Publication:

Hepatoprotective efficacy of ursodeoxycholic acid in pediatric acute lymphoblastic leukemia

Background Acute lymphoblastic leukemia (ALL) is the most prevalent pediatric malignancy, with an 80–85% survival rate. Despite advances, treatment-related hepatotoxicity remains a concern due to its impact on morbidity and chemotherapy adherence. Ursodeoxycholic acid (UDCA) has been proposed for its hepatoprotective potential during chemotherapy in pediatric ALL patients. This study evaluated UDCA's efficacy in preventing hepatotoxicity in children with ALL during the consolidation and interim maintenance chemotherapy phases.

Methods A randomized controlled trial was conducted from September 2018 to August 2019 at BSMMU on 50 children (aged 1–18 years) with newly diagnosed ALL undergoing chemotherapy per the UK ALL 2003 protocol. Participants were randomly assigned to receive either UDCA plus chemotherapy (case group) or chemotherapy alone (control group). Hepatic function tests (ALT, AST, bilirubin) were monitored biweekly for three months, with hepatotoxicity defined as transaminase levels exceeding three times the upper normal limit.

Results Fifty patients participated (25 in each group). Hepatotoxicity occurred in 32% of the case group versus 60% of the control group (p=0.040). Mean ALT and AST levels were significantly lower in the UDCA group compared to controls (p=0.004 and 0.001, respectively), particularly during the third to fifth follow-ups. Only one patient (4%) in the UDCA group required dose adjustments, compared to 40% in the control group.

Conclusion UDCA co-administration reduced hepatic enzyme elevations and minimized chemotherapy interruptions, demonstrating hepatoprotective effects. Larger studies with longer follow-ups are needed to validate its safety and efficacy.