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◆ Frontiers in medicine2026-01-01

Treatment response and survival outcomes with BCR-ABL tyrosine kinase inhibitors in chronic myeloid leukemia: a retrospective study.

Yufen Liu, Liang Zhang, Lu Zeng

一句话结论 · In one sentence

Excellent molecular responses and favourable survival outcomes were observed among real-world patients with CML treated with BCR-ABL tyrosine kinase inhibitors. Molecular response remained an important prognostic indicator, supporting continued molecular monitoring and risk-adapted treatment. However, the available follow-up was insufficient to evaluate late-onset toxicities and longer-term treatment outcomes fully; therefore, extended follow-up studies with detailed molecular and genetic characterisation are warranted.

原始摘要(英文原文)· Original abstract
BACKGROUND: Chronic myeloid leukemia (CML) is a highly controllable cancer after the introduction of BCR-ABL tyrosine kinase inhibitors (TKIs). However, in real-world settings, there is limited evidence on treatment response, resistance, and survival. OBJECTIVE: To measure treatment response, overall survival, and predictors of molecular response in patients with CML treated with BCR-ABL TKIs in routine clinical practice. METHODOLOGY: This was a retrospective cohort study consisting of 650 adult patients with confirmed CML who received at least one BCR-ABL TKI between January 2023 and December 2025. All demographic, clinical, treatment, and follow-up data were abstracted from the institution's medical records. Assessment was performed for hematologic, cytogenetic, and molecular responses according to standard criteria. Overall survival (OS) and progression-free survival (PFS) were assessed using Kaplan-Meier analysis, and predictors of response and survival were identified using multivariable regression models. RESULTS: The mean age was 49.8 ± 14.2 years, and 57.8% were male. The majority of patients were seen in the chronic phase (91.1%). Complete hematologic response, complete cytogenetic response, major molecular response (MMR) and deep molecular response (DMR) were observed in 93.4, 83.4, 76.3 and 48.9% of patients, respectively. The second-generation TKIs had a significantly higher MMR rate than imatinib (82.9% vs. 71.8%, p < 0.001). 17.4% of patients were treatment-resistant, and 14.9% were treatment-intolerant. Three-year OS and PFS were 95.8 and 93.4%. MMR was independently predicted by younger age, chronic-phase disease, lower ELTS risk and second-generation TKIs. CONCLUSION: Excellent molecular responses and favourable survival outcomes were observed among real-world patients with CML treated with BCR-ABL tyrosine kinase inhibitors. Molecular response remained an important prognostic indicator, supporting continued molecular monitoring and risk-adapted treatment. However, the available follow-up was insufficient to evaluate late-onset toxicities and longer-term treatment outcomes fully; therefore, extended follow-up studies with detailed molecular and genetic characterisation are warranted.
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Treatment response and survival outcomes with BCR-ABL tyrosine kinase inhibitors in chronic myeloid leukemia: a retrospective study. — 科研速览 Science Skim