The Role of Sirolimus in the Treatment of Lymphatic Malformations | ||||
| The Egyptian Journal of Hospital Medicine | ||||
| Article 118, Volume 100, Issue 1, July 2025, Page 3370-3377 PDF (407.87 K) | ||||
| Document Type: Original Article | ||||
| DOI: 10.21608/ejhm.2025.444603 | ||||
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| Abstract | ||||
| Background: Lymphatic malformations (LMs) are complex vascular anomalies that pose significant therapeutic challenges due to their infiltrative nature and potential for serious complications. Sirolimus, a macrolide with antiproliferative and immunosuppressive properties, has emerged as a promising pharmacologic treatment. Objective: To assess the effect of sirolimus in patients with lymphatic malformations in terms of the improvement of symptoms and QOL and the reduction of the size of the lesion. Patients and methods: This prospective interventional study evaluated the efficacy and safety of sirolimus in 16 pediatric patients (aged 0.5–15.8 years) with microcystic, mixed, or lymphaticovenous malformations. Patients received oral sirolimus at a dose of 0.05–0.07 mg/kg twice daily, with serum levels maintained between 4–12 ng/mL. Clinical response was assessed through symptom improvement, quality of life (QOL) evaluation, and radiological follow-up with MRI at 3, 6, and 9 months. Results: The majority of patients demonstrated significant clinical improvement, with 66.7% showing improvement in disfigurement and pain, 100% improvement in fluid leakage, ulceration, and bleeding, and notable reductions in lesion size on MRI. QOL improved significantly in 50% of patients and partially in 37.5%. Adverse events were generally mild and manageable, including pneumonia (12.5%), glossitis (6.25%), hypercholesterolemia (18.75%), neutropenia (12.5%), and elevated liver enzymes (12.5%). Conclusion: These findings support the use of sirolimus as a safe and effective treatment option for pediatric patients with difficult-to-treat lymphatic malformations. | ||||
| Keywords | ||||
| Sirolimus; Vascular anomalies; Vascular malformations; Lymphatic malformations | ||||
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