Yamamoto S, Wilczek H E, Nowak G, Larsson M, Oksanen A, Iwata T, Gjertsen H, Söderdahl G, Wikström L, Ando Y, Suhr O B, Ericzon B-G
Division of Transplantation Surgery, Department of Clinical Science, Intervention and Technology, Karolinska Institute, Karolinska University Hospital, Huddinge, Stockholm, Sweden.
Am J Transplant. 2007 Nov;7(11):2597-604. doi: 10.1111/j.1600-6143.2007.01969.x. Epub 2007 Sep 14.
Orthotopic liver transplantation (LTx) is currently the only available treatment that has been proven to halt the progress of familial amyloidotic polyneuropathy (FAP). The aim of this study was to assess mortality and symptomatic response to LTx for FAP. All 86 FAP patients transplanted at our hospital between April 1990 and November 2005 were included in the study. Five patients underwent retransplantation. The 1-, 3- and 5-year patient survival rates in patients transplanted during 1996-2005 were 94.6%, 92.3% and 92.3%, respectively, a significant difference from the rates of 76.7%, 66.7% and 66.7%, respectively, during 1990-1995 (p = 0.0003). Multivariate analysis revealed that the age at the time of LTx (>or=40 years), duration of the disease (>or=7 years) and modified body mass index (mBMI) (<600) were independent prognostic factors for patient survival. A halt in the progress of symptoms was noted in most patients, but only a minority experienced an improvement after LTx. To optimize the posttransplant prognosis, LTx should be performed in the early stages of the disease, and close post-LTx monitoring of heart function by echocardiography and of heart arrhythmia by Holter ECG is mandatory.
原位肝移植（LTx）是目前唯一已被证实可阻止家族性淀粉样多神经病（FAP）进展的有效治疗方法。本研究的目的是评估FAP患者接受LTx后的死亡率及症状反应。1990年4月至2005年11月间在我院接受移植的所有86例FAP患者均纳入本研究。5例患者接受了再次移植。1996 - 2005年期间接受移植患者的1年、3年和5年生存率分别为94.6%、92.3%和92.3%，与1990 - 1995年期间分别为76.7%、66.7%和66.7%的生存率有显著差异（p = 0.0003）。多因素分析显示，LTx时的年龄（≥40岁）、疾病持续时间（≥7年）和改良体重指数（mBMI）（<600）是患者生存的独立预后因素。大多数患者症状进展停止，但只有少数患者在LTx后症状改善。为优化移植后预后，应在疾病早期进行LTx，并且必须通过超声心动图密切监测LTx后的心脏功能以及通过动态心电图监测心律失常。
