Abstract

Purpose
Acute promyelocytic leukemia (APL) is curable, but relapse remains a concern, particularly in patients treated with all-trans retinoic acid (ATRA) and chemotherapy-based regimens. The identification of prognostic factors for relapse is important for enhanced survival outcomes.
Materials and Methods
This retrospective multicenter study analyzed the clinical outcomes and prognostic factors for relapse in 286 Korean patients treated with ATRA and idarubicin-based chemotherapy protocols between 2002 and 2024.
Results
Propensity score-matched analysis revealed key prognostic factors, such as post-consolidation measurable residual disease (MRD) (hazard ratio [HR]: 20.16, p<0.001) and male sex (HR: 5.96; p=0.016). No significant benefit of ATRA-based maintenance therapy was observed in relapse-free survival, compared with observation alone. We also found that FLT3-internal tandem duplication (ITD) mutations were associated with an increased risk of relapse.
Conclusion
These findings highlight prognostic factors for relapse and the importance of individualized therapeutic strategies for high-risk patients. Moreover, our findings indicate that post-consolidation MRD is the most significant predictor of relapse, emphasizing the need for molecular profiling and longitudinal monitoring. Future prospective studies should validate these prognostic markers and refine personalized therapeutic approaches for APL.

• Keywords: Acute promyelocytic leukemia, All-trans retinoic acid, Recurrence, Prognosis, Measurable Residual disease, Real-world data