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Sirolimus for the treatment of children with various complicated vascular anomalies
Herwig Lackner1, Anna Karastaneva2, Wolfgang Schwinger3
1Department of Pediatrics and Adolescent Medicine, Division of Pediatric Hematology/Oncology, Medical University of Graz, Graz, Austria. herwig.lackner@medunigraz.at.
Abstract:
Vascular anomalies include a heterogeneous group of disorders that are categorized as vascular tumors or vascular malformations. Treatment options include resection, embolization, laser therapy, and sclerotherapy or medical treatment such as propranolol, steroids, interferon, and cytostatic chemotherapy. Mammalian target of rapamycin seems to play a key role in the signal pathway of angiogenesis and subsequently in the development of vascular anomalies. Recently, the successful use of sirolimus has been reported in children with lymphatic malformations and kaposiform hemangioendotheliomas. We report on six patients with different vascular anomalies (kaposiform hemangioendothelioma n = 2, combined lymphatico-venous malformation n = 2, pulmonary lymphangiectasia n = 1, and orbital lymphatic malformation n = 1) who were treated with peroral sirolimus. Three of the children initially presented with a Kasabach-Merrit phenomenon. Median duration of treatment was 10 months; two children are still on treatment. Three children each achieved complete and partial remission. Kasabach-Merrit phenomenon resolved within 1 month in all patients. Treatment with sirolimus was tolerated well; only mild reversible leukopenia was observed.
Conclusion:
Sirolimus proved to be effective in children with complicated lymphatic or lymphatico-venous malformations and kaposiform hemangioendotheliomas. Treatment was tolerated well with acceptable side effects. The optimum length of treatment and possible long-term side effects have to be evaluated.
What Is Known:
• Vascular anomalies including vascular tumors and vascular malformations may lead to life-threatening conditions.• Some patients are refractory to established treatment and/or are not available for local invasive procedures.
What Is New:
• We reviewed the literature focusing treatment of vascular anomalies inc hildren and adolescents.• Our data support recent studies that sirolimus is an effective treatment option in patients with complicated vascular tumors andmalformations
Insights
Sirolimus effectively treats complex vascular anomalies in children, including kaposiform hemangioendotheliomas and lymphatic malformations. This treatment was well-tolerated, with rapid resolution of Kasabach-Merritt phenomenon and acceptable side effects.
Area of Science:
- Pediatric vascular medicine
- Angiogenesis signaling pathways
- Pharmacological treatments for vascular disorders
Background:
- Vascular anomalies, encompassing tumors and malformations, can present as life-threatening conditions.
- Established treatments may be ineffective or unsuitable for some pediatric patients.
- The mammalian target of rapamycin (mTOR) pathway is implicated in angiogenesis and vascular anomaly development.
Purpose of the Study:
- To evaluate the efficacy and safety of sirolimus in children with complex vascular anomalies.
- To assess the impact of sirolimus on Kasabach-Merritt phenomenon in affected children.
Main Methods:
- Retrospective case series of six pediatric patients with diverse vascular anomalies treated with oral sirolimus.
- Patients included kaposiform hemangioendothelioma, combined lymphatico-venous malformation, pulmonary lymphangiectasia, and orbital lymphatic malformation.
- Treatment duration, remission status, and adverse events were monitored.
Main Results:
- Sirolimus treatment led to complete or partial remission in all six patients.
- Kasabach-Merritt phenomenon resolved within one month in all affected children.
- Treatment was generally well-tolerated, with mild, reversible leukopenia as the primary side effect.
Conclusions:
- Sirolimus is an effective therapeutic option for complicated pediatric vascular anomalies, particularly lymphatic/lymphatico-venous malformations and kaposiform hemangioendotheliomas.
- The drug demonstrated a favorable safety profile with manageable side effects.
- Further research is needed to determine optimal treatment duration and long-term outcomes.
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