Real-World Study Identifies Factors Associated With Successful TKI Discontinuation in CML
Key Takeaways:
- Among 698 patients with chronic myeloid leukemia (CML) who discontinued tyrosine kinase inhibitor (TKI) therapy, rates of treatment-free remission (TFR) rates decreased from 66.5% at 6 months to 55.5% at 24 months.
- Deep molecular response (DMR), Sokal scores, and treatment duration emerged as notable predictors of TFR among patients with CML.
- The findings provide clinical factors that may help physicians assess the likelihood of maintaining TFR after TKI discontinuation and inform postdiscontinuation monitoring.
TFR is an important treatment goal for patients with CML, and research has shown that approximately 50% of patients can maintain TFR after discontinuing TKI therapy.
Researchers sought to identify the factors associated with successful treatment discontinuation and develop models to predict TFR among patients with CML.
The study included 698 patients with CML from 11 centers in China who discontinued TKI therapy between 2001 and 2020. The primary outcome was maintenance of major molecular response (MMR) within 12 months after treatment discontinuation.
TFR After TKI Discontinuation
After treatment discontinuation, 318 patients (45.6%) lost MMR. Loss of DMR was associated with a greater likelihood of relapse after TKI discontinuation.
TFR decreased from 66.5% at 6 months to 55.5% at 24 months. Sustained TFR rates were 73.5%, 60.0%, 44.6%, and 20.6% for patients with 0, 1, 2, and 3 or more nonoptimal factors, respectively.
Higher TFR rates were associated with lower Sokal scores, absence of treatment resistance, longer treatment duration, and sustained DMR. These factors may help identify patients more likely to maintain TFR after TKI discontinuation and inform postdiscontinuation monitoring.
Implications for Managed Care
The findings suggest that TKI discontinuation and sustained TFR may be achievable in appropriately selected patients, including some with nonoptimal clinical characteristics. Identifying predictors of successful TFR may help inform patient selection and monitoring strategies following treatment discontinuation.
The authors concluded, "Patients achieving durable DMR can safely discontinue therapy with rigorous monitoring."
Reference
Cheng F, Wang Y, Zhu Y, et al. Real-world evidence-based study on treatment-free remission in chronic myeloid leukemia. JNCCN. 2026. doi:10.6004/jnccn.2026.7026
