Open AccessTreatment-free remission in chronic myeloid leukemia patients treated front-line with nilotinib: 10-year followup of the GIMEMA CML 0307 study
Author(s) -
Gabriele Gugliotta,
Fausto Castagnetti,
Massimo Breccia,
Luciano Levato,
Tamara Intermesoli,
Mariella D’Adda,
Marzia Salvucci,
Fabio Stagno,
Giovanna RegeCambrin,
Mario Tiribelli,
Bruno Martino,
Monica Bocchia,
Michele Cedrone,
Elena Trabacchi,
Francesco Cavazzini,
Ferdinando Porretto,
Federica Sorà,
Maria Pina Simula,
Francesco Albano,
Simona Soverini,
Robin Foà,
Fabrizio Pane,
Michèle Cavo,
Giuseppe Saglio,
Michele Baccarani,
Gianantonio Rosti
Publication year - 2022
Publication title -
haematologica
Language(s) - English
Resource type - Journals
SCImago Journal Rank - 2.782
H-Index - 142
eISSN - 1592-8721
pISSN - 0390-6078
DOI - 10.3324/haematol.2021.280175
Subject(s) - nilotinib , medicine , myeloid leukemia , surgery , gastroenterology , imatinib
We report the final analysis, with a 10-year follow-up, of the phase II study GIMEMA CML 0307 (NCT 00481052), which enrolled 73 adult patients (median age 51 years, range 18-83) with newly diagnosed chronic-phase (CP)-chronic myeloid leukemia (CML) to investigate the efficacy and the toxicity of front-line treatment with nilotinib (NIL). The initial dose was 400 mg twice daily; the dose was reduced to 300 mg twice daily as soon as this dose was approved and registered. The 10-year overall survival and progression-free survival were 94.5%. At the last contact, 36 (49.3%) patients were continuing NIL (22 patients at 300 mg twice daily, 14 at lower doses), 18 (24.7%) patients were in treatment-free remission (TFR), 14 (19.2%) were receiving other tyrosinekinase inhibitors and 4 (5.5%) patients have died.The rates of major (MMR) and deep (MR4) molecular responses by 10 years were 96% and 83%, respectively. The median time to MMR and MR4 were 6 and 18 months, respectively. After a median duration of NIL treatment of 88 months, 24 (32.9%) patients discontinued NIL while in stable deep molecular response. In these patients, the 2-years estimated treatment-free survival was 72.6%. The overall TFR rate, calculated on all enrolled patients, was 24.7% (18/73 patients). Seventeen patients (23.3%), at a median age of 69 years, had at least one arterial obstructive event.In conclusion, the use of NIL front-line in CP-CML can induce a stable TFR in a relevant number of patients, although cardiovascular toxicity remains of concern