@article{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_2016, place={Pavia, Italy}, title={Nilotinib 300 mg twice daily: an academic single-arm study of newly diagnosed chronic phase chronic myeloid leukemia patients}, volume={101}, url={https://haematologica.org/article/view/7846}, DOI={10.3324/haematol.2016.144949}, abstractNote={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<sup>4.0</sup>) and 17% (MR<sup>4.5</sup>) of patients at 2 years; 58% of the enrolled patients achieved a MR<sup>4.0</sup> at least once, with a sustained MR<sup>4.0</sup> 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 (<em><a href="http://clinicaltrials.gov">clinicaltrials.gov</a> identifier: 01535391</em&gt;).}, number={10}, journal={Haematologica}, author={Fausto Castagnetti and Massimo Breccia and Gabriele Gugliotta and Bruno Martino and Mariella D’Adda and Fabio Stagno and Angelo Michele Carella and Paolo Avanzini and Mario Tiribelli and Elena Trabacchi and Giuseppe Visani and Marco Gobbi and Marzia Salvucci and Luciano Levato and Gianni Binotto and Silvana Franca Capalbo and Maria Teresa Bochicchio and Simona Soverini and Michele Cavo and Giovanni Martinelli and Giuliana Alimena and Fabrizio Pane and Giuseppe Saglio and Gianantonio Rosti and Michele Baccarani}, year={2016}, month={Sep.}, pages={1200-1207} }