Assessment of endpoints in the pediatric population: congenital heart disease and idiopathic pulmonary arterial hypertension
Assessment of endpoints in the pediatric population: congenital heart disease and idiopathic pulmonary arterial hypertension
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DOI：
10.1097/01.mcp.0000370209.45756.a1
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发表时间：
2010-05-01
影响因子：
3.3
通讯作者：
Beghetti, Maurice
中科院分区：
文献类型：
--
作者：
Haworth, Sheila G.;Beghetti, Maurice
综述目的肺动脉高压(PAH)儿童是一个不同的患者群体，需要不同的疾病管理和治疗方法。在发达国家，儿童的肺动脉高压比成人更常见。它经常与先天性心脏病有关。数量较少的儿童患有迅速进展的特发性PAH。PAH的自然病史和对治疗的反应在儿童和成人中可能不同。例如，在特发性PAH中，婴儿和年幼的儿童可能会出现疲劳和发育障碍，以及晕厥，而青少年可能会出现与成人相似的症状。儿童早期通常需要积极的治疗，因为疾病进展更快。最近的发现到目前为止，还没有发表针对儿科患者的针对肺动脉高压的药物的随机试验。试验方法需要改进，选择最合适的终点。最近的研究表明，使用双重内皮素受体拮抗剂波生坦可以使儿童受益。总结儿童的治疗反应很难量化。在实施和解释成人使用的传统治疗终点方面存在挑战：主要是运动能力，但也包括功能等级和生活质量。有可能在未来的儿科试验中克服这些问题，通过使用一个复合终点，即临床恶化时间，就像在成年PAH人群中一样。复合终点使用诸如症状进展和是否需要额外治疗、住院、移植或死亡等参数。
Purpose of reviewChildren with pulmonary arterial hypertension (PAH) are a distinct patient population, requiring a different approach to disease management and treatment. In developed countries, pulmonary hypertension is more common in children than adults. It is frequently associated with congenital heart disease. A smaller number of children have rapidly progressive idiopathic PAH. The natural history of PAH and the response to treatment can differ in children and adults. In idiopathic PAH, for example, infants and younger children may present with fatigue and impaired growth, as well as syncope, whereas teenagers may present with symptoms similar to those seen in adults. Aggressive therapy is usually required early in young children because the disease can progress more rapidly.Recent findingsThus far, no randomized trial of a pulmonary hypertension-specific drug in pediatric patients has been published. Trial methodology needs refining with selection of the most appropriate endpoints. Recent studies suggest that treatment with the dual endothelin receptor antagonist, bosentan, can benefit children.SummaryTreatment response can be difficult to quantitate in children. There are challenges involved in performing and interpreting the traditional treatment endpoints used in adults: primarily exercise capacity, but also functional class and quality of life. It may be possible to overcome these problems in future pediatric trials by using a composite endpoint, time to clinical worsening, as in the adult PAH population. A composite endpoint uses parameters such as symptomatic progression and the need for additional therapy, hospitalization, transplantation, or death.