Real-World Evidence-Based Study on Treatment-Free Remission in Chronic Myeloid Leukemia
Fang Cheng1,2,3, Yu Wang4, Yu Zhu5
11Department of Pharmacy, Union Hospital, Tongji Medical College, Huazhong University of Science and Technology, Wuhan, Hubei, China.
Summary
Treatment-free remission (TFR) is achievable for chronic myeloid leukemia (CML) patients with deep molecular response (DMR). Predictive models can guide safe TKI discontinuation and TFR attempts with monitoring.
Area of Science:
- Hematology
- Oncology
- Pharmacology
Background:
- Treatment-free remission (TFR) is an emerging goal for chronic myeloid leukemia (CML).
- Defining precise conditions for treatment discontinuation is crucial for CML management.
- Developing predictive models for TFR in Chinese CML patients is needed.
Purpose of the Study:
- To define conditions for tyrosine kinase inhibitor (TKI) discontinuation in CML.
- To develop TFR prediction models for a Chinese CML population.
- To assess the safety and efficacy of TFR in CML.
Main Methods:
- Retrospective analysis of 802 CML patients undergoing TKI discontinuation across 11 centers.
- Inclusion criteria: ≥3 years TKI therapy and ≥2 years deep molecular response (DMR).
- Primary endpoint: Major molecular response (MMR) maintenance within 12 months post-discontinuation.
Main Results:
- 45.6% of 698 eligible patients lost MMR, primarily within 6 months.
- Sustained TFR rates were 57.7% at 12 months and 55.5% at 24 months.
- Predictive factors for TFR included longer TKI duration, sustained DMR, low Sokal risk, and no resistance. Predictive models and a calculator were developed.
Conclusions:
- Durable DMR allows safe TKI discontinuation in CML patients with monitoring.
- TFR is viable even with nonoptimal factors if DMR is adequate and monitoring is close.
- Reinitiation of TKI therapy ensures MMR/DMR recovery without progression.