Is long-term treatment-free remission possible after frontline nilotinib treatment in patients with chronic phase chronic myeloid leukemia?

This study provided an updated 5-year assessment of treatment-free remission (TFR) after frontline nilotinib (Tasigna) treatment in patients with chronic-phase (CP) chronic myeloid leukemia (CML). The data showed sustained, long-term high TFR rates with effectiveness and safety in these patients.

Long-term treatments with targeted therapies such as nilotibib for CP-CML have successfully increased patient survival. Treatment goals have now shifted towards improved quality of life and reduced side effects.

The BCR-ABL1 gene indicates the presence of abnormal blood cells in patients with CML. When this gene is very low in the blood, it means patients have achieved a major molecular response (MMR). In these patients, a treatment-free remission (TFR) can be recommended. TFR occurs when patients achieve a deep and stable treatment response without continuing treatment.

Previous studies have shown good TFR rates for at least 6 months after stopping therapy. However, longer-term data for TFR rates in patients treated with frontline nilotinib is still needed.

The study evaluated data from 203 patients with CP-CML treated with nilotinib. All patients had at least 2 years of frontline nilotinib therapy and a year of nilotinib consolidation therapy. Consolidation therapy is given after remission is achieved to avoid relapse. Patients were followed up for 5 years.

190 patients with a MMR entered TFR, after nilotinib treatment was stopped. 81 patients (42.6%) remained in TFR for up to 5 years. Loss of DMR occurred in 91 patients and led to nilotinib re-initiation until MMR was regained. Of these, 90 patients (98.9%) regained MMR.

48.2% of patients were alive and without treatment at 5 years. It was estimated that 95.6% of patients will survive for 5 years without disease progression.

During treatment re-initiation, 19.8% of patients experienced bone and muscle pain, 18.7% had increased blood cholesterol and 17.6 % had heart and blood vessel problems.

The study found that frontline nilotinib treatment resulted in sustained, long-term TFR rates and was effective and safe in patients with CP-CML.