Clinical outcomes for 2788 patients with transthyretin amyloidosis: Tafamidis meglumine early access program in France.

IF 2.3 3区医学 Q2 CARDIAC & CARDIOVASCULAR SYSTEMS Archives of Cardiovascular Diseases Pub Date : 2024-10-05 DOI:10.1016/j.acvd.2024.08.006

Olivier Lairez, Patricia Réant, Jocelyn Inamo, Julian Jeanneteau, Fabrice Bauer, Gilbert Habib, Jean-Christophe Eicher, Benoit Lequeux, Damien Legallois, Constant Josse, Aurelie Hippocrate, Mathilde Bartoli, Margaux Dubois, Charlotte Noirot Cosson, Pierre-Alexandre Squara, Stephane Fievez, Aurore Quinault, Jeremie Rudant, Mounira Kharoubi, Thibaud Damy

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Abstract

Background: Early access experience in France with tafamidis meglumine, a selective transthyretin stabilizer for transthyretin-related amyloidosis cardiomyopathy (ATTR-CM), following transthyretin-related amyloidosis (ATTR) polyneuropathy approval and positive ATTR-ACT study results.

Aim: To describe the characteristics and clinical outcomes for patients in the French ATTR-CM tafamidis meglumine early access programme (28 Nov 2018 to 01 Jun 2021).

Methods: Patients with confirmed ATTR-CM received tafamidis meglumine 20mg/day or 80mg/day. Demographic and clinical data were collected prospectively until patients discontinued treatment or died, or the programme ended.

Results: Overall, 222 physicians from 126 centres enrolled 2788 patients. The median age was 82years, 81.6% were male and New York Heart Association severity was class I for 12.8%, class II for 60.1% and class III for 27.0%. Overall, 1943 (74.6%) had genetic testing, and the results were available at tafamidis start for 1208 (62.2%) patients: 995 (82.4%) had wild-type ATTR and 213 (17.6%) had hereditary ATTR. Most patients started treatment≤12months after diagnosis (88.3%): 2268 (81.3%) at 20mg/day, with 401 (17.7%) increasing to 80mg/day. Median follow-up duration was 11.8months. New York Heart Association class improved or remained stable for 1299 (77.6%), whereas 376 (22.4%) worsened between inclusion and last follow-up. Among patients initiated at 80mg, 297 (81.1%) improved or remained stable and 69 (18.9%) worsened. New York Heart Association class progression did not vary with age. The 18-month survival rates were 89.8% (95% confidence interval: 87.0-92.0) among patients aged<80years, and 86.5% (95% confidence interval: 83.9-88.7) among those aged≥80years.

Conclusions: Early tafamidis meglumine access was given to 2788 patients with ATTR-CM. New York Heart Association class progression and survival were consistent with previously published data.

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2788 名转甲状腺素淀粉样变性患者的临床疗效：法国的塔法米迪斯巨鲁明早期使用计划。

背景：继经甲状腺素相关淀粉样变性（ATTR）多发性神经病获得批准和ATTR-ACT研究结果呈阳性后，法国开始使用他法米迪葡胺（一种治疗经甲状腺素相关淀粉样变性心肌病（ATTR-CM）的选择性经甲状腺素稳定剂）。目的：描述法国ATTR-CM他法米迪葡胺早期使用计划（2018年11月28日至2021年6月1日）患者的特征和临床结果：确诊为ATTR-CM的患者接受他法米迪葡甲胺20毫克/天或80毫克/天的治疗。前瞻性收集人口统计学和临床数据，直至患者停止治疗或死亡，或计划结束：共有来自 126 个中心的 222 名医生招募了 2788 名患者。中位年龄为 82 岁，81.6% 为男性，纽约心脏协会严重程度为 I 级的占 12.8%，II 级的占 60.1%，III 级的占 27.0%。总体而言，有 1943 名（74.6%）患者进行了基因检测，其中有 1208 名（62.2%）患者在他法米迪开始使用时已获得检测结果：其中995人（82.4%）为野生型ATTR，213人（17.6%）为遗传性ATTR。大多数患者在确诊后 12 个月内开始治疗（88.3%）：2268 例（81.3%）患者的治疗剂量为 20 毫克/天，401 例（17.7%）患者的治疗剂量增至 80 毫克/天。中位随访时间为 11.8 个月。1299例（77.6%）患者的纽约心脏协会分级有所改善或保持稳定，376例（22.4%）患者的分级在入组和最后一次随访期间有所恶化。在开始服用 80 毫克的患者中，297 人（81.1%）的病情有所改善或保持稳定，69 人（18.9%）的病情恶化。纽约心脏协会分级的进展与年龄无关。18个月的生存率为89.8%（95%置信区间：87.0-92.0）：2788名ATTR-CM患者接受了早期他法米迪葡甲胺治疗。纽约心脏协会分级进展和存活率与之前公布的数据一致。

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来源期刊

Archives of Cardiovascular Diseases 医学-心血管系统

CiteScore

4.40

自引率

6.70%

发文量

审稿时长

34 days

期刊介绍： The Journal publishes original peer-reviewed clinical and research articles, epidemiological studies, new methodological clinical approaches, review articles and editorials. Topics covered include coronary artery and valve diseases, interventional and pediatric cardiology, cardiovascular surgery, cardiomyopathy and heart failure, arrhythmias and stimulation, cardiovascular imaging, vascular medicine and hypertension, epidemiology and risk factors, and large multicenter studies. Archives of Cardiovascular Diseases also publishes abstracts of papers presented at the annual sessions of the Journées Européennes de la Société Française de Cardiologie and the guidelines edited by the French Society of Cardiology.