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<p>Tyrosine kinase inhibitors (TKIs) have markedly improved survival in chronic myeloid leukemia (CML), but resistance, intolerance, and treatment sequencing remain challenging in real-world practice. This study evaluated treatment patterns, molecular responses, and long-term overall survival (OS) in patients with CML receiving sequential TKI therapy and aimed to identify prognostic factors influencing OS. This retrospective, single-center study included 260 adults with CML treated with TKIs between 2008 and 2024. OS was analyzed using the Kaplan–Meier method, and prognostic factors were identified using Cox regression. Mean age was 54.0 ± 11.6 years, and 52.7% were female; 94.2% were in chronic phase at diagnosis, with a median baseline BCR-ABL1 of 38.1% (international scale). During follow-up, 58.1% required second-line and 20.0% third-line therapy. Major and deep molecular responses were achieved in 73.1% and 40.8%, respectively. Five-year OS was 97.0%, 95.9%, and 92.0% for 1, 2, and ≥ 3 treatment lines (p = 0.331). A high Sokal risk score (adjusted hazard ratio [aHR] 6.96, p &lt; 0.001) and a 10% increase in baseline BCR-ABL1 (aHR 1.29, p = 0.002) were associated with mortality. Landmark analysis at 12 months showed no significant survival difference between single and multiple lines. Sequential TKI therapy was effective and safe, with five-year OS exceeding 90% across all treatment lines, even in heavily pretreated patients. High Sokal risk score and baseline BCR-ABL1 (a surrogate of initial leukemic burden rather than a validated ELN prognostic marker) emerged as <i>potential</i> independent predictors of mortality, underscoring the need for early risk-adapted management.</p>

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Long-Term Survival Outcomes and Influencing Prognostic Factors in Chronic Myeloid Leukemia Patients Receiving Sequential Tyrosine Kinase Inhibitor Therapy: A Retrospective Analysis of Real-World Data

  • 期刊论文

  • 发表日期：

    2026年7月24日

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  • Hakan Keski,
  • Selim Merdan

摘要

Tyrosine kinase inhibitors (TKIs) have markedly improved survival in chronic myeloid leukemia (CML), but resistance, intolerance, and treatment sequencing remain challenging in real-world practice. This study evaluated treatment patterns, molecular responses, and long-term overall survival (OS) in patients with CML receiving sequential TKI therapy and aimed to identify prognostic factors influencing OS. This retrospective, single-center study included 260 adults with CML treated with TKIs between 2008 and 2024. OS was analyzed using the Kaplan–Meier method, and prognostic factors were identified using Cox regression. Mean age was 54.0 ± 11.6 years, and 52.7% were female; 94.2% were in chronic phase at diagnosis, with a median baseline BCR-ABL1 of 38.1% (international scale). During follow-up, 58.1% required second-line and 20.0% third-line therapy. Major and deep molecular responses were achieved in 73.1% and 40.8%, respectively. Five-year OS was 97.0%, 95.9%, and 92.0% for 1, 2, and ≥ 3 treatment lines (p = 0.331). A high Sokal risk score (adjusted hazard ratio [aHR] 6.96, p < 0.001) and a 10% increase in baseline BCR-ABL1 (aHR 1.29, p = 0.002) were associated with mortality. Landmark analysis at 12 months showed no significant survival difference between single and multiple lines. Sequential TKI therapy was effective and safe, with five-year OS exceeding 90% across all treatment lines, even in heavily pretreated patients. High Sokal risk score and baseline BCR-ABL1 (a surrogate of initial leukemic burden rather than a validated ELN prognostic marker) emerged as potential independent predictors of mortality, underscoring the need for early risk-adapted management.