FIRST-LINE DATA

Focus on sustained DMR, a key criterion of TFR eligibility in the firstline1,2

  • Enrollment criteria: adults with Ph+ CML-CP; e13a2/b2a2 and/or e14a2/b3a2 transcripts; ≥2 years on frontline TASIGNA; and MR4.5 at screening
  • The study design emphasized the importance of frequent monitoring of patients who stop TKI to ensure timely retreatment2
  • Patients entered the TFR phase after sustaining a deep molecular response during the consolidation phase of the trial. Patients with MR4.5 in their last assessment, no assessment worse than MR4.0, and ≤2 assessments between MR4.0 and MR4.5 were eligible to enter the TFR phase and safely stop TASIGNA treatment2

SECOND-LINE DATA

For those patients who switched from imatinib: Focus on sustained DMR, a key criterion for the opportunity of TFR eligibility in the second-line1,3

  • Enrollment criteria: adults with Ph+ CML-CP; e13a2/b2a2 and/or e14a2/b3a2 transcripts; ≥3 years of TKI therapy (first imatinib for >4 weeks, then TASIGNA for ≥2 years); No documented MR4.5 at time of switch to TASIGNA; and achieved MR4.5 on TASIGNA3
  • The study design emphasized the importance of frequent monitoring of patients who stop TKI therapy to ensure timely retreatment3
  • Patients entered the TFR phase after achieving a deep molecular response during the consolidation phase of the trial. Patients who maintained MR4.5 during the consolidation phase (no loss of MR4.5 confirmed in a consecutive assessment within 4 weeks) were eligible to enter the TFR phase and safely stop TASIGNA treatment3

ENESTfreedom is a single-arm, phase II trial assessing the potential for treatment-free remission in 215 patients with Ph+ CML-CP following frontline nilotinib treatment. The primary efficacy endpoint was the proportion of patients who were in MMR without reinitiation of treatment at week 48 of the TFR phase.

ENESTop is a single-arm, phase II trial assessing treatment-free remission in 163 patients with Ph+ CML-CP who had switched from imatinib to nilotinib. The primary efficacy endpoint was the proportion of patients with successful TFR at 48 weeks, defined as no loss of MMR or confirmed loss of MR4 (2 consecutive RQ-PCR tests) within the first 48 weeks of TFR.

  1. TASIGNA® (nilotinib) Summary of Product Characteristics. Basel, Switzerland: Novartis Pharma AG; May 2017.
  2. Hochhaus A, et al. ENESTfreedom Study. Leukemia [published online March 17, 2017]. 2017:1-7. doi:10.1038/leu.2017.63.
  3. Hughes TP, et al. J Clin Oncol. 2016;34(s):Abstract 7054.