Paediatric Liver Centre, Kings College Hospital, SE5 9RS London, UK.
J Pediatr Surg. 2011 Sep;46(9):1689-94. doi: 10.1016/j.jpedsurg.2011.04.013.
Biliary atresia (BA) is a rare, potentially life-threatening condition of the newborn presenting with conjugated jaundice. Typically, it is treated by an initial attempt to restore bile flow (the Kasai portoenterostomy [KP]) as soon as possible after diagnosis and, if this fails, liver transplantation. Since 1999, the treatment of BA has been centralized to 3 centers in England and Wales able to offer both treatment options. The aim of this study was to review the outcome of this policy change and provide a national benchmark.
The management of all infants born within England and Wales during the period January 1999 to December 2009 was assessed using 3 key performance indicators such as median time to KP, percentage clearance of jaundice (≤20 mol/L) post-KP, and 5- and 10-year native liver and true survival estimates. Data are quoted as median (range), and P < .05 was considered significant.
A total of 443 infants had confirmed BA; and of these, most were isolated BA (n = 359), with 84 having other significant anomalies (but predominantly BA splenic malformation syndrome). Four infants died before any biliary intervention. Kasai portoenterostomy was performed in 424 infants (median age, 54 [range 7-209] days), and a primary liver transplant was performed in 15. Clearance of jaundice post-KP was achieved in 232 (55%). There were 41 deaths, including 4 (10%) without any intervention, 24 (58%) post-KP usually because of end-stage liver disease and mostly on a transplant waiting list, and 13 (32%) post-LT usually because of multiorgan failure. Overall, the 5- and 10-year native liver survival estimates were 46% (95% confidence interval [CI], 41-51) and 40% (95% CI, 34-46), respectively. The 5- and 10-year true patient survival estimates were 90% (95% CI, 88-93) and 89% (95% CI, 86-93), respectively. Outcome was worse for those with other anomalies (lower clearance of jaundice post-KP [43% vs 57%; odds ratio, 1.7; 95% CI, 1.04-2.8]; P = .02) and an increased mortality overall (eg, at 5 years, 72 [95% CI, 64-83] vs 94 [95% CI, 91-96]; χ(2) = 33; P < .0001).
National outcome measures in BA appear better than those from previously published series from comparable countries and may be attributed to centralization of surgical and medical resources.
胆道闭锁（BA）是一种罕见的、潜在危及生命的新生儿疾病，表现为结合性黄疸。通常，在诊断后尽快进行最初的胆汁流恢复尝试（Kasai 门腔分流术[KP]），如果失败则进行肝移植。自 1999 年以来，英国和威尔士的 3 个中心已经能够提供这两种治疗方案，BA 的治疗已经集中在这 3 个中心。本研究的目的是回顾这一政策变化的结果，并提供一个国家基准。
使用 3 个关键绩效指标评估 1999 年 1 月至 2009 年 12 月期间在英格兰和威尔士出生的所有婴儿的管理情况，如 KP 的中位时间、KP 后黄疸清除率（≤20 mol/L）、5 年和 10 年的肝固有和真实存活率。数据以中位数（范围）表示，P<.05 被认为具有统计学意义。
共 443 名婴儿被确诊为 BA；其中大多数为孤立性 BA（n=359），84 名婴儿有其他显著异常（但主要为 BA 脾畸形综合征）。有 4 名婴儿在任何胆道干预之前死亡。424 名婴儿接受了 KP（中位年龄为 54[范围 7-209]天），15 名婴儿接受了原发性肝移植。232 名婴儿（55%）的黄疸清除率达到 KP 后。有 41 例死亡，包括 4 例（10%）未接受任何干预，24 例（58%）KP 后通常是因为终末期肝病，主要在移植等待名单上，13 例（32%）LT 后通常是因为多器官衰竭。总的来说，5 年和 10 年的肝固有存活率估计分别为 46%（95%置信区间[CI]，41-51）和 40%（95% CI，34-46）。5 年和 10 年的真实患者存活率估计分别为 90%（95% CI，88-93）和 89%（95% CI，86-93）。有其他异常的患者（KP 后黄疸清除率较低[43% vs 57%；优势比，1.7；95% CI，1.04-2.8]；P=.02）和总体死亡率更高（例如，5 岁时，72 例[95% CI，64-83] vs 94 例[95% CI，91-96]；χ(2)=33；P<.0001）。
BA 的国家结果指标似乎优于来自可比国家的先前发表系列的结果指标，这可能归因于手术和医疗资源的集中化。
