European LeukemiaNet 2020 recommendations for treating chronic myeloid leukemia
A newer version of this guideline has been published: 2025 European LeukemiaNet recommendations for the management of chronic myeloid leukemia (2025)
- Study type
- Clinical guideline
- Study name
- ELN 2020 (CML)
- Design
- Recommendations of an ELN expert panel based on published evidence and panel consensus (75 to 100% agreement in most cases)
- Source
- Leukemia · 2020
In plain words
This European guideline says that most patients with chronic-phase CML can live a near-normal lifespan with one daily tablet (a tyrosine kinase inhibitor). The response to treatment is measured with a PCR blood test at months 3, 6 and 12, so that the drug can be changed if needed. In patients who have had a very deep and lasting response for several years, the drug can be stopped under close medical supervision, and about half of them stay in remission without needing the drug.
Key guideline recommendations
- ELN panel consensus
First-line treatment of chronic-phase CML is a tyrosine kinase inhibitor (imatinib, dasatinib, nilotinib or bosutinib); generic imatinib is a cost-effective initial treatment, and the choice of drug should be based on comorbidities, possible adverse effects and the ELTS risk score.
- ELN panel consensus
Response milestones (BCR-ABL1 on the International Scale): 3 months — optimal ≤10%, failure >10% if confirmed within 1 to 3 months; 6 months — optimal ≤1%, warning >1 to 10%, failure >10%; 12 months — optimal ≤0.1% (major molecular response, MMR), warning >0.1 to 1%, failure >1%. At any time: loss of MMR is a warning, and BCR-ABL1 >1%, resistance mutations or high-risk chromosomal abnormalities are failure.
- ELN panel consensus
In case of failure or resistance, a change of treatment is mandatory and should be accompanied by BCR-ABL1 kinase domain mutation testing; with the T315I mutation, only ponatinib is effective. An inadequate response to two or more TKIs should prompt early evaluation for allogeneic transplantation.
- ELN panel consensus
TKI discontinuation for treatment-free remission (TFR) — minimum requirements: CML in first chronic phase only; motivated patient with structured communication; access to high-quality quantitative PCR on the International Scale with rapid turnaround; acceptance of frequent monitoring after stopping (monthly for the first 6 months, every 2 months in months 6 to 12 and every 3 months thereafter); first-line treatment (or second line only because of intolerance); typical e13a2 or e14a2 transcript; treatment duration more than 5 years (more than 4 years for a second-generation TKI); duration of DMR (MR4 or better) more than 2 years; no prior treatment failure.
- ELN panel consensus
Optimal conditions for TKI discontinuation: treatment duration more than 5 years, and DMR duration more than 3 years for MR4 or more than 2 years for MR4.5. Loss of MMR after stopping is considered TFR failure, and treatment should be restarted.
- ELN panel consensus
TKI treatment should be stopped during pregnancy.
Study population
Patients with chronic myeloid leukaemia (CML) in chronic phase and advanced phases; covering first-line and subsequent treatment, molecular response monitoring, and treatment discontinuation for treatment-free remission (TFR).
Clinical significance and place in guidelines
The ELN 2020 molecular milestones (3, 6 and 12 months) and TKI discontinuation criteria are the basis of everyday monitoring and decision-making in CML worldwide. After publication of this guideline, asciminib was approved in the United States first for later lines and then (based on ASC4FIRST) for first line, which is not covered in this edition.
Limitations
The recommendations are based on published evidence and panel consensus and have no formal grading of the level of evidence. This edition was published before the approval of first-line asciminib.
This summary was prepared by the editorial team from the published paper; the original paper is the final reference. Read the original abstract on PubMed.