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 E. Trabacchi, Giuseppe Visani, Marco Gobbi, Marzia Salvucci, Luciano Levato, Gianni Binotto, Silvana Franca Capalbo, Maria Teresa Bochicchio, Simona Soverini, Michele Cavo, Giovanni MartinelliGiuliana Alimena, Fabrizio Pane, Giuseppe Saglio, Gianantonio Rosti, Michele Baccarani, The GIMEMA CML Working Party

Research output: Contribution to journalArticle

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% (MR4.0) and 17% (MR4.5) of patients at 2 years; 58% of the enrolled patients achieved a MR4.0 at least once, with a sustained MR4.0 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).

Original languageEnglish
Pages (from-to)1200-1207
Number of pages8
JournalHaematologica
Volume101
Issue number10
DOIs
Publication statusPublished - Sep 30 2016

ASJC Scopus subject areas

  • Hematology

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    Castagnetti, F., Breccia, M., Gugliotta, G., Martino, B., D’Adda, M., Stagno, F., Carella, A. M., Avanzini, P., Tiribelli, M., Trabacchi, E. E., Visani, G., Gobbi, M., Salvucci, M., Levato, L., Binotto, G., Capalbo, S. F., Bochicchio, M. T., Soverini, S., Cavo, M., ... The GIMEMA CML Working Party (2016). Nilotinib 300 mg twice daily: An academic single-arm study of newly diagnosed chronic phase chronic myeloid leukemia patients. Haematologica, 101(10), 1200-1207. https://doi.org/10.3324/haematol.2016.144949