Eltrombopag-induced Metabolic Acidosis and Hepatic Encephalopathy in Pediatric ITP

Area of Science:

• Pediatric Hematology
• Pharmacovigilance
• Drug Safety

Background:

• Eltrombopag is indicated for chronic immune thrombocytopenia purpura (ITP) in pediatric patients aged 1 year and older with insufficient response to corticosteroids and IVIG.
• Immune thrombocytopenia purpura is an autoimmune disorder characterized by low platelet counts.

Observation:

• A 2-year-old boy with chronic ITP developed acute liver failure, metabolic acidosis, and encephalopathy.
• These severe adverse events occurred despite standard dosing of eltrombopag.
• The patient had a documented insufficient response to prior standard therapies.

Findings:

• This case represents the first reported instance of eltrombopag-induced hepatic encephalopathy in a pediatric patient.
• The adverse effects were life-threatening, necessitating close monitoring and prompt medical intervention.
• Standard drug dosing was associated with severe toxicity in this individual.

Implications:

• Prescribers must exercise significant caution when initiating eltrombopag in the pediatric population.
• Enhanced monitoring for liver function and neurological status is crucial in children receiving eltrombopag.
• This case underscores the importance of pharmacovigilance for identifying rare but severe adverse drug reactions in pediatric patients.