Everolimus-associated perianal ulcers in an eight-month-old heart transplant recipient

Area of Science:

• Pediatric Gastroenterology
• Pharmacology
• Immunology

Background:

• Mammalian target of rapamycin (mTOR) inhibitors are widely used in pediatric patients for various conditions.
• While oral ulcers are a known side effect, other gastrointestinal manifestations of mTOR inhibitor therapy in children are not well-documented.

Observation:

• An eight-month-old infant developed progressive perianal ulcers three months post-heart transplantation.
• The infant was receiving everolimus, a commonly used mTOR inhibitor.

Findings:

• Diagnostic investigations ruled out other causes for the perianal ulceration.
• The perianal ulcers were attributed to everolimus therapy, leading to drug discontinuation.

Implications:

• This case expands the known spectrum of gastrointestinal adverse effects associated with mTOR inhibitors in pediatric populations.
• Early recognition and management of such side effects are crucial for optimizing treatment outcomes in children receiving mTOR inhibitors.