Abstract
The ENESTfreedom trial assessed the feasibility of treatment-free remission (TFR) in patients with chronic myeloid leukemia in chronic phase (CML-CP) following frontline nilotinib treatment. Results for long-term outcomes after a 5-year follow-up are presented herein. Patients who had received ≥2 years of frontline nilotinib therapy and achieved MR4.5 underwent a 1-year nilotinib treatment consolidation phase before attempting TFR. At the 5-year data cut-off, 81/190 patients entering the TFR phase (42.6%) were still in TFR, with 76 (40.0%) in MR4.5. Patients who lost major molecular response (MMR) entered a treatment re-initiation phase; 90/91 patients entering this phase (98.9%) regained MMR and 84/91 patients (92.3%) regained MR4.5. The Kaplan–Meier estimated treatment-free survival rate at 5 years was 48.2%. No disease progression or CML-related deaths were reported. Whereas the incidence of adverse events (AEs) declined from 96 weeks following the start of TFR, an increase in AE frequency was observed for patients in the treatment re-initiation phase. Low Sokal risk score, BCR-ABL1IS levels at 48 weeks of TFR and stable MR4.5 response for the first year of TFR were associated with higher TFR rates. Overall, these results support the efficacy and safety of attempting TFR following upfront nilotinib therapy of >3 years in patients with CML-CP.
Highlights
With patients in Western nations having a life expectancy similar to those of age-matched individuals in the general population [1,2,3,4,5,6]. This success has shifted the focus of chronic myeloid leukemia (CML) treatment from survival to improved quality of life and reduction of adverse events (AEs) [7,8,9]
Whereas previously life-long treatment with tyrosine kinase inhibitors (TKIs) was recommended, current guidelines for CML management support the safety of the treatment-free remission (TFR) approach for certain patients and provide guidance on patient selection and monitoring [5, 12, 13]
To evaluate the long-term outcomes of patients in TFR following frontline nilotinib treatment, we present here an updated analysis of the ENESTfreedom study after a 5-year follow-up period
Summary
Heidelberg, Mannheim, Germany Developmental Therapeutics Consortium, Chicago, IL, USA Division of Haematology, SA Pathology, Royal Adelaide. Life-long treatment represents a major cost burden on healthcare systems, considering the increase in life expectancy, and may result in non-adherence to treatment for those patients who cannot afford it [9, 11] For these reasons, whereas previously life-long treatment with TKIs was recommended, current guidelines for CML management support the safety of the treatment-free remission (TFR) approach for certain patients and provide guidance on patient selection and monitoring [5, 12, 13]. To evaluate the long-term outcomes of patients in TFR following frontline nilotinib treatment, we present here an updated analysis of the ENESTfreedom study after a 5-year follow-up period
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