Doshi Sahil D, Bittermann Therese, Schiano Thomas D, Goldberg David Seth
1 Perelman School of Medicine, University of Pennsylvania, Philadelphia, PA. 2 Division of Gastroenterology, Department of Medicine, Hospital of the University of Pennsylvania, University of Pennsylvania, Philadelphia, PA. 3 Division of Liver Diseases, Department of Medicine, Icahn School of Medicine at Mount Sinai, New York, NY. 4 Center for Clinical Epidemiology and Biostatistics, Department of Biostatistics and Epidemiology, Perelman School of Medicine, University of Pennsylvania, Philadelphia, PA. 5 Leonard Davis Institute of Health Economics, University of Pennsylvania, Philadelphia, PA.
Transplantation. 2017 Aug;101(8):1838-1844. doi: 10.1097/TP.0000000000001711.
Polycystic liver disease (PCLD) is characterized by cystic replacement of the hepatic parenchyma, leading to hepatic dysfunction, portal hypertension, and hepatomegaly. Patients with liver dysfunction and/or symptomatic disease are eligible for liver transplantation. However, little is known about these patients' waitlist outcomes relative to others with chronic liver disease.
We used Organ Procurement and Transplantation Network/United Network for Organ Sharing data from February 27, 2002 to December 31, 2015 to compare waitlist outcomes of adult patients with PCLD to those with chronic liver failure (CLF) and hepatocellular carcinoma.
The study cohort included 620 patients with PCLD, 18 240 patients with hepatocellular carcinoma, and 98 567 patients with CLF. Compared with CLF patients, PCLD patients had significantly lower bilirubin and international normalized ratio at waitlisting, and less ascites and encephalopathy. However, they were significantly more likely to have severe chronic kidney disease. Moreover, patients with PCLD were more than 70% more likely to be transplanted compared with patients with CLF (odds ratio, 1.72; 95% confidence interval, 1.46-2.02) and had significantly longer posttransplant survival (P < 0.001). PCLD patients with exceptions were 5.7 times more likely to be transplanted than those without (odds ratio, 5.67; 95% confidence interval, 3.95-8.15) and measures of hepatic/renal dysfunction were inversely associated with the receipt of exceptions.
Despite having more preserved liver synthetic function than patients with CLF on the waitlist, patients with PCLD are preferentially transplanted because they frequently receive exception points in an unstandardized fashion.
多囊肝病（PCLD）的特征是肝实质被囊肿替代，导致肝功能障碍、门静脉高压和肝肿大。肝功能不全和/或有症状性疾病的患者适合肝移植。然而，相对于其他慢性肝病患者，这些患者在等待名单上的结局鲜为人知。
我们使用了器官获取与移植网络/器官共享联合网络2002年2月27日至2015年12月31日的数据，比较成年PCLD患者与慢性肝衰竭（CLF）和肝细胞癌患者在等待名单上的结局。
研究队列包括620例PCLD患者、18240例肝细胞癌患者和98567例CLF患者。与CLF患者相比，PCLD患者在列入等待名单时胆红素和国际标准化比值显著更低，腹水和肝性脑病更少。然而，他们患严重慢性肾病的可能性显著更高。此外，与CLF患者相比，PCLD患者接受移植的可能性高出70%以上（比值比，1.72；95%置信区间，1.46 - 2.02），且移植后生存期显著更长（P < 0.001）。有例外情况的PCLD患者接受移植的可能性是无例外情况患者的5.7倍（比值比，5.67；95%置信区间，3.95 - 8.15），肝/肾功能障碍指标与获得例外情况呈负相关。
尽管在等待名单上PCLD患者的肝脏合成功能比CLF患者保留得更多，但PCLD患者仍被优先移植，因为他们经常以不标准化的方式获得例外积分。
