Treatment-Free Remission in CML: Long-Term Monitoring and Care Approach

In the management of CML, assessing treatment response is centered around achieving defined molecular milestones, which are critical indicators of long-term outcomes. Guidelines recommend transcript levels be reduced to below 10% by 3 to 6 months and ideally below 1% by 6 months, with a complete cytogenetic response by 12 months. Failure to meet these benchmarks prompts an evaluation for underlying causes, such as drug-drug interactions or patient adherence issues. Before considering treatment failure or resistance, clinicians typically repeat polymerase chain reaction (PCR) testing to confirm trends and may proceed with mutational analysis if abnormalities persist.

For patients who achieve deep and sustained molecular responses (MRs), TFR may become a viable goal. Candidates for TFR should have been on a tyrosine kinase inhibitor (TKI) for at least 3 years and maintained a deep MR (MR4 or MR4.5) for 2 or more years without prior signs of resistance or aggressive disease. Once therapy is discontinued, patients are closely monitored with monthly PCR testing for the first 6 months, followed by less frequent intervals. Although most patients who relapse after TFR can regain prior responses upon resuming the same therapy, the decision to discontinue is highly individualized, taking into account patient anxiety, adherence history, and clinical suitability.

Medication adherence remains a cornerstone of successful CML treatment, and pharmacists play a key role in supporting this. Through close collaboration with nurses and specialty pharmacies, they help monitor for missed doses, potential interactions (such as with proton pump inhibitors), and barriers to therapy. Counseling strategies that use open, nonjudgmental language can encourage honesty about adherence challenges. This multidisciplinary approach ensures patients are not only clinically monitored but also emotionally and practically supported throughout their treatment journey, especially when considering transitions like TFR.