Abstract
Treatment of chronic myeloid leukemia (CML) has evolved dramatically in recent years. In this regard, the introduction of second-generation tyrosine kinase inhibitors (TKI) has revolutionized therapeutic goals, and it is now desirable to obtain treatment-free remission (TFR), i.e. when a patient who has stopped TKI therapy maintains a major molecular response and does not need to restart treatment. This report summarizes the main findings from a group of expert hematologists in Italy who met to discuss treatment and management of patients with CML with focus on broad-ranging aspects of TFR. A survey was used to obtain information about the clinicians’ experience with TFR and to better understand the clinical and psychological issues that patients and physicians face when considering TFR. The overall goal was to explore the possibility of discontinuing treatment from multiple points of view, considering both clinical aspects of TFR as well as psychological management of patients. Practical information is provided on aspects associated with initiating TFR, clinical data supporting it, the role of monitoring, and management of discontinuation-related adverse events. This publication outlines many of the shortcomings and highlights proposed solutions for routine clinical practice, and provides an overview of the literature relative to TFR.
Highlights
Treatment of chronic myeloid leukemia (CML) has evolved dramatically in recent years
The overarching aim of the project was to explore the possibility of discontinuing treatment from multiple points of view, considering the clinical aspects of treatment-free remission (TFR), and psychological management of patients
The most recent trials that led to the approval of secondgeneration tyrosine kinase inhibitors (TKI) in first line therapy (DASISION and ENESTnd) showed that nilotinib and dasatinib are associated with a much greater depth of response compared to imatinib (MR4.5 at 5 years were 54% vs 31% and 42% vs 33%, respectively) [20, 21]
Summary
Treatment of chronic myeloid leukemia (CML) has evolved dramatically in recent years. In this regard, the introduction of second-generation tyrosine kinase inhibitors (TKI) has revolutionized therapeutic goals. While in the past the goal for patients with CML was limited to hematological control of disease in the long term, for many patients it is desirable to obtain treatment-free. Achieving Treatment-Free Remission in CML remission (TFR). TFR occurs when a patient who has stopped TKI therapy maintains a major molecular response (MMR) and does not need to restart treatment. Patients in the chronic phase who have maintained a deep MR (DMR) that is stable for at least 2 years are considered to be good candidates for discontinuation of TKI therapy. After discontinuation of a TKI, overt recurrence is considered when loss of MMR is confirmed [1]
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