Bevacizumab as a treatment for hereditary hemorrhagic telangiectasia in children: a case report
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Published on: October 27, 2014
Fabio E Ospina1,2, Alex Echeverri2,3, Iván Posso-Osorio1,2
1Instituto de Investigaciones Clínicas, Fundación Valle del Lili, Cali, Colombia.
Insights
Hereditary hemorrhagic telangiectasia (HHT) in a child improved with bevacizumab. This treatment, targeting vascular endothelial growth factor, offers a promising option for HHT patients when other therapies are limited.
Area of Science:
- Vascular biology
- Genetics
- Pediatric medicine
Background:
- Hereditary hemorrhagic telangiectasia (HHT) is an autosomal dominant disorder.
- HHT is characterized by arteriovenous malformations affecting multiple organs.
- Systemic treatment options for generalized HHT are limited.
Observation:
- A 5-year-old female patient presented with HHT.
- Cardiopulmonary function deteriorated due to pulmonary arteriovenous shunts and epistaxis.
- The patient required increased oxygen support.
Findings:
- Treatment with bevacizumab, a monoclonal antibody inhibiting vascular endothelial growth factor (VEGF), was administered.
- The patient experienced a good clinical outcome following bevacizumab therapy.
Implications:
- Bevacizumab shows potential as a systemic therapy for HHT.
- This case suggests bevacizumab is effective in the pediatric population with HHT.
- Further research into bevacizumab for pediatric HHT is warranted.
Case Description:
Five-year-old female patient with hereditary hemorrhagic telangiectasia.
Clinical Findings:
Deterioration of cardiopulmonary function with higher oxygen requirements secondary to pulmonary arteriovenous shunts, epistaxis.
Treatment And Outcome:
The patient was treated with the monoclonal antibody bevacizumab, which inhibits the vascular endothelial growth factor, with good clinical outcome.
Clinical Relevance:
Hereditary hemorrhagic telangiectasia is an autosomal dominant disorder characterized by arteriovenous malformations in different organs, making its clinical presentations varied. Systemic therapeutic options for a generalized disease are limited. The monoclonal antibody bevacizumab, seems to be a good option in this disorder. Although reported as successful in adult population, its use in pediatric population has not yet been reported. Here we report the use of bevacizumab in a 5-year-old female patient with hereditary hemorrhagic telangiectasia, showing clinical benefits and good outcome.
