Abstract

Study design: A 2018-2023 prospective cohort study assessed sirolimus safety and efficacy for non-surgically treating complex venolymphatic malformations.
Purpose: Sirolimus's role was evaluated when surgery was high-risk or prior therapies failed.
Methods: Twenty-one patients received oral sirolimus and trimethoprim-sulfamethoxazole based on body weight. Primary outcome measures were lesion resolution, proptosis reduction and vision improvement. Secondary outcome measures were side effects, and recurrence.
Results: The mean age of presentation was 12.3 years. At a mean follow-up of 12.6 months, proptosis improved in 92% of cases, vision improved in 77% of cases, ptosis resolved in 92% of cases, and complete resolution of the lesion occurred in 85% of cases. One case had a recurrence. There were no adverse systemic side effects.
Conclusion: Sirolimus offers a safe, effective option for complex venolymphatic malformations, especially when surgery carries high risk.

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