PDF(Pubmed)
Abstract：
Autosomal recessive polycystic kidney disease (ARPKD) is often associated with hepatobiliary disease in the form of hepatic fibrosis and/or Caroli disease. Combined liver-kidney transplantation (CLKT) is a transplant modality of choice in children with both end-stage renal disease (ESRD) and severe hepatic disease. However, there is no consensus on whether children with ARPKD-associated ESRD without severe hepatic disease can be treated with isolated kidney transplantation (KT) without the need for CLKT. We retrospectively studied the efficacy of isolated KT in children with ARPKD without severe hepatic disease, and followed the course of hepatic disease post KT. This is a single-center study of three children with ARPKD and ESRD who underwent isolated KT. None of them had severe hepatic disease at the time of KT. All children were clinically diagnosed with ARPKD in the immediate postnatal period. All had hepatic fibrosis of varying degrees and two had intrahepatic biliary duct (IHBD) dilatation. None had gastrointestinal (GI) bleed, portal hypertension or cholangitis. Two children had preemptive KT. Pre-transplant unilateral or bilateral native nephrectomy were performed for two children, and one underwent unilateral native nephrectomy at the time of KT. The median creatinine clearance at a median post-KT follow-up of 24 months was 60.3 mL/min/1.73 m2. The two-year graft and patient survival were both 100%. Post KT, all three patients continued to demonstrate evidence of hepatic fibrosis and IHBD on sonogram; however, none of them were either evaluated for or required liver transplantation given normal synthetic liver function and absence of portal hypertension or other severe hepatobiliary disease. There were no adverse events observed such as cholangitis, GI bleed, or multiorgan failure. Hence, an excellent short-term graft and patient survival was demonstrated in this study of children with ARPKD and mild to moderate hepatic disease who received isolated KT. Long-term follow-up and larger studies are important to assess the efficacy of isolated KT in this subset of children with ARPKD.
摘要：
常染色体隐性多囊肾病（ARPKD）通常与肝纤维化和/或Caroli病形式的肝胆疾病有关。肝肾联合移植（CLKT）是患有终末期肾病（ESRD）和严重肝病的儿童的首选移植方式。然而,对于ARPKD相关ESRD无严重肝脏疾病的儿童是否可以在不需要CLKT的情况下接受离体肾移植(KT)治疗,目前尚无共识.我们回顾性研究了孤立性KT在无严重肝病的ARPKD患儿中的疗效。并遵循KT后肝病的病程。这是一项针对三名ARPKD和ESRD儿童的单中心研究，他们接受了孤立的KT。在KT时，他们都没有严重的肝病。所有儿童在出生后立即被临床诊断为ARPKD。所有患者均有不同程度的肝纤维化，其中2例出现肝内胆管（IHBD）扩张。没有人胃肠道（GI）出血，门静脉高压症或胆管炎。两个孩子有先发制人的KT。对两名儿童进行了移植前单侧或双侧天然肾切除术，其中一人在KT时接受了单侧天然肾切除术。KT随访24个月后的肌酐清除率中位数为60.3mL/min/1.73m2。两年的移植物和患者生存率均为100％。KT后,所有三名患者继续在超声图上证明肝纤维化和IHBD的证据;然而，考虑到正常的合成肝功能，没有门脉高压或其他严重的肝胆疾病，没有评估或需要肝移植。没有观察到胆管炎等不良事件，消化道出血,或多器官衰竭。因此，本研究对接受孤立性KT治疗的ARPKD合并轻至中度肝病患儿进行了研究,证明其短期移植物和患者生存率优异.长期随访和更大规模的研究对于评估孤立KT在这一ARPKD儿童亚组中的疗效很重要。
