Key clinical point: More than 3 years of frontline nilotinib treatment was effective with sustained long-term high treatment-free remission (TFR) rates and manageable safety in patients with Philadelphia chromosome-positive (Ph+) chronic-phase chronic myeloid leukemia (CML-CP).
Major finding: Among patients who entered TFR, 41.6% remained in major molecular response (MMR), with 40.0% in MR 4.5, whereas 98.9% of patients who lost MMR regained after treatment reinitiation. No disease progression or CML-related deaths were observed, and the adverse event profile was consistent with that reported previously.
Study details: Findings are from a 5-year follow-up of phase 2 ENESTfreedom trial including 190 adult patients with Ph+ CML-CP who received at least 2 years of frontline nilotinib treatment, achieved MR 4.5, and attempted TFR after undergoing a 1-year nilotinib treatment consolidation phase.
Disclosures: This study was funded by Novartis Pharmaceuticals. The lead author along with other authors reported ties with various pharmaceutical companies including Novartis. P Aimone, S Li, and K Titorenko reported being employees of Novartis.
Source: Radich JP et al. Leukemia. 2021 Mar 11. doi: 10.1038/s41375-021-01205-5 .