Long-term treatment-free remission in patients with chronic myeloid leukemia after second-line nilotinib: ENESTop 5-year update

Timothy P. Hughes, Nelma Cristina D. Clementino, Mikhail Fominykh, Jeffrey H. Lipton, Anna G. Turkina, Elena Beatriz Moiraghi, Franck E. Nicolini, Naoto Takahashi, Tomasz Sacha, Dong Wook Kim, Rafik Fellague-Chebra, Ranjan Tiwari, Catherine Bouard, Francois Xavier Mahon

Research output: Contribution to journalArticlepeer-review


The ENESTop study evaluated treatment-free remission (TFR) in patients with chronic myeloid leukemia (CML) in chronic phase who had received ≥3 years of tyrosine kinase inhibitor therapy and achieved sustained deep molecular response only after switching from imatinib to nilotinib. After 1-year nilotinib consolidation, 126 patients attempted TFR. At 48 weeks (primary analysis), 57.9% (73/126) were in TFR. In the present analysis at 5 years, 42.9% (54/126) were in TFR. Since the 48-week analysis, among patients who left the TFR phase, 58% (11/19) did not have a loss of molecular response and discontinued for other reasons. Of the 59 patients who reinitiated nilotinib upon loss of major molecular response (MMR) or confirmed loss of MR4, 98.3% regained MMR, 94.9% regained MR4, and 93.2% regained MR4.5. Overall adverse event rates decreased over the 5 years of TFR. In patients reinitiating nilotinib, there was a cumulative increase in cardiovascular events with longer nilotinib exposure. No disease progression or CML-related deaths were reported. Overall, these results confirm the durability and safety of TFR for patients receiving second-line nilotinib. Cardiovascular risk should be carefully managed, particularly when reinitiating treatment after TFR.

Original languageEnglish
Pages (from-to)1631-1642
Number of pages12
Issue number6
Publication statusPublished or Issued - Jun 2021

ASJC Scopus subject areas

  • Hematology
  • Oncology
  • Cancer Research

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