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Research Article: Liver involvement in pediatric acute leukemia: subtype differences and complex hepatic injury as a novel marker for AML risk stratification

Date Published: 2026-05-07

Abstract:
This study aimed to investigate the prevalence, pattern of liver involvement at diagnosis of pediatric acute leukemia, and its associations with clinical features and prognosis. From January 2016 to December 2017, 304 consecutive children newly diagnosed with acute leukemia (190 pre-B acute lymphoblastic leukemia (ALL), 25 T-ALL, and 89 acute myeloid leukemia (AML)), were enrolled. Liver involvement was defined as hepatomegaly (HM), hepatocellular injury (HI), hepatic dysfunction (HD) and cholestasis. The prevalence of liver involvement varied significantly across leukemia subtypes: T-ALL (80.0%), pre-B-ALL (64.0%), and AML (37.0%) (P<0.001). HM was the most common isolated manifestation in all subtypes. Lower platelet counts were significantly associated with HM and HI in pre-B-ALL, and with HD in T-ALL (all P < 0.05). HI was positively correlated with age in pre-B-ALL (median age: 9.0 vs. 4.0 years, P < 0.001), while HM was associated with higher white blood cell (WBC) counts in T-ALL (228.0 vs. 10×10 9 /L, P = 0.035). HD showed a trend of association with elevated WBC counts in AML (P = 0.054). Liver involvement was not associated with early treatment response or long-term survival in ALL. In AML, however, complex hepatic injury and WBC count emerged as independent adverse prognostic factors: AML patients without liver involvement had the best 5-year overall survival (OS: 87.5 ± 4.4%) and event-free survival (EFS: 80.4 ± 5.3%), whereas those with complex hepatic injury had the poorest outcomes (5-year OS: 58.3 ± 14.2%, EFS:50.0 ± 14.4%). Liver involvement exhibits subtype-specific patterns in pediatric acute leukemia and is associated with age, WBC and platelet counts. Notably, complex hepatic injury may serve as a potential adverse prognostic marker for pediatric AML.

Introduction:
Acute leukemia, consisting of lymphoid (80%) and myeloid (20%) variants, is one of the most common malignancies in children, accounting for approximately 40% of all pediatric malignancies ( 1 ). While originating from bone marrow, extramedullary infiltration (especially of the liver) is a key feature of the disease ( 2 ). Leukemic liver involvement presents as hepatomegaly (HM), hepatocellular injury (HI), hepatic dysfunction (HD), or cholestasis, with asymptomatic HM being the most prevalent ( 3 – 6 ). As the…

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