Treatment-free remission attempts in patients with chronic myeloid leukemia (CML), who have discontinued second-line nilotinib therapy after achieving a deep molecular response, are safe and likely to be achievable in the majority of patients in this setting, a study presented at the 2016 American Society of Clinical Oncology (ASCO) Annual Meeting has shown.1

For this single-arm, phase 2 trial, researchers enrolled 163 patients with CML in chronic phase who were treated with a tyrosine kinase inhibitor for at least 3 years, including imatinib for more than 4 weeks followed by nilotinib for at least 2 years, and who had achieved a deep molecular response with niolotinib.

Patients continued nilotinib for 1 year, at which point patients without confirmed loss of deep molecular response were eligible to discontinue nilotinib. Upon confirmed loss of deep or major molecular response, nilotinib was reinitiated.

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Results showed that 126 patients entered treatment-free remission after a median therapy duration  of 53 months. Of these patients, 57.9% (95% CI, 48.8-66.7) remained in treatment-free remission at 48 weeks.

Researchers found that during treatment-free remission, 18 patients had confirmed loss of deep molecular response, 34 lost major molecular response, and 1 patient came off the study due to atypical transcript. Of the 51 patients who reinitiated nilotinib, 98% re-achieved at least a major molecular response and most regained a deep molecule response.

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No new safety signals were observed on nilotinib treatment. All grade musculoskeletal pain (42.1% versus 14.3%) and fluid retention (10.3% versus 1.6%) occurred more frequently in treatment-free remission group, in contrast with patients receiving nilotinib consolidation.
Cardiovascular events were more common in the nilotinib group (4.8% versus 0%).                                  


  1. Hughes TP, Boquimpani C, Kim DW, Benyamini N, Clementino NC, Shuvaev V, et al. Treatment-free remission (TFR) in patients (pts) with chronic myeloid leukemia in chronic phase (CML-CP) treated with second-line nilotinib (NIL): First results from the ENESTop study. J Clin Oncol. 2016; 34 (suppl; abstr 7054).