What does this research mean for the field?

A prognostic score can successfully predict treatment-free remission probabilities in chronic myeloid leukaemia patients after discontinuation of tyrosine kinase inhibitors. Novelty: ClaimNovelty.NOVEL_FINDING. Consensus alignment: ConsensusAlignment.NEUTRAL.

What question did this study set out to answer?

The study aims to identify predictors of successful treatment-free remission in chronic myeloid leukaemia patients.

March 12, 2026Open Access

Introducing a prognostic score for successful treatment‐free remission in chronic myeloid leukaemia

Key Points

The study aims to identify predictors of successful treatment-free remission in chronic myeloid leukaemia patients.
Conducted a single-centre cohort study involving 197 patients with chronic myeloid leukaemia.
Patients had deep molecular response for over a year and had been on therapy for at least three years.
Developed a prognostic score based on multivariable analysis of various patient characteristics.
Approximately 50% of patients lost deep molecular response within two years after discontinuation of therapy.
Key factors associated with higher treatment-free remission probability included male sex and a faster achievement of deep molecular response.
The prognostic score distinguished between three groups with significantly different probabilities of treatment-free remission.

Abstract

The modern management of chronic myeloid leukaemia (CML) identifies a new therapeutic goal of treatment-free remission (TFR). Half of CML patients in durable deep molecular response (DMR) (MR4 or better) can remain off tyrosine kinase inhibitors (TKIs) without experiencing loss of major molecular response. Despite a large number of TFR studies to date, there are no consistent predictors of successful TFR. We conducted a single-centre cohort study on 197 patients discontinuing TKIs in DMR ≥1 year and TKI therapy ≥3 years. After TKI discontinuation, 98 patients (49.7%) lost MR4; of these, 90 (91.8%; and 45.7% of the whole cohort) lost major molecular response (MMR or MR3) after a median of 3.8 months (1-93.3). The 2-year probability of TFR (pTFR) was 57.7%. In multivariable analysis, male sex, age at diagnosis >40 years, faster achievement of MR4 and longer duration of DMR were the only variables significantly associated with higher pTFR. Based on the multivariable analysis results, we built a TFR prognostic score (TPS) able to distinguish three groups with different 2-year pTFR: good (89.9%), intermediate (61.2%) and poor (18.4%) TFR probability (p < 0.0001). We validated the TPS on an independent cohort of 91 patients. We propose that the TPS could become a useful guide for CML clinicians.

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