myeloid leukemia patients in early chronic phase. A deep molecular response was achieved in 46% (MR) and 17% (MR) of patients at 2 years; 58% of the enrolled patients achieved a MR at least once, with a sustained MR in 52% of them. With a median observation of 29 months (range 24-37 months), 77% of patients were still on treatment with nilotinib. The reasons for permanent discontinuation were: 3% progression, 5% failure or suboptimal response, 8% adverse events, 1% treatment-free remission, and 5% other reasons. Thirteen thrombotic arterial events were reported in 12 patients. A prospective evaluation of metabolic effects showed an increase of fasting glucose without significant variations of glycated hemoglobin, an increase of total cholesterol (both low density lipoprotein and high density lipoprotein fractions) and a decrease of triglycerides. This study confirms a high and rapid efficacy of nilotinib 300 mg twice daily and provides detailed information on the type and incidence of non-hematologic and metabolic adverse events (clinicaltrials.gov identifier: 01535391).

Address: Institute of Hematology "L. and A. Seràgnoli", Department of Experimental, Diagnostic and Specialty Medicine, "S. Orsola-Malpighi" University Hospital, University of Bologna, Italy [email protected].; Department of Cellular Biotechnologies and Hematology, "Sapienza" University of Rome, Italy.; Institute of Hematology "L. and A. Seràgnoli", Department of Experimental, Diagnostic and Specialty Medicine, "S. Orsola-Malpighi" University Hospital, University of Bologna, Italy.; Hematology Unit, Azienda Ospedaliera "Bianchi-Melacrino-Morelli", Reggio Calabria, Italy.; Hematology Unit, Azienda Ospedaliera "Spedali Civili", Brescia, Italy.; Chair of Hematology, University of Catania, Italy.; U.O. Ematologia I, IRCCS AOU S. Martino-IST, Genova, Italy.; Hematology Unit, Arcispedale Santa Maria Nuova, IRCCS, Reggio Emilia, Italy.; Division of Hematology and BMT, Department of Experimental and Clinical Medical Sciences, Azienda Ospedaliero-Universitaria di Udine, Italy.; Hematology and Bone Marrow Transplantation Unit, Department of Hematology and Oncology, "G. da Saliceto" Hospital, Piacenza, Italy.; Hematology and Stem Cell Transplantation Unit, Azienda Ospedaliera Ospedali Riuniti Marche Nord (AORMN), Pesaro, Italy.; Clinical Hematology Unit, IRCCS AOU S. Martino-IST, Genova, Italy.; Hematology Unit, "Santa Maria delle Croci" Hospital, Ravenna, Italy.; Hematology Unit, "Pugliese-Ciaccio" Hospital, Catanzaro, Italy.; Hematology and Clinical Immunology Unit, University of Padova, Padova, Italy.; Hematology Unit, Azienda Ospedaliero-Universitaria Ospedali Riuniti, Foggia, Italy.; Chair of Hematology, Department of Biochemistry and Medical Biotechnologies, "Federico II" University, Napoli, Italy.; Chair of Hematology, Department of Clinical and Biological Sciences, "S. Luigi Gonzaga" University Hospital, University of Torino, Orbassano (TO), Italy.; Department of Hematology and Oncology "L. and A. Seràgnoli", University of Bologna, Italy.

DOI: 10.3324/haematol.2016.144949

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MeSH Terms: Adolescent, Adult, Aged, Aged, 80 and over, Blood Glucose, Cholesterol, Female, Humans, Leukemia, Myeloid, Chronic-Phase, Male, Middle Aged, Protein Kinase Inhibitors, Pyrimidines, Remission Induction, Thrombosis, Treatment Outcome, Triglycerides, Young Adult