Department of Pediatrics, The Children's Hospital of Philadelphia, Philadelphia, PA, USA.
J Pediatr Gastroenterol Nutr. 2010 Jan;50(1):11-5. doi: 10.1097/MPG.0b013e3181c1601f.
Alagille syndrome is a multisystem disorder in which progressive liver disease with persistent cholestasis and dramatic pruritus often warrant consideration for liver transplantation. The most important part of the transplant assessment is evaluation of the cardiac and renal involvement. Preoperatively, cardiac performance often must be tested with dynamic stress tests, mimicking hemodynamic changes during liver transplant. Many aspects of the syndrome including cholestasis, pruritus, and hypercholesterolemia improve posttransplant, but short stature is rarely significantly affected. One- and 5-year patient and graft survival after liver transplant is comparable to other elective indications, but effects of long-term immunosuppressants on evolution of other components of the syndrome, including vascular, bone, and renal disease, remain largely unknown.
肝内胆管发育不良综合征是一种多系统疾病，其特征为进行性肝脏疾病伴持续胆汁淤积和显著瘙痒，常需考虑进行肝移植。移植评估最重要的部分是评估心脏和肾脏受累情况。术前，心脏功能通常必须通过动态应激测试进行测试，以模拟肝移植期间的血液动力学变化。该综合征的许多方面，包括胆汁淤积、瘙痒和高胆固醇血症，在移植后都会得到改善，但身材矮小很少受到显著影响。肝移植后 1 年和 5 年患者和移植物存活率与其他选择性适应证相当，但长期免疫抑制剂对该综合征其他成分（包括血管、骨骼和肾脏疾病）的发展的影响在很大程度上仍不清楚。
