Leonhardt J, Kuebler J F, Leute P J, Turowski C, Becker T, Pfister E-D, Ure B, Petersen C
St. Bernward Hospital, Pediatric Surgery, Hildesheim, Germany.
Eur J Pediatr Surg. 2011 Mar;21(2):82-7. doi: 10.1055/s-0030-1268476. Epub 2010 Dec 14.
Aim of the study was to carry out a 5-year survey of German patients with biliary atresia (BA) and to launch a discussion regarding the feasibility of voluntary registries in unregulated healthcare systems.
A retrospective analysis of German BA patients born between 2001 and 2005, based on data collected from the voluntary European Biliary Atresia Registry (EBAR), was carried out and supplemented by data from all BA patients who underwent liver transplantation at the only 4 pediatric transplantation centers (pLTx) in Germany which are so far not registered at EBAR. Survival rates were calculated using Kaplan-Meier analysis and compared by Cox regression to determine the predictive value of age at surgery and the influence of the center size (fewer or more than 5 patients/study period) on overall survival and survival with native liver.
A critical review of the 148 German EBAR charts revealed that 11 patients (7.4%) had no biliary atresia. The remaining 137 patients from EBAR together with 46 BA patients who underwent LTx without prior registration at EBAR were evaluated with a median follow-up of 39 months (range: 25-85 months). 29 hospitals performed a total of 159 Kasai procedures, but only 7 centers treated 5 or more patients (116 patients, range: 5-68), and 22 hospitals performed less than 5 KP (43 patients, range: 1-4). Primary LTx was performed in 21 patients (11.5%) and 3 patients died without surgical intervention. 16 patients were lost to follow-up (8.7%). Overall survival after 2 years was 83.3% (139 patients), including 105 patients (63%) who had undergone LTx and 34 patients (20.3%) with native liver. 28 patients died (16.7%), 8 after LTx (5.8%). The experience of the center was the only factor with a significant predictive value for jaundice-free survival with native liver (p=0.001).
25% of all German BA patients were not registered at EBAR, and 29 clinics were involved in the surgical management of BA patients. Therefore a new approach consisting of an internet-based decentralized registry for rare neonatal liver diseases is outlined which could improve the future management of patients with BA. The centralization of such patients at experienced centers with higher caseloads is necessary in Germany and would improve the outcome of patients with biliary atresia.
本研究的目的是对德国胆道闭锁（BA）患者进行为期5年的调查，并就无监管医疗系统中自愿登记的可行性展开讨论。
基于从自愿性欧洲胆道闭锁登记处（EBAR）收集的数据，对2001年至2005年出生的德国BA患者进行回顾性分析，并补充了德国仅有的4家儿科移植中心（pLTx）中所有接受肝移植但尚未在EBAR登记的BA患者的数据。使用Kaplan-Meier分析计算生存率，并通过Cox回归进行比较，以确定手术年龄的预测价值以及中心规模（研究期间少于或多于5例患者）对总体生存和自体肝生存的影响。
对148份德国EBAR图表进行严格审查后发现，11例患者（7.4%）并非胆道闭锁。其余137例来自EBAR的患者以及46例在EBAR未预先登记就接受肝移植的BA患者接受了评估，中位随访时间为39个月（范围：25 - 85个月）。29家医院共进行了159例Kasai手术，但只有7个中心治疗了5例或更多患者（116例患者，范围：5 - 68例），22家医院进行的Kasai手术少于5例（43例患者，范围：1 - 4例）。21例患者（11.5%）接受了初次肝移植，3例患者未接受手术干预即死亡。16例患者失访（8.7%）。2年后的总体生存率为83.3%（139例患者），其中包括105例接受肝移植的患者（63%）和34例自体肝存活的患者（20.3%）。28例患者死亡（16.7%），8例在肝移植后死亡（5.8%）。中心经验是自体肝无黄疸生存的唯一具有显著预测价值的因素（p = 0.001）。
德国所有BA患者中有25%未在EBAR登记，29家诊所参与了BA患者的手术管理。因此，概述了一种新方法，即建立一个基于互联网的罕见新生儿肝病分散式登记处，这可能会改善未来BA患者的管理。在德国，将此类患者集中到病例量较高的经验丰富的中心是必要的，这将改善胆道闭锁患者的治疗结果。
