Early liver transplantation improves familial amyloidotic polyneuropathy patients' survival
Early liver transplantation improves familial amyloidotic polyneuropathy patients' survival
复制标题
早期肝移植可改善家族性淀粉样多发性神经病患者的生存率
DOI：
10.1080/13506120500363609
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发表时间：
2005
期刊：
影响因子：
5.5
通讯作者：
B. Ericzon
中科院分区：
文献类型：
--
作者：
O. Suhr;S. Friman;B. Ericzon
自从1990年第一例肝移植治疗家族性淀粉样多发性神经病以来，它已经成为这种系统性淀粉样疾病的公认治疗方法。肝移植停止了突变的淀粉样甲状腺素运载蛋白的产生，从而停止了淀粉样蛋白的形成，也停止了大多数症状的进展。与非移植受试者相比，移植患者从疾病发作起的生存期改善尚未得到证实，部分原因是疾病的自然相对缓慢进展，预期中位生存期为13年。在这项回顾性研究中，我们比较了早期初始系列（n = 34）的移植，其中接受严重营养不良的患者，与后来的系列（n = 27）的移植，以及对照组（n = 19）的非移植患者。与非移植的历史对照组相比，改良体重指数（mBMI）高于600的移植患者的生存率有所改善。到目前为止，尚未发现早期和晚期系列之间的生存率存在差异。我们之前建议主要根据患者的营养状况选择患者，这似乎是有道理的，因为现在可以证明，与非移植患者对照组相比，营养状况保持不变的移植患者组（mBMI > 600）的生存率有所提高。
Since the first liver transplantation for familial amyloidotic polyneuropathy was performed in 1990, it has become an accepted treatment for this systemic amyloid disorder. Liver transplantation halts the production of the mutated amyloidogenic transthyretin, and thereby amyloid formation, and also progression of the majority of symptoms. Improvement in survival from onset of disease in transplanted patients compared to non-transplanted subjects has of yet not been demonstrated, partly because of the natural relatively slow progression of the disease with an expected median survival of 13 years. In this retrospective study we compared the early initial series (n = 34) of transplantations, where severely malnourished patients were accepted, with a later series (n = 27) of transplants, as well as a control group (n = 19) consisting of non-transplanted patients. For transplanted patients with an modified body mass index (mBMI) above 600 an improved survival was noted compared with that of non-transplanted historical controls. So far no difference in survival between the early and late series has been found. Our previous recommendation of selection of patients primarily according to their nutritional status appears to be well justified, since it is now possible to demonstrate an increased survival for the transplanted group of patients with a preserved nutritional status (mBMI > 600) compared to the control group of non-transplanted patients.