Clinical outcomes of allogeneic cell transplantation for patients with chronic myeloid leukemia

Benjamin McCormick1,2, Anthony Findley2, Kendall Diebold1

  • 1Division of Hematology/Oncology, University of Alabama at Birmingham, Birmingham, AL, USA.

Leukemia & Lymphoma
|October 10, 2025
PubMed

Insights

Allogeneic hematopoietic cell transplantation (allo-HCT) is effective for chronic myeloid leukemia (CML) in blast phase or TKI-resistant CML. Outcomes vary by CML subtype, with myeloid blast phase showing higher relapse rates.

Area of Science:

  • Hematology
  • Oncology
  • Stem Cell Transplantation

Background:

  • Allogeneic hematopoietic cell transplantation (allo-HCT) is a critical therapy for advanced chronic myeloid leukemia (CML).
  • Patients with CML in blast phase (CML-BP) or tyrosine kinase inhibitor (TKI)-resistant CML-CP often require intensive treatment options.
  • Allo-HCT offers a potential curative approach for these challenging CML subtypes.

Purpose of the Study:

  • To evaluate the efficacy and outcomes of allo-HCT in patients with CML-BP and TKI-resistant CML-CP.
  • To identify factors influencing survival and relapse rates post-allo-HCT in these CML populations.
  • To assess the impact of disease biology and cytogenetic abnormalities on allo-HCT outcomes.

Main Methods:

  • Retrospective analysis of 75 patients undergoing allo-HCT across four U.S. centers (2005-2023).
  • Categorization of patients into CML-BP (myeloid or lymphoid) and TKI-resistant CML-CP groups.
  • Comparison of overall survival (OS) and leukemia-free survival (LFS) based on disease characteristics and cytogenetics.

Main Results:

  • Median OS was 50 months for myeloid blast phase (MBP), while not reached for lymphoid blast phase (LBP) and TKI-resistant CML-CP.
  • MBP exhibited higher relapse rates and inferior LFS compared to LBP and TKI-resistant CML-CP.
  • Transformed MBP, de novo MBP, and presence of 7/7q- deletions were associated with worse outcomes. Post-transplant TKI maintenance showed no significant impact.

Conclusions:

  • Allo-HCT is a viable treatment for CML-BP and TKI-resistant CML-CP.
  • Disease biology, specifically MBP, and cytogenetic features significantly impact allo-HCT outcomes.
  • Further research may refine patient selection and post-transplant strategies for improved CML management.

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