治疗 ATTR 心肌病时 99mTc-DPD 心肌摄取量的减少。

IF 5.2 2区 医学 Q1 BIOCHEMISTRY & MOLECULAR BIOLOGY
René Rettl, Raffaella Calabretta, Franz Duca, Christina Binder, Christina Kronberger, Robin Willixhofer, Michael Poledniczek, Carolina Donà, Christian Nitsche, Dietrich Beitzke, Christian Loewe, Michaela Auer-Grumbach, Diana Bonderman, Stefan Kastl, Christian Hengstenberg, Roza Badr Eslam, Johannes Kastner, Jutta Bergler-Klein, Marcus Hacker, Andreas Kammerlander
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引用次数: 0

摘要

目的:帕替西兰和伊诺替生等新型核糖核酸干扰(RNAi)疗法已被证明有益于遗传性转甲状腺素淀粉样变性(ATTRv)患者的神经系统病程和生活质量。我们的目的是利用定量单光子发射计算机断层扫描/计算机断层扫描(SPECT/CT)成像确定RNAi疗法对ATTRv相关心肌病(ATTRv-CM)患者心肌淀粉样蛋白负荷的影响。我们还将他们与接受他法米迪治疗的野生型ATTR-CM(ATTRwt-CM)患者进行了比较:ATTRv-CM 患者在接受 RNAi 疗法(帕替西兰:n = 5;伊诺特生:n = 4)治疗 12 个月(IQR:11.0-12.0)前后接受了[99mTc]放射标记的二磷酸-1,2-丙二羧酸(99mTc-DPD)闪烁扫描和定量 SPECT/CT 成像检查。通过标准化摄取值(SUV)保留指数(基线:5.09 g/mL vs. 随访:3.19 g/mL,p = .028)测量,RNAi 治疗明显降低了 ATTRv-CM 患者的心肌定量摄取,但心功能没有明显改善。塔法米地治疗使随访[9.0个月(IQR:7.0-10.0)]时的SUV潴留指数显著降低(4.96 g/mL vs. 3.27 g/mL,p n = 40),但未对心脏功能产生有益影响:结论:RNAi疗法可明显降低ATTRv-CM患者的心肌定量摄取,与ATTRwt-CM患者的他法米迪治疗效果相当,但对心功能无影响。序列99m锝-DPD SPECT/CT成像可能是量化和监测ATTRv-CM和ATTRwt-CM患者对疾病特异性疗法反应的重要工具。
本文章由计算机程序翻译,如有差异,请以英文原文为准。
Reduction in 99mTc-DPD myocardial uptake with therapy of ATTR cardiomyopathy.

Aims: Novel ribonucleic acid interference (RNAi) therapeutics such as patisiran and inotersen have been shown to benefit neurologic disease course and quality of life in patients with hereditary transthyretin amyloidosis (ATTRv). We aimed to determine the impact of RNAi therapeutics on myocardial amyloid load using quantitative single photon emission computed tomography/computed tomography (SPECT/CT) imaging in patients with ATTRv-related cardiomyopathy (ATTRv-CM). We furthermore compared them with wild-type ATTR-CM (ATTRwt-CM) patients treated with tafamidis.Methods and results: ATTRv-CM patients underwent [99mTc]-radiolabeled diphosphono-1,2-propanodicarboxylic acid (99mTc-DPD) scintigraphy and quantitative SPECT/CT imaging before and after 12 months (IQR: 11.0-12.0) of treatment with RNAi therapeutics (patisiran: n = 5, inotersen: n = 4). RNAi treatment significantly reduced quantitative myocardial uptake as measured by standardised uptake value (SUV) retention index (baseline: 5.09 g/mL vs. follow-up: 3.19 g/mL, p = .028) in ATTRv-CM patients without significant improvement in cardiac function. Tafamidis treatment resulted in a significant reduction in SUV retention index (4.96 g/mL vs. 3.27 g/mL, p < .001) in ATTRwt-CM patients (historical control cohort: n = 40) at follow-up [9.0 months (IQR: 7.0-10.0)] without beneficial impact on cardiac function.Conclusions: RNAi therapeutics significantly reduce quantitative myocardial uptake in ATTRv-CM patients, comparable to tafamidis treatment in ATTRwt-CM patients, without impact on cardiac function. Serial 99mTc-DPD SPECT/CT imaging may be a valuable tool to quantify and monitor response to disease-specific therapies in both ATTRv-CM and ATTRwt-CM.

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来源期刊
Amyloid-Journal of Protein Folding Disorders
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
48
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.
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