Abstract

Background

By stabilizing transthyretin, tafamidis delays progression of amyloidosis due to transthyretin variant (ATTRv) and replaced liver transplantation (LT) as the first-line therapy. No study compared these two therapeutic strategies.

Methods

In a monocentric retrospective cohort analysis, patients with ATTRv amyloidosis treated with either tafamidis or LT were compared using a propensity score and a competing risk analysis for three endpoints: all-cause mortality, cardiac worsening (heart failure or cardiovascular death) and neurological worsening (worsening in PolyNeuropathy Disability score).

Results

345 patients treated with tafamidis (n = 129) or LT (n = 216) were analyzed, and 144 patients were matched (72 patients in each group, median age 54 years, 60% carrying the V30M mutation, 81% of stage I, 69% with cardiac involvement, median follow-up: 68 months). Patients treated with tafamidis had longer survival than LT patients (HR: 0.35; p = .032). Conversely, they also presented a 3.0-fold higher risk of cardiac worsening and a 7.1-fold higher risk of neurological worsening (p = .0071 and p < .0001 respectively).

Conclusions

ATTRv amyloidosis patients treated with tafamidis would present a better survival but also a faster deterioration of their cardiac and neurological statuses as compared with LT. Further studies are needed to clarify the therapeutic strategy in ATTRv amyloidosis.

中文翻译：

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他法米迪与肝移植一线治疗遗传性转甲状腺素蛋白淀粉样变性的比较

摘要

背景

通过稳定转甲状腺素蛋白，tafamidis 可延缓转甲状腺素蛋白变异 (ATTRv) 引起的淀粉样变性的进展，并取代肝移植 (LT) 作为一线治疗。没有研究比较这两种治疗策略。

方法

在一项单中心回顾性队列分析中，使用倾向评分和竞争风险分析对接受他法米迪或 LT 治疗的 ATTRv 淀粉样变性患者进行了比较，以评估三个终点：全因死亡率、心脏恶化（心力衰竭或心血管死亡）和神经系统恶化。多发性神经病残疾评分恶化）。

结果

对 345 例接受他法米迪 ( n  = 129) 或 LT ( n  = 216) 治疗的患者进行了分析，并匹配了 144 例患者（每组 72 例患者，中位年龄 54 岁，60% 携带 V30M 突变，81% 为 I 期， 69% 患有心脏受累，中位随访时间：68 个月）。使用他法米迪治疗的患者比 LT 患者的生存期更长（HR：0.35；p  = .032）。相反，他们的心脏恶化风险也高出 3.0 倍，神经系统恶化风险高出 7.1 倍（分别为p  = .0071 和p <  .0001）。

结论

与 LT 相比，接受 Tafamidis 治疗的 ATTRv 淀粉样变性患者的生存率更高，但心脏和神经系统状态恶化得更快。需要进一步的研究来阐明 ATTRv 淀粉样变性的治疗策略。