Treatment-Free Remission in Chronic Myeloid Leukemia: Can We Identify Prognostic Factors?

Simple Summary Chronic myeloid leukemia (CML) is a blood cancer. Unlike other cancers CML treatment is lifelong and many patients experience side effects. For those patients who respond well to treatment and achieve deep molecular remission, quality of life is impacted because of continuous treatment. In this review, we look at emerging clinical trials which aim to investigate which patients can safely stop treatment. Treatment-free remission is the ultimate goal for CML patients, but there is still a gap in our knowledge as to why some patients can achieve treatment-free remission, while others relapse when treatment is stopped. Here we discuss if there are any prognostic factors that can predict the best candidates who qualify for treatment discontinuation, with a view to keeping them in remission. Following the development of tyrosine kinase inhibitors (TKI), the survival of patients with chronic myeloid leukaemia (CML) drastically improved. With the introduction of these agents, CML is now considered a chronic disease for some patients. Taking into consideration the side effects, toxicity, and high cost, discontinuing TKI became a goal for patients with chronic phase CML. Patients who achieved deep molecular response (DMR) and discontinued TKI, remained in treatment-free remission (TFR). Currently, the data from the published literature demonstrate that 40-60% of patients achieve TFR, with relapses occurring within the first six months. In addition, almost all patients who relapsed regained a molecular response upon retreatment, indicating TKI discontinuation is safe. However, there is still a gap in understanding the mechanisms behind TFR, and whether there are prognostic factors that can predict the best candidates who qualify for TKI discontinuation with a view to keeping them in TFR. Furthermore, the information about a second TFR attempt and the role of gradual de-escalation of TKI before complete cessation is limited. This review highlights the factors predicting success or failure of TFR. In addition, it examines the feasibility of a second TFR attempt after the failure of the first one, and the current guidelines concerning TFR in clinical practice.

Automated summary

Chronic myeloid leukemia (CML) is a lifelong blood cancer treatment that can lead to side effects and impact quality of life for some patients. Clinical trials are investigating which patients can safely stop treatment in order to achieve treatment-free remission. Currently, there is limited understanding of the mechanisms behind treatment-free remission and identifying prognostic factors for successful discontinuation of treatment.