Second-line nilotinib may lead to maintained molecular response and treatment-free remission that can last 48 weeks or longer for patients with chronic myeloid leukemia (CML), findings from a phase 2 study suggest.
Treatment-free remission (TFR) is an emerging treatment goal for patients with CML in the chronic phase, according to François-Xavier Mahon, MD, PhD, of the University of Bordeaux (France) and his colleagues. “Potential motivators and benefits of achieving TFR may include relief of treatment side effects, reduced risk for long-term [tyrosine kinase inhibitor] toxicity, and the ability to plan a family,” they wrote. “When TFR is a treatment goal, achievement of [deep molecular response] is a key prerequisite.”
Established molecular response benchmarks include major molecular response (MMR), MR4 and MR4.5, according to the researchers.
In an open-label phase 2, the researchers enrolled patients with Philadelphia chromosome-positive CML who received nilotinib for 2 years or longer after having received imatinib for longer than 4 weeks. The other key criterion for enrollment was achieving MR4.5 during treatment with nilotinib, according to the study, published in .
In total, 163 patients were enrolled and entered the 1-year consolidation phase. Of those patients, 126 were eligible for the TFR phase during which nilotinib treatment was stopped.