Ten years of international experience with liver transplantation for familial amyloidotic polyneuropathy: Results from the familial amyloidotic polyneuropathy world transplant registry
Ten years of international experience with liver transplantation for familial amyloidotic polyneuropathy: Results from the familial amyloidotic polyneuropathy world transplant registry
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DOI：
10.1097/01.tp.0000092307.98347.cb
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发表时间：
2004-01-15
期刊：
影响因子：
6.2
通讯作者：
Ericzon, BG
中科院分区：
文献类型：
--
作者：
Herlenius, G;Wilczek, HE;Ericzon, BG
背景资料。转甲状腺素(TransThyretin，TTR)淀粉样变性是一组全身性淀粉样变性疾病，由致性淀粉样变异体引起。如果不治疗，它会慢慢导致严重的残疾症状，这些症状会无情地发展，直到患者死亡。由于突变形式的TTR主要在肝脏中产生，成功的原位肝移植(OLT)可消除变异TTR分子的来源，是目前已知的唯一根治方法。家族性淀粉样变性多发性神经病(FAP)患者的OLT于1990年在瑞典卡罗林斯卡研究所首次进行，因为结果很有希望，其他中心也采用了这一方法。为了获得尽可能多的关于这种治疗的经验，1995年发起了家族性淀粉样变性多发性神经病世界移植登记(FAPWTR)，本文介绍了10年的登记结果。共有16个国家和地区的54个中心为FAP实施了OLT，目前全球每年约有60个OLT。在过去的十年中，共有539名患者接受了579次原位肝移植。患者的存活率很高(总的5年患者存活率为77%)，与其他慢性肝病患者接受原位肝移植的存活率相当，但需要更长的随访时间才能将移植后的结果与疾病的自然病程进行比较。主要死亡原因为心脏相关死亡(39%)。我们相信，FAPWTR已经成为一个有价值的工具，它将有助于准确评估FAP患者进行OLT的潜在风险和好处，并促进从事该领域的中心之间富有成效的合作。
Background. Transthyretin (TTR) amyloidosis is a group of systemic amyloidoses disorders caused by an amyloidogenic TTR variant. Untreated, it slowly leads to severely disabling symptoms that relentlessly progress until the death of the patient. Because the mutant form of TTR is produced mainly in the liver, successful orthotopic liver transplantation (OLT) results in the elimination of the source of the variant TTR molecule and is presently the only known curative treatment. OLT in patients with familial amyloidotic polyneuropathy (FAP) was first performed in 1990 at the Karolinska Institute in Sweden, and because the results were promising other centers took up the procedure.Methods. To gain as great an experience as possible regarding this treatment, the Familial Amyloidotic Polyneuropathy World Transplant Registry (FAPWTR) was initiated in 1995, and this article presents the 10-year registry results.Results. A total of 54 centers in 16 countries have performed OLT for FAP, and today approximately 60 OLTs are performed annually worldwide. During the last decade, a total of 539 patients have undergone 579 OLTs. Patient survival is excellent (overall 5-year patient survival 77%) and comparable to the survival with OLT performed for other chronic liver disorders, but longer follow-up is needed to compare the outcome after OLT with the natural course of the disease. The main cause of death was cardiac related (39%).Conclusions. We believe that the FAPWTR has become a valuable tool that will help to accurately evaluate the potential risks and benefits of OLT in patients with FAP and promote a fruitful collaboration between centers engaged in this field.