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Development of a blood test-based predictive scoring tool for treatment response in chronic myeloid leukemia.

Kohjin Suzuki1,2, Tomoiku Takaku3,4, Naoki Watanabe1

  • 1Department of Hematology, Juntendo University Graduate School of Medicine, Tokyo, Japan.

International Journal of Hematology
|December 13, 2025
PubMed
Summary

A new scoring tool predicts optimal response (OR) in chronic myeloid leukemia (CML) patients within 12 months. This tool uses diagnostic blood parameters to guide personalized treatment selection and improve outcomes.

Keywords:
Chronic myeloid leukemiaOptimal responseTreatment response prediction

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Area of Science:

  • Hematology
  • Oncology
  • Molecular Biology

Background:

  • European LeukemiaNet (ELN) guidelines define optimal response (OR) in chronic myeloid leukemia (CML) as achieving major molecular response (MMR) within 12 months.
  • Currently, no validated tools exist to predict treatment response at the time of diagnosis for CML patients.

Purpose of the Study:

  • To develop and validate a predictive scoring tool for optimal response (OR) and deep molecular response (DMR) in CML.
  • To utilize readily available peripheral blood parameters at diagnosis for predicting treatment outcomes.

Main Methods:

  • Retrospective analysis of 535 CML patients from the CML Cooperative Study Group database.
  • Logistic regression analysis to identify significant predictors of OR, including white blood cell count, platelet count, eosinophil percentage, and imatinib use.
  • Construction and validation of a novel predictive scoring tool based on identified parameters.

Main Results:

  • The novel scoring tool was significantly higher in the non-OR group (MMR after 13 months) compared to the OR group (MMR within 12 months).
  • The developed score demonstrated superior predictive accuracy for OR compared to existing prognostic scores.
  • Lower scores correlated with higher rates of deep molecular response (DMR) achievement.
  • The score effectively predicted OR in imatinib-treated patients with low/intermediate ELTS risk.

Conclusions:

  • A novel scoring tool using diagnostic peripheral blood parameters can predict optimal and deep molecular response in CML.
  • This tool has the potential to guide individualized treatment selection, including upfront tyrosine kinase inhibitor choice.
  • The development of this scoring tool advances precision medicine in the management of chronic myeloid leukemia.