Comparison between tafamidis and liver transplantation as first-line therapy for hereditary transthyretin amyloidosis.

IF 5.2 2区医学 Q1 BIOCHEMISTRY & MOLECULAR BIOLOGY Amyloid-Journal of Protein Folding Disorders Pub Date : 2023-09-01 DOI:10.1080/13506129.2023.2177986

Pierre Socie, Anouar Benmalek, Cécile Cauquil, Eve Piekarski, Ilias Kounis, Ludivine Eliahou, Antoine Rousseau, François Rouzet, Andoni Echaniz-Laguna, Didier Samuel, David Adams, Michel S Slama, Vincent Algalarrondo

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引用次数: 2

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.

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他法非底斯与肝移植作为遗传性甲状腺转蛋白淀粉样变一线治疗的比较。

背景:通过稳定转甲状腺素，他法底斯延缓了由转甲状腺素变异(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，使用倾向性评分和三个终点的竞争风险分析对接受他法非迪或肝移植治疗的ATTRv淀粉样变性患者进行比较: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(多发性神经病变残疾评分恶化)。结果:345例接受他法底斯(n = 129)或LT (n = 216)治疗的患者进行了分析，144例患者进行了匹配(每组72例，中位年龄54岁，60%携带V30M突变，81%为I期，69%为心脏受损伤，中位随访:68个月)。接受他法非底斯治疗的患者比LT患者生存时间更长(HR: 0.35;p = .032)。相反，他们心脏恶化的风险也高出3.0倍，神经系统恶化的风险高出7.1倍(p =。0071和p。0001分别)。结论:与lt相比，接受他法非地治疗的ATTRv淀粉样变患者生存率更高，但心脏和神经系统状况恶化更快。需要进一步的研究来阐明ATTRv淀粉样变的治疗策略。

本文章由计算机程序翻译，如有差异，请以英文原文为准。

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来源期刊

Amyloid-Journal of Protein Folding Disorders 生物-生化与分子生物学

CiteScore

10.60

自引率

10.90%

发文量

审稿时长

6-12 weeks

期刊介绍： Amyloid: the Journal of Protein Folding Disorders is dedicated to the study of all aspects of the protein groups and associated disorders that are classified as the amyloidoses as well as other disorders associated with abnormal protein folding. The journals major focus points are: etiology, pathogenesis, histopathology, chemical structure, nature of fibrillogenesis; whilst also publishing papers on the basic and chemical genetic aspects of many of these disorders. Amyloid is recognised as one of the leading publications on amyloid protein classifications and the associated disorders, as well as clinical studies on all aspects of amyloid related neurodegenerative diseases and major clinical studies on inherited amyloidosis, especially those related to transthyretin. The Journal also publishes book reviews, meeting reports, editorials, thesis abstracts, review articles and symposia in the various areas listed above.