Early Identification of Differentiation Syndrome During ATRA and Arsenic Therapy in Pediatric NHR-APL: Insights From a 14-Year Multicenter Cohort.
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BACKGROUND: The combination of all-trans retinoic acid (ATRA) with arsenic trioxide (ATO) has dramatically improved the cure rate of acute promyelocytic leukemia (APL). However, differentiation syndrome (DS) remains a significant cause of early mortality in APL patients. This severe complication typically occurs during induction therapy with ATRA and/or ATO. Early prediction and timely intervention are crucial to reducing DS-related mortality. This study aimed to identify risk factors for DS and develop a predictive model tailored to children with non-high-risk (NHR) APL. METHODS: We conducted a multicenter retrospective analysis of 166 pediatric NHR-APL patients treated between 2011 and 2024. Clinical characteristics were compared between DS and non-DS groups. Logistic regression was used to identify independent predictors, and a nomogram was developed based on these variables. The model was evaluated using the area under the curve (AUC) and decision curve analysis (DCA). RESULTS: Seventeen patients (10.2%) developed DS during induction. Multivariate analysis identified five independent risk factors: female sex, non-chemotherapy group, BCR subtype 3, elevated peripheral promyelocyte proportion (≥ 44%), and platelet count (≥ 20 × 109/L). The predictive nomogram achieved an AUC of 0.89 in the training set and 0.75 in the validation set. DCA demonstrated favorable clinical utility of the model. CONCLUSIONS: We developed and validated the first nomogram for predicting DS in pediatric NHR-APL. This tool enables early risk stratification and may guide clinical decision-making to reduce early mortality.