Hinton Robert B
Division of Cardiology, Cincinnati Children's Hospital Medical Center, Cincinnati, Ohio, USA.
Curr Opin Pediatr. 2014 Oct;26(5):546-52. doi: 10.1097/MOP.0000000000000137.
Aortic valve disease (AVD) is a growing public health problem, and the pathogenesis underlying AVD is complex. The lack of durable bioprostheses and pharmacologic therapies remain central needs in care. The purpose of this review is to highlight recent clinical studies that impact the care of children with AVD and is to explore ongoing translational research efforts.
Clinical studies have evaluated the durability of bioprosthetics and surgical strategies, tested statins during early disease, and identified new predictive biomarkers. Large animal models have demonstrated the effectiveness of a novel bioprosthetic scaffold. Mouse models of latent AVD have advanced our ability to elucidate natural history and perform preclinical studies that test new treatments in the context of early disease.
Current priorities for AVD patients include identifying new pharmacologic treatments and developing durable bioprostheses. Multidisciplinary efforts are needed that bridge pediatric and adult programs, and bring together different types of expertise and leverage network and consortium resources. As our understanding of the underlying complex genetics is better defined, companion diagnostics may transform future clinical trials and ultimately improve the care of patients with AVD by promoting personalized medicine and early intervention.
主动脉瓣疾病(AVD)是一个日益严重的公共卫生问题,其发病机制复杂。缺乏耐用的生物假体和药物治疗仍是治疗中的核心需求。本综述的目的是强调近期影响AVD患儿治疗的临床研究,并探讨正在进行的转化研究工作。
临床研究评估了生物假体的耐用性和手术策略,在疾病早期对他汀类药物进行了试验,并确定了新的预测生物标志物。大型动物模型已证明一种新型生物假体支架的有效性。潜伏性AVD的小鼠模型提高了我们阐明自然史以及在疾病早期背景下进行测试新治疗方法的临床前研究的能力。
AVD患者当前的首要任务包括确定新的药物治疗方法和开发耐用的生物假体。需要多学科努力,连接儿科和成人项目,汇集不同类型的专业知识,并利用网络和联盟资源。随着我们对潜在复杂遗传学的理解更加明确,伴随诊断可能会改变未来的临床试验,并最终通过促进个性化医疗和早期干预来改善AVD患者的治疗。