2788例转甲状腺素蛋白淀粉样变性患者的临床结局：法国曲氟胺葡甲胺早期获取项目
Clinical outcomes for 2788 patients with transthyretin amyloidosis: Tafamidis meglumine early access program in France.
作者信息
Lairez Olivier, Réant Patricia, Inamo Jocelyn, Jeanneteau Julien, Bauer Fabrice, Habib Gilbert, Eicher Jean-Christophe, Lequeux Benoit, Legallois Damien, Josse Constant, Hippocrate Aurelie, Bartoli Mathilde, Dubois Margaux, Noirot Cosson Charlotte, Squara Pierre-Alexandre, Fievez Stephane, Quinault Aurore, Rudant Jeremie, Kharoubi Mounira, Damy Thibaud
机构信息
Cardiac Imaging Centre, Department of Cardiology, Toulouse University Hospital, 31059 Toulouse, France.
Cardiology Department, Haut-Levêque Hospital, Bordeaux University Hospital, University of Bordeaux, Inserm 1045, IHU Lyric, CIC1401, 33600 Pessac, France.
出版信息
Arch Cardiovasc Dis. 2025 Feb;118(2):123-132. doi: 10.1016/j.acvd.2024.08.006. Epub 2024 Oct 5.
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.
背景
在转甲状腺素蛋白相关淀粉样变性多发性神经病获批以及转甲状腺素蛋白相关淀粉样变性心肌病（ATTR-CM）的ATTR-ACT研究取得阳性结果之后，法国对曲氟胺葡甲胺（一种用于ATTR-CM的选择性转甲状腺素蛋白稳定剂）有了早期使用经验。
目的
描述法国ATTR-CM曲氟胺葡甲胺早期使用项目（2018年11月28日至2021年6月1日）中患者的特征和临床结局。
方法
确诊为ATTR-CM的患者接受20mg/天或80mg/天的曲氟胺葡甲胺治疗。前瞻性收集人口统计学和临床数据，直至患者停止治疗、死亡或项目结束。
结果
总体而言，来自126个中心的222名医生纳入了2788例患者。中位年龄为82岁，81.6%为男性，纽约心脏协会心功能分级为I级的占12.8%，II级的占60.1%，III级的占27.0%。总体而言，1943例（74.6%）进行了基因检测，1208例（62.2%）患者在开始使用曲氟胺时获得了检测结果：995例（82.4%）为野生型ATTR，213例（17.6%）为遗传性ATTR。大多数患者在诊断后≤12个月开始治疗（88.3%）：2268例（81.3%）接受20mg/天治疗，401例（17.7%）增加至80mg/天。中位随访时间为11.8个月。纽约心脏协会心功能分级改善或保持稳定的有1299例（77.6%），而在纳入研究至最后一次随访期间，376例（22.4%）病情恶化。在开始使用80mg剂量的患者中，297例（81.1%）改善或保持稳定，69例（18.9%）病情恶化。纽约心脏协会心功能分级进展情况与年龄无关。年龄<80岁患者的18个月生存率为89.8%（95%置信区间：87.0-92.0），年龄≥80岁患者的18个月生存率为86.5%（95%置信区间：83.9-88.7）。
结论
2788例ATTR-CM患者早期使用了曲氟胺葡甲胺。纽约心脏协会心功能分级进展和生存率与先前发表的数据一致。
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