Ospina Fabio E, Echeverri Alex, Posso-Osorio Iván, Jaimes Lina, Gutierrez Jaiber, Tobón Gabriel J
Instituto de Investigaciones Clínicas, Fundación Valle del Lili, Cali, Colombia.
Grupo de Investigación en Reumatología, Autoinmunidad y Medicina Traslacional (GIRAT) Departamento de Reumatologia, Fundación Valle del Lili, Cali, Colombia.
Colomb Med (Cali). 2017 Jun 30;48(2):88-93.
Five-year-old female patient with hereditary hemorrhagic telangiectasia.
Deterioration of cardiopulmonary function with higher oxygen requirements secondary to pulmonary arteriovenous shunts, epistaxis.
The patient was treated with the monoclonal antibody bevacizumab, which inhibits the vascular endothelial growth factor, with good clinical outcome.
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.
一名患有遗传性出血性毛细血管扩张症的5岁女性患者。
继发于肺动静脉分流，心肺功能恶化，需氧量增加，鼻出血。
患者接受了抑制血管内皮生长因子的单克隆抗体贝伐单抗治疗，临床效果良好。
遗传性出血性毛细血管扩张症是一种常染色体显性疾病，其特征为不同器官的动静脉畸形，临床表现多样。针对这种全身性疾病的系统治疗选择有限。单克隆抗体贝伐单抗似乎是治疗该疾病的一个良好选择。虽然在成人中使用已报道成功，但在儿科人群中的使用尚未见报道。在此，我们报告了贝伐单抗在一名患有遗传性出血性毛细血管扩张症的5岁女性患者中的应用，显示出临床益处和良好结果。
