Outcomes for Recipients of Liver Transplantation for Alpha-1-Antitrypsin Deficiency-Related Cirrhosis
Outcomes for Recipients of Liver Transplantation for Alpha-1-Antitrypsin Deficiency-Related Cirrhosis
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DOI：
10.1002/lt.23744
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发表时间：
2013-12-01
影响因子：
4.6
通讯作者：
Krowka, Michael J.
中科院分区：
文献类型：
--
作者：
Carey, Elizabeth J.;Iyer, Vivek N.;Krowka, Michael J.
α-1-抗胰蛋白酶(AAT)缺乏症是一种罕见的遗传性疾病，由丝氨酸蛋白酶抑制剂AAT的异常产生引起。肝移植治疗AAT缺乏所致的肝硬变，恢复AAT的正常产生。有关AAT缺乏症患者肝移植后结局的报道很少。本研究的目的是确定在3个大型移植中心接受肝移植治疗的AAT缺乏症患者的特征和结果。纳入1987-2012年间在这3个移植中心接受肝移植的所有AAT缺乏症(ZZ或SZ表型)患者。最近的50名MZ表型患者也被纳入进行比较。数据是从内部数据库和医疗记录中追溯收集的。73名患者(50名ZZ表型和23名SZ表型)接受了肝移植。平均年龄为52.8岁，大多数患者(75.6%)是男性。移植前，ZZ患者的血清丙氨酸氨基转移酶水平低于SZ患者(28.3vs58.0mgdL，P<；0.001)。超过40%的SZ患者有额外的肝病，而ZZ组和MZ组分别为8%和90%。肝移植前，ZZ组和SZ组肺功能无明显差异。17例患者(均为ZZ表型)在肝移植前后进行了肺功能测试。多数患者1秒用力呼气量(FEV1)继续下降。术后1、3、5、10年生存率分别为86%、83%、80%和72%，SZ组分别为91%、86%、79%和79%。综上所述，ZZ或SZ AAT缺乏症患者的肝移植后存活率很高。尽管肝移植后AAT水平恢复正常，但在一些ZZ和SZ患者中，肝移植后FEV1仍意外下降。《肝脏移植》19：1370-1376,2013。(C)2013年AASLD。
Alpha-1-antitrypsin (AAT) deficiency is a rare genetic disease caused by an abnormal production of the serine protease inhibitor AAT. Liver transplantation (LT) cures cirrhosis caused by AAT deficiency and restores the normal production of AAT. There are few reports on the post-LT outcomes of patients with AAT deficiency. The aim of this study was to determine the characteristics and outcomes of patients undergoing LT for AAT deficiency at 3 large transplant centers. All patients undergoing LT at these 3 transplant centers from 1987 to 2012 for AAT deficiency (ZZ or SZ phenotype) were included. The most recent 50 patients with the MZ phenotype were also included for comparison. Data were collected retrospectively from internal databases and medical records. Seventy-three patients (50 with the ZZ phenotype and 23 with the SZ phenotype) underwent LT. The mean age was 52.8 years, and the majority of the patients (75.6%) were men. Before LT, serum AAT levels were lower for the ZZ patients versus the SZ patients (28.3 versus 58.0 mg/dL, P < 0.001). More than 40% of the SZ patients had an additional liver disease, whereas 8% in the ZZ group and 90% in the MZ group did. Before LT, there was no significant difference in pulmonary function between the ZZ and SZ groups. Seventeen patients (all with ZZ phenotype) had pulmonary function tests performed before and after LT. The forced expiratory volume in 1 second (FEV1) continued to decline for the majority. The 1-, 3-, 5-, and 10-year post-LT survival rates were 86%, 83%, 80%, and 72%, respectively, for the ZZ patients and 91%, 86%, 79%, and 79%, respectively, for the SZ patients. In conclusion, survival after LT for patients with ZZ or SZ AAT deficiency is excellent. Despite the normalization of AAT levels after LT, FEV1 continues to decline unexpectedly after LT in some ZZ and SZ patients. Liver Transpl 19:1370-1376, 2013. (c) 2013 AASLD.