Laboratory recommendations for scoring deep molecular responses following treatment for chronic myeloid leukemia
N C P Cross1, H E White1, D Colomer2
11] Faculty of Medicine, University of Southampton, Southampton, UK [2] National Genetics Reference Laboratory (Wessex), Salisbury District Hospital, Salisbury, UK.
Abstract:
Treatment of chronic myeloid leukemia (CML) with tyrosine kinase inhibitors has advanced to a stage where many patients achieve very low or undetectable levels of disease. Remarkably, some of these patients remain in sustained remission when treatment is withdrawn, suggesting that they may be at least operationally cured of their disease. Accurate definition of deep molecular responses (MRs) is therefore increasingly important for optimal patient management and comparison of independent data sets. We previously published proposals for broad standardized definitions of MR at different levels of sensitivity. Here we present detailed laboratory recommendations, developed as part of the European Treatment and Outcome Study for CML (EUTOS), to enable testing laboratories to score MR in a reproducible manner for CML patients expressing the most common BCR-ABL1 variants.
Insights
Many chronic myeloid leukemia (CML) patients achieve deep molecular responses (MRs) with tyrosine kinase inhibitors. Standardized definitions and laboratory recommendations are crucial for assessing sustained remission and potential cures in CML treatment.
Area of Science:
- Hematology
- Oncology
- Molecular Biology
Background:
- Tyrosine kinase inhibitors have significantly improved chronic myeloid leukemia (CML) treatment outcomes.
- Many CML patients now achieve deep molecular responses (MRs), with some maintaining remission after treatment cessation, indicating potential operational cures.
- Accurate assessment of MRs is vital for patient management and comparing research data.
Purpose of the Study:
- To present detailed laboratory recommendations for scoring molecular responses in CML patients.
- To enable reproducible measurement of MRs, particularly for common BCR-ABL1 variants.
- To support the standardization of MR definitions within the European Treatment and Outcome Study for CML (EUTOS).
Main Methods:
- Development of detailed laboratory protocols for molecular response assessment.
- Focus on reproducibility in scoring MRs for CML patients.
- Inclusion of recommendations for common BCR-ABL1 variants.
Main Results:
- Established standardized laboratory recommendations for scoring MRs in CML.
- Provided a framework for reproducible MR testing.
- Addressed the assessment of MRs in patients with common BCR-ABL1 variants.
Conclusions:
- Standardized laboratory scoring of MRs is essential for managing CML patients achieving deep responses.
- These recommendations facilitate consistent and reproducible assessment of molecular remission in CML.
- The EUTOS-developed guidelines aid in evaluating sustained remission and potential cures in CML.
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