Okamoto Sadahisa, Wixner Jonas, Obayashi Konen, Ando Yukio, Ericzon Bo-Göran, Friman Styrbjörn, Uchino Makoto, Suhr Ole B
Department of Medicine, Umeå University Hospital, Umeå, Sweden.
Liver Transpl. 2009 Oct;15(10):1229-35. doi: 10.1002/lt.21817.
Liver transplantation (LTx) for familial amyloidotic polyneuropathy (FAP) is an accepted treatment for this fatal disease. However, the long-term outcome with respect to that of nontransplanted patients has not been fully elucidated. The aim of this study was to compare the long-term survival of Swedish LTx FAP patients with that of historical controls, especially with respect to the age at onset of the disease and gender. In order to evaluate the outcome of LTx as a treatment for FAP, survival was calculated from the onset of disease. One hundred forty-one FAP patients, 108 transplanted and 33 not transplanted, were included in the study. Significantly increased survival was noted for LTx patients in comparison with controls. The outcome was especially favorable for those with an early onset of the disease (age at onset < 50 years) in comparison with early-onset controls (P < 0.001). In contrast, no significant difference for late-onset cases (> or = 50 years) was found. Transplanted late-onset females had significantly improved survival in comparison with transplanted late-onset males (P = 0.02). We were unable to find significant differences in survival between patients with long (> or = 7 years) or short (<7 years) disease duration at transplantation. The survival of male patients with late-onset disease appeared not to improve with LTx. LTx is an efficacious treatment for improving the survival of early-onset FAP patients. Further studies are needed to analyze the cause of the poorer outcome for late-onset male patients.
肝移植（LTx）用于治疗家族性淀粉样多神经病（FAP），是这种致命疾病公认的治疗方法。然而，与未接受移植的患者相比，其长期预后尚未完全阐明。本研究的目的是比较瑞典接受肝移植的FAP患者与历史对照者的长期生存率，特别是在疾病发病年龄和性别方面。为了评估肝移植作为FAP治疗方法的疗效，从疾病发病时开始计算生存率。本研究纳入了141例FAP患者，其中108例接受了移植，33例未接受移植。与对照组相比，肝移植患者的生存率显著提高。与早发型对照组相比，疾病早发（发病年龄<50岁）的患者预后尤其良好（P<0.001）。相比之下，晚发型病例（≥50岁）未发现显著差异。与晚发型男性肝移植患者相比，晚发型女性肝移植患者的生存率显著提高（P = 0.02）。我们未能发现移植时疾病病程长（≥7年）或短（<7年）的患者在生存率上存在显著差异。晚发型男性患者的生存率似乎并未因肝移植而提高。肝移植是提高早发型FAP患者生存率的有效治疗方法。需要进一步研究分析晚发型男性患者预后较差的原因。
