# Risk factors and management strategies in acute promyelocytic leukemia: a real-world single-center retrospective study

## TL;DR

This study identifies risk factors for early death in acute promyelocytic leukemia patients and suggests strategies to improve outcomes through timely diagnosis and targeted care.

## Contribution

The study identifies novel independent risk factors for early death in APL patients and emphasizes the need for risk-adapted therapy and CNS prophylaxis.

## Key findings

- High WBC count, prolonged PT, and CD2 positivity are independent predictors of early death in APL.
- Severe thrombocytopenia was not an independent risk factor due to aggressive platelet transfusion policies.
- Relapses in high-risk patients predominantly involved the central nervous system.

## Abstract

Acute promyelocytic leukemia (APL) has become a highly curable malignancy with the advent of targeted therapies. However, early death (ED), predominantly caused by severe hemorrhagic complications, continues to be the most significant obstacle to achieving high survival rates in real-world clinical practice, where outcomes often lag behind those reported in controlled trials.

This retrospective study aimed to identify the key clinical and laboratory factors associated with ED and overall survival (OS) in a cohort of newly diagnosed APL patients. The study also sought to evaluate the impact of current supportive care strategies and identify opportunities for improved management.

This single-center, retrospective analysis included 132 patients newly diagnosed with APL between June 2017 and January 2023. Data on demographics, baseline laboratory values, immunophenotyping, treatment, and outcomes were collected. Univariate and multivariate logistic regression analyses were used to identify independent predictors of ED, while Kaplan-Meier and Cox regression models were used to assess survival outcomes.

The ED rate was 12.88% (17 patients), with hemorrhage being the primary cause. Multivariate analysis identified three independent risk factors for ED: a high white blood cell (WBC) count (>201 × 109/L), prolonged prothrombin time (PT), and CD2 positivity. While severe thrombocytopenia was common, it was not an independent predictor, likely due to an aggressive platelet transfusion policy. Elevated lactate dehydrogenase (LDH) levels were significantly associated with poorer OS. Furthermore, all relapses in the high-risk group involved the central nervous system (CNS).

This study found that high initial WBC count, prolonged PT, and CD2 positivity are critical independent predictors of ED in APL. The findings underscore the necessity of prompt diagnosis, risk-adapted therapy, and comprehensive multidisciplinary care, including aggressive management of coagulopathy. Enhanced CNS prophylaxis should be considered for high-risk patients to prevent relapse.