THE CONCEPT OF "CURE"
The CML-community has set the achievement of a cure for CML as being their number one strategic priority. But what is "cure"? How could a "cure" in CML look like? In preparation for the Academic CML-CAB, CML-CAB members have debated around this question in meetings and interactive workshops, trying to reach consensus on this question. The first results of this community consensus were presented at the Academic CML-CAB on 12 October. In an urgent appeal, the CML-community insisted that CML (which today has become a chronic disease for most patients thanks to 5 TKIs currently available) is often considered a “lucky cancer”, a “done deal”, or a “ticked box”. However, in many countries still today there are severe access issues (both to therapy and PCR). Also the side effects burden of a life-long therapy, the toxicity and non-adherence should not be underestimated. Furthermore, TKI-treatment is a real burden especially on younger patients (since it seriously hinders family planning) as well as a financial burden on healthcare systems due to increasing prevalence. TFR requires a stable deep molecular response which is only possible after many years of deep remission (every +1 year DMR -3% relapse rate). Furthermore, TFR success is only likely for at best 25-35% of patients, the reasons for this being: 1.) requirements to stop treatment aren´t met, 2.) no or limited access to PCR testing, and/or 3.) 50% relapse rate even with best TKI. Moreover, life-long PCR monitoring (if available at all) is a constant reminder of the disease. Will healthcare systems cover this after years of TFR? What about late relapses? While TFR does not eradicate disease, the stigma of cancer adheres to patients, treatment discontinuation causes fear, anxiety and depression, and there remains a risk of undetected (late) relapses that may lead to progression and death.
The CML- community therefore calls for
– All CML patients (which is not given with TFR)
- Recognition that cure is an unmet need in CML, not a done deal with TFR
- Joint definition of the problem the community/CML-CAB trying to solve:
– can live a normal, long life (which is usually given with TFR)
– with equivalent quality of life of a normal person (there is uncertainty if
this is given with TFR)
– in absence of any CML disease (which is not given with TFR)
– without need of any CML therapy (which is given with TFR)
– without need of continuous monitoring (which is not given with TFR)
- Commitment and collaboration between all stakeholders, first and foremost patient community and CML researchers to finding a real cure for CML
- More funding for research on a cure for CML
- Building a joint, patient-centric “Research Network for CML cure” that helps coordinate, drive and fund research in that space
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