Division of Pediatric Gastroenterology, Hepatology and Nutrition, University of Pittsburgh, Pittsburgh, PA.
Pittsburgh Liver Research Center, University of Pittsburgh, Pittsburgh, PA.
Liver Transpl. 2019 Jun;25(6):911-921. doi: 10.1002/lt.25433. Epub 2019 Apr 15.
Indications for liver transplantation (LT) in metabolic disease are evolving. We reviewed the US experience with primary LT for metabolic disease in the Scientific Registry for Transplant Recipients (October 1987 to June 2017) to determine the following: temporal changes in indications, longterm outcomes, and factors predicting survival. Patients were grouped by the presence of structural liver disease (SLD) and whether the defect was confined to the liver. There were 5996 patients who underwent LT for metabolic disease, 2354 (39.3%) being children. LT for metabolic disease increased in children but not in adults. Children experienced a 6-fold increase in LT for metabolic disease without SLD. Indications for LT remained stable in adults. Living donor liver transplantation increased between era 1 and era 3 from 5.6% to 7.6% in children and 0% to 4.5% in adults. Patient and graft survival improved with time. The latest 5-year patient survival rates were 94.5% and 81.5% in children and adults, respectively. Outcomes were worse in adults and in those with extrahepatic disease (P < 0.01), whereas SLD did not affect outcomes. Survival improved with younger age at LT until age <2 years. On multivariate analysis, diagnostic category, inpatient status, age at LT, and transplant era significantly predicted outcomes in all ages with male sex predicting survival in childhood only. Children without structural disease were less likely to die awaiting LT and had improved post-LT survival compared with children with chronic liver disease. In conclusion, LT for metabolic disease is increasingly used for phenotypic correction in children; extrahepatic manifestations significantly impact survival at all ages; where indicated, transplantation should not be unnecessarily delayed; and the development of new allocation models may be required.
肝移植（LT）在代谢性疾病中的适应证正在不断发展。我们回顾了美国器官移植受者登记处（1987 年 10 月至 2017 年 6 月）的 LT 治疗代谢性疾病的经验，以确定以下内容：适应证的时间变化、长期结果和预测生存率的因素。患者根据结构性肝病（SLD）的存在和缺陷是否仅限于肝脏进行分组。共有 5996 例代谢性疾病患者接受 LT，其中 2354 例（39.3%）为儿童。儿童接受 LT 治疗代谢性疾病的比例增加，但成人没有增加。儿童在没有 SLD 的情况下，接受 LT 治疗代谢性疾病的比例增加了 6 倍。成人的 LT 适应证保持稳定。在儿童中，活体供肝移植从第 1 个时代到第 3 个时代从 5.6%增加到 7.6%，在成人中从 0%增加到 4.5%。随着时间的推移，患者和移植物的存活率有所提高。最近 5 年儿童的患者存活率分别为 94.5%和 81.5%。结果在成人和有肝外疾病的患者中较差（P<0.01），而 SLD 不影响结果。LT 年龄越小，存活率越高，直到<2 岁。多因素分析显示，诊断类别、住院状态、LT 年龄和移植时代在所有年龄段均显著预测结局，而男性仅在儿童期预测生存率。没有 SLD 的儿童在等待 LT 时死亡的可能性较小，并且与患有慢性肝病的儿童相比，LT 后存活率有所提高。总之，LT 治疗代谢性疾病越来越多地用于儿童的表型矫正；肝外表现显著影响所有年龄段的生存率；在有适应证的情况下，不应不必要地延迟移植；可能需要开发新的分配模型。
