锝-99m焦磷酸盐单光子发射计算机断层扫描/计算机断层扫描在监测遗传性转甲状腺素蛋白淀粉样心肌病患者RNA干扰疗法治疗变化中的应用。
Use of technetium-99m-pyrophosphate single-photon emission computed tomography/computed tomography in monitoring therapeutic changes of RNA interference therapeutics in patients with hereditary transthyretin amyloid cardiomyopathy.
作者信息
Hung Yi-Hsin, Yu An-Li, Chen Yi-Chieh, Tsai Cheng-Hsuan, Su Mao-Yuan, Shun Chia-Tung, Hsueh Hsueh-Wen, Jyh-Ming Juang Jimmy, Lee Ming-Jen, Tseng Ping-Huei, Hsu Chia-Hua, Hsieh Sung-Tsang, Ko Chi-Lun, Lin Kon-Ping, Yu Wen-Chung, Cheng Mei-Fang, Chao Chi-Chao, Lin Yen-Hung
机构信息
Department of Internal Medicine, Division of Cardiology, National Taiwan University Hospital and National Taiwan University College of Medicine, Taipei, Taiwan.
Department of Internal Medicine, Division of Cardiology, National Taiwan University Hospital and National Taiwan University College of Medicine, Taipei, Taiwan; Department of Internal Medicine, Division of Cardiology, National Taiwan University Hospital Hsin-Chu Branch, Hsinchu, Taiwan.
出版信息
J Formos Med Assoc. 2025 Apr;124(4):333-339. doi: 10.1016/j.jfma.2024.10.005. Epub 2024 Oct 10.
BACKGROUND
RNA interference therapeutics reduce transthyretin production; however, their effect on hereditary transthyretin amyloid cardiomyopathy (ATTR-CA) remains unclear. We aimed to investigate alterations in technetium-99 m (Tc)-pyrophosphate (PYP) single-photon emission computed tomography/computed tomography (SPECT/CT) outcomes in patients receiving patisiran or vutrisiran.
METHODS
We retrospectively identified individuals with hereditary ATTR-CA who received patisiran or vutrisiran. First and second Tc-PYP SPECT/CT data, including visual grading, planar heart to contralateral lung (H/CL) ratio, and volumetric heart to lung (H/L) ratio were assessed.
RESULTS
Eight patients with hereditary ATTR-CA were enrolled. Cohort A included four patients who underwent their first Tc-PYP SPECT/CT imaging at the initiation of small interfering RNA (siRNA) treatment, while cohort B comprised four patients who had been receiving siRNA treatment before their first Tc-PYP SPECT/CT imaging (median duration 1281 days). Overall, there were numerical reductions in planar H/CL ratio (1.7 ± 0.2 to 1.6 ± 0.1, p = 0.050) and a significant improvement in volumetric H/L ratio (4.0 ± 0.9 to 3.5 ± 0.4, p = 0.035). Although without significance, subgroup analysis showed more pronounced changes in cohort A for both planar H/CL ratio and volumetric H/L ratio (-20.1 ± 12.6% and -17.1 ± 11.4%) compared to cohort B (-3.3 ± 11.2% and -4.3 ± 12.7%).
CONCLUSION
Our results demonstrated a significant decrease in volumetric H/L ratio in hereditary ATTR-CA patients receiving RNA interference therapeutics.
背景
RNA干扰疗法可减少转甲状腺素蛋白的产生；然而，其对遗传性转甲状腺素蛋白淀粉样心肌病（ATTR-CA）的影响仍不清楚。我们旨在研究接受帕替拉韦或伏曲瑞韦的患者中锝-99m（Tc）-焦磷酸盐（PYP）单光子发射计算机断层扫描/计算机断层扫描（SPECT/CT）结果的变化。
方法
我们回顾性地确定了接受帕替拉韦或伏曲瑞韦的遗传性ATTR-CA患者。评估了首次和第二次Tc-PYP SPECT/CT数据，包括视觉分级、平面心脏与对侧肺（H/CL）比值和体积心脏与肺（H/L）比值。
结果
纳入了8例遗传性ATTR-CA患者。队列A包括4例在小干扰RNA（siRNA）治疗开始时进行首次Tc-PYP SPECT/CT成像的患者，而队列B包括4例在首次Tc-PYP SPECT/CT成像前接受siRNA治疗的患者（中位持续时间1281天）。总体而言，平面H/CL比值有数值下降（1.7±0.2至1.6±0.1，p = 0.050），体积H/L比值有显著改善（4.0±0.9至3.5±0.4，p = 0.035）。虽然无统计学意义，但亚组分析显示，与队列B（-3.3±11.2%和-4.3±12.7%）相比，队列A的平面H/CL比值和体积H/L比值变化更明显（-20.1±12.6%和-17.1±11.4%）。
结论
我们的结果表明，接受RNA干扰疗法的遗传性ATTR-CA患者的体积H/L比值显著降低。
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