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The initial molecular response predicts the deep molecular response but not treatment-free remission maintenance in a real-world chronic myeloid leukemia cohort

  • 0Hématologie FBeirorlaongdiq, uFer,a nCcHe U Clermont-Ferrand, Clermont; Equipe d'Accueil EA7453 CHELTER, Université Clermont Auvergne, Clermont-Ferrand.
Haematologica +

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May 2, 2024

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May 2, 2024

View abstract on PubMed

Summary

This summary is machine-generated.

Identifying early molecular predictors for stable treatment-free remission (TFR) in chronic myeloid leukemia is crucial. While early BCR::ABL1 levels and halving time predict treatment response, they do not reliably predict TKI discontinuation or sustained TFR.

Area Of Science

  • Hematology
  • Molecular Biology
  • Oncology

Background

  • Identifying early molecular predictors for stable treatment-free remission (TFR) after tyrosine kinase inhibitor (TKI) discontinuation in chronic myeloid leukemia (CML) remains a challenge.
  • Previous studies have described the predictive values of residual disease (BCR::ABL1 quantification) and BCR::ABL1 transcript halving time (HT), but direct comparisons are lacking.

Purpose Of The Study

  • To compare the predictive performance of early molecular response parameters for predicting optimal response, TKI discontinuation criteria achievement, and TFR maintenance in CML patients.
  • To evaluate the influence of first-line treatment (imatinib vs. second-generation TKI) on these predictions.

Main Methods

  • A real-world cohort of 408 CML patients was analyzed.
  • The study compared the EUTOS long-term survival (ELTS) score, BCR::ABL1 HT, and residual disease levels at months 3 and 6.
  • Outcomes assessed included molecular response, achievement of TKI discontinuation criteria, and TFR maintenance.

Main Results

  • BCR::ABL1 HT and month 3 residual disease showed similar predictive performance.
  • Month 6 residual disease demonstrated the best performance for predicting optimal molecular response (MR4/MR4.5).
  • No early parameter reliably predicted the achievement of TKI discontinuation criteria or sustained TFR maintenance.

Conclusions

  • Early therapeutic response parameters effectively indicate TKI efficacy and identify best responders in CML.
  • These early markers do not predict the ability to discontinue TKIs or maintain TFR, suggesting other factors influence TFR.
  • A relationship between ELTS score, early milestones, and TFR maintenance was observed specifically in the second-generation TKI group.