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Erschienen in: European Journal of Pediatrics 12/2015

01.12.2015 | Original Article

Sirolimus for the treatment of children with various complicated vascular anomalies

verfasst von: Herwig Lackner, Anna Karastaneva, Wolfgang Schwinger, Martin Benesch, Petra Sovinz, Markus Seidel, Daniela Sperl, Sofia Lanz, Emir Haxhija, Friedrich Reiterer, Erich Sorantin, Christian E. Urban

Erschienen in: European Journal of Pediatrics | Ausgabe 12/2015

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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 in children and adolescents.
Our data support recent studies that sirolimus is an effective treatment option in patients with complicated vascular tumors and malformations.
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Metadaten
Titel
Sirolimus for the treatment of children with various complicated vascular anomalies
verfasst von
Herwig Lackner
Anna Karastaneva
Wolfgang Schwinger
Martin Benesch
Petra Sovinz
Markus Seidel
Daniela Sperl
Sofia Lanz
Emir Haxhija
Friedrich Reiterer
Erich Sorantin
Christian E. Urban
Publikationsdatum
01.12.2015
Verlag
Springer Berlin Heidelberg
Erschienen in
European Journal of Pediatrics / Ausgabe 12/2015
Print ISSN: 0340-6199
Elektronische ISSN: 1432-1076
DOI
https://doi.org/10.1007/s00431-015-2572-y

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