Treatment-free remission following frontline nilotinib in patients with chronic myeloid leukemia in chronic phase: results from the ENESTfreedom study.

A Hochhaus, T Masszi, F J Giles, J Radich, David Ross, M T Gomez Casares, Andrzej Hellmann, Jesper Stentoft, Eibhlin Conneally, Valentin Garcia-Gutierrez, Norbert Gattermann, W Wiktor-Jedrzejczak, Philipp Le Coutre, Bruno Martino, Susanne Saussele, H D Menssen, W Deng, Nancy Krunic, Veronique Bedoucha, Guiseppe Saglio

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The single-arm, phase 2 ENESTfreedom trial assessed the potential for treatment-free remission (TFR; i.e., the ability to maintain a molecular response after stopping therapy) following frontline nilotinib treatment. Patients with Philadelphia chromosome-positive chronic myeloid leukemia in chronic phase with MR4.5 (BCR-ABL1 <0.0032% on the International Scale (BCR-ABL1IS)) and ≥ 2 years of frontline nilotinib therapy were enrolled. Patients with sustained deep molecular response during the 1-year nilotinib consolidation phase were eligible to stop treatment and enter the TFR phase. Patients with loss of major molecular response (MMR; BCR-ABL1IS ≤0.1%) during the TFR phase reinitiated nilotinib. In total, 215 patients entered the consolidation phase, of whom 190 entered the TFR phase. The median duration of nilotinib before stopping treatment was 43.5 months. At 48 weeks after stopping nilotinib, 98 patients (51.6%; 95% confidence interval, 44.2-58.9%) remained in MMR or better (primary end point). Of the 86 patients who restarted nilotinib in the treatment reinitiation phase after loss of MMR, 98.8% and 88.4%, respectively, regained MMR and MR 4.5 by the data cutoff date. Consistent with prior reports of imatinib-treated patients, musculoskeletal pain-related events were reported in 24.7% of patients in the TFR phase (consolidation phase, 16.3%).

Original languageEnglish
Pages (from-to)1525-1531
Number of pages7
Issue number7
Publication statusPublished - 1 Jul 2017
Externally publishedYes

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  • chronic myeloid leukemia
  • nilotinib
  • Treatment-free remission
  • ENESTfreedom study


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