Primary prophylaxis of variceal hemorrhage in children with portal hypertension: a framework for future research.
Primary prophylaxis of variceal hemorrhage in children with portal hypertension: a framework for future research.
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DOI：
10.1097/mpg.0b013e318205993a
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发表时间：
2011-03
影响因子：
2.9
通讯作者：
Shneider BL
中科院分区：
文献类型：
--
作者：
Ling SC;Walters T;McKiernan PJ;Schwarz KB;Garcia-Tsao G;Shneider BL
非选择性β受体阻滞剂治疗和内镜下静脉曲张结扎术可降低成人食管静脉曲张出血的发生率，但其在儿童中的应用存在争议。由于缺乏适当的随机对照试验，没有证据为基础的建议，预防性管理的儿童静脉曲张出血的风险。在最近的美国肝病研究协会年会专家聚会中，在试图设计和实施使用这些疗法的儿童初级预防临床试验时发现了重大挑战。这些挑战使得这样的试验不可行，主要是由于所需的样本量大，对β受体阻滞剂的适当剂量的了解不足，以及难以招募到内镜下静脉曲张结扎试验。儿科研究应侧重于解决静脉曲张的自然史和诊断、静脉曲张出血的预测、β受体阻滞剂和结扎治疗的最佳方法以及探索儿童治疗疗效的替代研究设计等问题。
Nonselective β-blocker therapy and endoscopic variceal ligation reduce the incidence of variceal hemorrhage in cirrhotic adults, but their use in children is controversial. There are no evidence-based recommendations for the prophylactic management of children at risk of variceal hemorrhage due to the lack of appropriate randomized controlled trials. In a recent gathering of experts at the American Association for the Study of Liver Diseases annual meeting, significant challenges were identified in attempting to design and implement a clinical trial of primary prophylaxis in children using either of these therapies. These challenges render such a trial unfeasible, primarily due to the large sample size required, inadequate knowledge of appropriate dosing of β-blockers, and difficulty in recruiting to a trial of endoscopic variceal ligation. Pediatric research should focus on addressing questions of natural history and diagnosis of varices, prediction of variceal bleeding, optimal approaches to β-blocker and ligation therapy, and alternative study designs to explore therapeutic efficacy in children.