Assessing treatment-free remission in adult Ph+ CML-CP patients
ENESTfreedom, evaluated stopping treatment in adult Ph+ CML-CP patients who started on TASIGNA and met strict eligibility criteria to attempt TFR1,2
ENESTfreedom is a single-arm, phase II trial, conducted at 132 sites across 19 countries, 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.1
ENESTfreedom is the largest frontline TFR study for TASIGNA (n=190 entered TFR phase) and also the second-largest frontline CML study overall.2
The study design emphasized the importance of frequent monitoring of patients who stop TKI to ensure timely retreatment.2
Patients entered the TFR phase after sustaining a deep molecular response during the consolidation phase of the trial. The 4 last quarterly RQ-PCR assessment must fulfill the following criteria to be eligible to enter the TFR phase and stop TASIGNA treatment: patients with MR4.5 in their last assessment; no assessment worse than MR4.0; and ≤2 assessments between MR4.0 and MR4.5.2
a Sustained DMR defined as (looking at the most recent 4 quarterly RQ-PCR assessments) MR4.5 in the last assessment, no assessment worse than MR4.0, and ≤2 assessments between MR4.0 and MR4.5.
b Patients with a loss of MR4.0 during the TFR phase will undergo PCR monitoring every 2 weeks.
c Patients who did not sustain DMR during the consolidation phase continued to receive TASIGNA. If DMR was reached and sustained, then those patients could enter the TFR-2 phase of the trial (data not yet available for this phase).
CP, chronic phase; DMR, deep molecular response; MMR, major molecular response; MR4, molecular response 4 (4-log reductions); MR4.5, molecular response 4.5 (4.5-log reductions); TFR, treatment-free remission.
- TASIGNA® (nilotinib) Summary of Product Characteristics. Basel, Switzerland: Novartis Pharma AG; May 2017.
- Hochhaus A, et al. Leukemia [published online March 17, 2017]. 2017:1-7. doi:10.1038/leu.2017.63.