Medizinische Universität Graz Austria/Österreich - Forschungsportal - Medical University of Graz
Gewählte Publikation:
SHR Neuro Krebs Kardio Lipid Stoffw Microb
Lackner, H; Karastaneva, A; Schwinger, W; Benesch, M; Sovinz, P; Seidel, M; Sperl, D; Lanz, S; Haxhija, E; Reiterer, F; Sorantin, E; Urban, CE.
Sirolimus for the treatment of children with various complicated vascular anomalies.
Eur J Pediatr. 2015; 174(12):1579-1584
Doi: 10.1007/s00431-015-2572-y
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- Führende Autor*innen der Med Uni Graz
- Lackner Herwig
- Sperl Daniela Ingrid
- Co-Autor*innen der Med Uni Graz
- Benesch Martin
- Haxhija Emir
- Karastaneva Anna
- Lanz Sofia
- Reiterer Friedrich
- Ritter-Sovinz Petra
- Schwinger Wolfgang
- Seidel Markus
- Sorantin Erich
- Urban Ernst-Christian
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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. 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. • 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. • 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
- Find related publications in this database (using NLM MeSH Indexing)
- Angiogenesis Inhibitors - adverse effects
- Angiogenesis Inhibitors - therapeutic use
- Female -
- Humans -
- Infant -
- Infant, Newborn -
- Male -
- Retrospective Studies -
- Sirolimus - adverse effects
- Sirolimus - therapeutic use
- Tomography, X-Ray Computed -
- Treatment Outcome -
- Vascular Malformations - drug therapy
- Find related publications in this database (Keywords)
- Sirolimus
- Vascular anomalies
- Children
- Treatment