Treatment free remission


Treatment free remission in CML

The advent of TKIs into the management of patients with CML has a profoundly improved the prognosis of CML patients, with a survival under continued therapy to be close to that of the normal population.

Following a lot of public and professional debate about previous and ongoing STOP trials in CML, stopping CML treatment has become a widely debated topic. According to current first-line studies (ENEST1st, ENESTnd, DASISION), about 65-75% of CML patients will, over years, achieve a durable deep molecular response (minimum MR4) on current TKI treatments. Of those who then stop treatment over some years, about half of them seem to remain in durable therapy-free remission.

Therefore, Treatment Free Remission (TFR) in CML is referred to having a stable deep molecular response without the need for ongoing Tyrosine Kinase Inhibitor (TKI) treatment after having been in deep molecular residual disease over a longer period of time.


Recommendations on Treatment-Free Remission in CML 

First recommendations exist about how to manage to stop and re-start therapy, based on early data from previous studies like the French STIM studies, the largest stop study EUROSKI as well as company-sponsored studies ENESTop, ENESTfreedom and DASFREE.

NCCN (USA) and ESMO (Europe) have published first recommendations for stopping treatment outside the context of clinical trials and the institutional requirements of the centre and lab. A summary of what was presented at 59th ASH Annual Meeting and Exposition last 2017 is available HERE.

However, there is still no wide consensus among the CML experts, prognostic factors for TFR success are not yet clear, and the emotional impact is not yet well understood by doctors and patients.



The CML Advocates Network is currently undertaking a global project focused on TFR, because this topic is being debated by doctors, patients, regulators and pharmaceutical industry for different reasons, and the patient community feels this also needs careful, considerate communication in all channels based on the patients perspective.

The “TFR 4 CML PATIENTS” project consists of a global online survey of TFR patients to collect the viewpoints, needs and experiences of CML patients that are considering or have already started Treatment Free Remission (TFR). The aim is to provide CML patients and healthcare providers with more evidence when considering or managing TFR.

For more information about the TFR 4 CML PATIENTS survey, visit “TFR 4 CML PATIENTS SURVEY” webpage.








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