Evidence map›Paper›PMID 40826052›Full record

ArticleBMC cancer2025

Leveraging diverse liquid-liquid phase separation patterns to predict the prognosis and immunotherapy of pediatric acute myeloid leukemia.

Min Kong, Yezhen Yang, Zhixiang Wu, Ying Li, Zhijuan Kang, Qin Zhao, Huan Chang, Zuocheng Yang

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Article in BMC cancer, 2025. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Not yet cited in PubMed.

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5 · Who and what money

Authors and funding

8 authors.

Min KongDepartment of Pediatrics, the Third Xiangya Hospital of Central South University, Changsha, 410013, Hunan, China.
Yezhen YangDepartment of Ophthalmology, the Third Xiangya Hospital of Central South University, Changsha, 410013, Hunan, China.
Zhixiang WuDepartment of Pediatrics, the Third Xiangya Hospital of Central South University, Changsha, 410013, Hunan, China.
Ying LiDepartment of Pediatrics, the Third Xiangya Hospital of Central South University, Changsha, 410013, Hunan, China.
Zhijuan KangDepartment of Pediatrics, the Third Xiangya Hospital of Central South University, Changsha, 410013, Hunan, China.
Qin ZhaoDepartment of Pediatrics, the Third Xiangya Hospital of Central South University, Changsha, 410013, Hunan, China.
Huan ChangDepartment of Anesthesiology, the Third Xiangya Hospital of Central South University, Changsha, 410013, Hunan, China.
Zuocheng YangDepartment of Pediatrics, the Third Xiangya Hospital of Central South University, Changsha, 410013, Hunan, China. yang_zcr@126.com.

Funding

Natural Science Foundation of Hunan Province 2022JJ40739
6 · The paper itself

Abstract

backgroundDespite improvements in 5-year survival rates for pediatric acute myeloid leukemia (P-AML) over recent decades, the relapse rate remains high. This challenge is compounded by the absence of reliable prognostic biomarkers, which limits the effectiveness of predictive, preventive, and personalized approaches in P-AML management. Emerging evidence has indicated that the aberrant liquid-liquid phase separation (LLPS) can alter the spatiotemporal coordination of biomolecular condensates, thereby contributing to tumorigenesis and progression. However, the role of LLPS in P-AML remains unclear. MATERIALS AND

methodsA comprehensive multi-omics analysis of LLPS-related genes in P-AML was conducted using both bulk RNA sequencing and single-cell RNA sequencing data. Based on LLPS gene expression profiles, a prognostic risk model was developed through Kaplan-Meier survival analysis, least absolute shrinkage and selection operator (LASSO) regression, stepAIC, and Cox regression analyses. This model aims to predict patient prognosis, immune cell infiltration patterns, responses to immunotherapy, and sensitivity to targeted therapies in P-AML. Additionally, a nomogram integrating the risk model with clinical characteristics was constructed to enhance clinical applicability.

resultsThe LLPS-related risk model was developed as an independent prognostic indicator in the TARGET-AML cohort and validated in the external cohorts. Results demonstrated that patients in the low-risk group had significantly better overall survival than those in the high-risk group. In addition, a nomogram was constructed to enhance the clinical applicability of the risk model. Integrated analysis of bulk and single-cell transcriptome data revealed that the LLPS-related signature correlated with cancer hallmarks, immune checkpoint genes, and key components of the tumor microenvironment. Distinct drug sensitivities were observed between risk groups, with P-AML patients exhibiting varied responses to Docetaxel, Paclitaxel, and Sunitinib.

conclusionIn summary, we developed a robust and effective risk model for predicting prognosis, tumor microenvironment characteristics, and responses to immunotherapy and targeted therapies in P-AML. This model offers valuable insights for advancing personalized and precision medicine approaches in P-AML treatment strategies.

Indexed as

Biomarkers, TumorImmunotherapyLeukemia, Myeloid, AcuteAdolescentChildChild, PreschoolFemaleGene Expression ProfilingHumansInfantKaplan-Meier EstimateMaleNomogramsPhase SeparationPrognosisSingle-Cell AnalysisBiomarkers, TumorImmune infiltrationImmunotherapy efficacyLiquid-liquid phase separationPediatric acute myeloid leukemiaPrognosis

Identifiers

PMID40826052
PMCPMC12359890

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