Division of Transplantation, Department of Surgery, University of Minnesota, Minneapolis, MN, USA.
Clin Transplant. 2011 Sep-Oct;25(5):731-6. doi: 10.1111/j.1399-0012.2010.01371.x. Epub 2010 Nov 16.
We examined the long-term outcome of transplantation for alpha 1-antitrypsin deficiency (A1ATD).
Data were reviewed on 42 transplants in 35 children with A1ATD over 42 yr and compared with 129 transplants in 116 children with biliary atresia (BA).
Over 50% of patients were followed up for >10 yr. A1ATD were older than BA at transplantation, median age, 6.0 vs. 1.0 yr (p < 0.0001), and transplanted earlier in the course of liver failure (total bilirubin, 2.7 mg/dL [1.4-6.9] vs. 9.7 mg/dL [2.9-15.4], p = 0.005). Patient survival was greater in A1ATD than BA: one-yr post-transplant, 82.7% vs. 67.9%; five yr, 76.5% vs. 60.2%; and 10 yr, 76.5% vs. 55.9% (p = 0.03). Death-censored graft survival was similar: one-yr post-transplant, 68.4% vs. 66.2%; five yr, 68.4% vs. 55.8%; and 10 yr, 68.4% vs. 52.5% (p = 0.2). Deaths were from infection, hemorrhage, and graft failure <6 months post-transplant. Patient survival improved at five yr from 33.3% pre-cyclosporine (CSA) (1969-1984) (n = 6) to 76.5% in the CSA era (1985-1994) (n = 17) and 100% with tacrolimus (1995-2006) (n = 12) (p = 0.007).
The age at transplantation and the degree of liver dysfunction were related to the differences in graft and patient survival between A1AT and BA.
我们研究了 α1-抗胰蛋白酶缺乏症（A1ATD）患者接受肝移植的长期结果。
回顾了 35 名 A1ATD 患儿的 42 例移植数据，这些患儿在 42 年中接受了治疗，将这些数据与 116 名胆道闭锁（BA）患儿的 129 例移植数据进行了比较。
超过 50%的患者接受了超过 10 年的随访。A1ATD 患者在移植时比 BA 患者年龄更大，中位数年龄为 6.0 岁 vs. 1.0 岁（p < 0.0001），且在肝功能衰竭的早期接受移植（总胆红素，2.7 mg/dL [1.4-6.9] vs. 9.7 mg/dL [2.9-15.4]，p = 0.005）。A1ATD 患者的存活率高于 BA 患者：移植后 1 年，82.7% vs. 67.9%；5 年，76.5% vs. 60.2%；10 年，76.5% vs. 55.9%（p = 0.03）。死亡校正后的移植物存活率相似：移植后 1 年，68.4% vs. 66.2%；5 年，68.4% vs. 55.8%；10 年，68.4% vs. 52.5%（p = 0.2）。死亡原因是感染、出血和移植后 <6 个月的移植物失败。在环孢素（CSA）时代（1985-1994 年）（n = 17），患者存活率从 CSA 之前的 33.3%（1969-1984 年）（n = 6）提高到 5 年后的 76.5%，在他克莫司时代（1995-2006 年）（n = 12），患者存活率达到 100%（p = 0.007）。
移植时的年龄和肝功能障碍程度与 A1AT 和 BA 之间的移植物和患者存活率差异有关。
