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[Identify the factors associated with treatment-free remission outcomes after imatinib discontinuation in children and adolescent patients with chronic myeloid leukemia].

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PMID41145282
JournalZhonghua xue ye xue za zhi = Zhonghua xueyexue zazhi
Publication Date2025-09-14
Ingested2026-08-02 12:05 AM
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Objective: To identify factors influencing treatment-free remission (TFR) outcomes in children and adolescent patients with chronic myeloid leukemia (CML) after imatinib (IM) discontinuation. Methods: This multicenter retrospective study analyzed 36 children and adolescent patients with CML from eight hematology centers in China (December 1, 2016, to September 27, 2024) who discontinued IM therapy with documented post-cessation outcomes. Clinical characteristics and molecular response dynamics were assessed. Univariate analysis and multivariate Cox proportional hazards regression models were employed to assess factors associated with TFR outcomes. Results: A total of 36 patients were documented, comprising 17 males and 19 females. The median ages at CML diagnosis and IM discontinuation were 11 years (IQR: 5,16) and 20 years (IQR: 14,25), respectively. The median time from IM initiation to first deep molecular response (DMR) was 21 months (IQR: 13, 38). Pre-discontinuation, patients received IM for a median duration of 96 months (IQR: 84, 121) and maintained DMR for 74 months (IQR: 63, 89). With a median post-discontinuation follow-up of 38 months (IQR: 15, 68), cumulative TFR rates at 6, 12, 24, and 36 months were 74.1%, 60.7%, 60.7%, and 56.0%, respectively, generating an overall TFR rate of 58.3%. Fifteen patients lost major molecular response at a median of 5 months post-discontinuation (IQR: 3, 11). All 15 patients resumed tyrosine kinase inhibitor therapy, comprising 13 who restarted IM and 2 who switched to dasatinib. By the last follow-up, 13 (86.7% ) patients regained DMR after a median treatment duration of 5 months (IQR: 3, 17), and no disease progression occurred in any patient. Withdrawal syndrome occurred in 2 (5.6% ) patients. Univariate analysis revealed significantly higher TFR rates in patients with pre-discontinuation IM duration of ≥100 months vs <100 months (82.4% vs 36.8%, P=0.017) and pre-discontinuation DMR duration of ≥72 months vs <72 months (84.2% vs 29.4%, P=0.003). Multivariate Cox analysis identified pre-discontinuation DMR duration as an independent protective factor for TFR (HR=5.419, 95% CI: 1.524-19.272, P=0.009) . Conclusion: DMR duration was identified as an independent protective factor influencing TFR outcomes in children and adolescent patients with CML after IM discontinuation. Patients who maintained DMR for ≥72 months before IM discontinuation demonstrated a significantly higher TFR rate.

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[Identify the factors associated with treatment-free remission outcomes after imatinib discontinuation in children and adolescent patients with chronic myeloid leukemia].

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