Treatment-Free Remission Outcomes in a BCR::ABL1 Digital PCR Selected Clinical Cohort of CML Patients.

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Tác giả: N M A Blijlevens, R Blommers, J J Cornelissen, M Corsten, M J Cruijsen, L G M Daenen, S de Jonge, E de Jongh, M Donker, M F Durian, I Geelen, P Hogenbirk, M Hoogendoorn, J J W M Janssen, K-S G Jie, S Kersting, S K Klein, C Kockerols, H Levenga, M-D Levin, M Mohlmann, W M Smit, P A W Te Boekhorst, P J M Valk, A J Vlot, P E Westerweel, T J Wustman, J J Zwaginga

Ngôn ngữ: eng

Ký hiệu phân loại:

Thông tin xuất bản: England : European journal of haematology , 2025

Mô tả vật lý:

Bộ sưu tập: NCBI

ID: 168394

 Approximately 40%-60% of patients reaching a stable deep molecular response during TKI treatment will maintain a state of remission after TKI discontinuation, denoted as treatment-free remission (TFR). Depth of molecular response assessed by BCR::ABL1 digital PCR prior to TKI discontinuation has demonstrated its significance as a reliable predictive parameter for TFR. A clinically applicable prediction cutoff of 0.0023%IS has been established and externally validated. In this study, BCR::ABL1 digital PCR, as most sensitive and stable assay of its kind, was investigated as a TFR prediction tool in the Netherlands, and evaluated for its predictive value to stop TKI treatment below the aforementioned cutoff. The primary endpoint of molecular recurrence (MolR, BCR::ABL1 >
  0.1%IS) at 12 months was prospectively assessed. Overall, 67 discontinued patients below the set BCR::ABL1 digital PCR cutoff were included. The overall MolR probability was 50% (95% CI, 36%-62%). In 38 patients treated for more than 6 years as commonly recommended as desirable treatment duration before TFR attempt, the MolR probability dropped to 36% (95% CI, 18%-50%). Patients attempting an early TKI discontinuation (treated for less than 6 years) had a high MolR probability of 76% (95% CI, 65%-89%). BCR::ABL1 digital PCR was successfully used in Dutch clinical practice. Our study indicates that in patients with a low BCR::ABL1 digital PCR result, a total TKI treatment duration of six or more years remains associated with a lower MolR rate and should generally be pursued. In patients treated for more than 6 years, BCR::ABL1 digital PCR was capable to identify stop candidates with a higher probability of TFR success.
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