Use of Technetium‐99m‐Pyrophosphate Single‐Photon Emission Computed Tomography/Computed Tomography in Monitoring Therapeutic Changes of Eplontersen in Patients With Hereditary Transthyretin Amyloid Cardiomyopathy
Hereditary transthyretin amyloid cardiomyopathy (hATTR-CM) is a progressive and fatal disease. Recent evidence indicates that bone scintigraphy may serve as a tool to monitor the effectiveness of hATTR-CM treatment. The objective of this study was to examine how eplontersen therapy influences the se...
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Published in | Journal of the American Heart Association Vol. 13; no. 2; p. e030512 |
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Main Authors | , , , , , , , , , , , , , , , , |
Format | Journal Article |
Language | English |
Published |
England
John Wiley and Sons Inc
16.01.2024
Wiley |
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Abstract | Hereditary transthyretin amyloid cardiomyopathy (hATTR-CM) is a progressive and fatal disease. Recent evidence indicates that bone scintigraphy may serve as a tool to monitor the effectiveness of hATTR-CM treatment. The objective of this study was to examine how eplontersen therapy influences the semiquantitative uptake of technetium-99m-pyrophosphate in individuals diagnosed with hATTR-CM.
We retrospectively analyzed a prospective cohort from the NEURO-TTRansform trial, including patients with hATTR-CM receiving eplontersen (45 mg/4 weeks). A control group comprised patients with hATTR-CM who had not received eplontersen, inotersen, tafamidis, or patisiran. Technetium-99m-pyrophosphate single-photon emission computed tomography/computed tomography was conducted at baseline and during follow-up. Thirteen patients with hATTR-CM were enrolled, with 6 receiving eplontersen and 7 serving as the control group. The median follow-up time was 544 days. The eplontersen group exhibited a significant decrease in volumetric heart and lung ratio (3.774 to 2.979,
=0.028), whereas the control group showed no significant change (4.079 to 3.915,
=0.237). Patients receiving eplontersen demonstrated a significantly greater reduction in volumetric heart and lung ratio compared with the control group (-20.7% versus -3.4%,
=0.007).
The volumetric heart and lung ratio used to quantify technetium-99m-pyrophosphate uptake showed a significant reduction subsequent to eplontersen treatment in individuals diagnosed with hATTR-CM. These findings suggest the potential efficacy of eplontersen in treating hATTR-CM and highlight the value of technetium-99m-pyrophosphate single-photon emission computed tomography/computed tomography as a tool for monitoring therapeutic effectiveness. |
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AbstractList | Hereditary transthyretin amyloid cardiomyopathy (hATTR-CM) is a progressive and fatal disease. Recent evidence indicates that bone scintigraphy may serve as a tool to monitor the effectiveness of hATTR-CM treatment. The objective of this study was to examine how eplontersen therapy influences the semiquantitative uptake of technetium-99m-pyrophosphate in individuals diagnosed with hATTR-CM.BACKGROUNDHereditary transthyretin amyloid cardiomyopathy (hATTR-CM) is a progressive and fatal disease. Recent evidence indicates that bone scintigraphy may serve as a tool to monitor the effectiveness of hATTR-CM treatment. The objective of this study was to examine how eplontersen therapy influences the semiquantitative uptake of technetium-99m-pyrophosphate in individuals diagnosed with hATTR-CM.We retrospectively analyzed a prospective cohort from the NEURO-TTRansform trial, including patients with hATTR-CM receiving eplontersen (45 mg/4 weeks). A control group comprised patients with hATTR-CM who had not received eplontersen, inotersen, tafamidis, or patisiran. Technetium-99m-pyrophosphate single-photon emission computed tomography/computed tomography was conducted at baseline and during follow-up. Thirteen patients with hATTR-CM were enrolled, with 6 receiving eplontersen and 7 serving as the control group. The median follow-up time was 544 days. The eplontersen group exhibited a significant decrease in volumetric heart and lung ratio (3.774 to 2.979, P=0.028), whereas the control group showed no significant change (4.079 to 3.915, P=0.237). Patients receiving eplontersen demonstrated a significantly greater reduction in volumetric heart and lung ratio compared with the control group (-20.7% versus -3.4%, P=0.007).METHODS AND RESULTSWe retrospectively analyzed a prospective cohort from the NEURO-TTRansform trial, including patients with hATTR-CM receiving eplontersen (45 mg/4 weeks). A control group comprised patients with hATTR-CM who had not received eplontersen, inotersen, tafamidis, or patisiran. Technetium-99m-pyrophosphate single-photon emission computed tomography/computed tomography was conducted at baseline and during follow-up. Thirteen patients with hATTR-CM were enrolled, with 6 receiving eplontersen and 7 serving as the control group. The median follow-up time was 544 days. The eplontersen group exhibited a significant decrease in volumetric heart and lung ratio (3.774 to 2.979, P=0.028), whereas the control group showed no significant change (4.079 to 3.915, P=0.237). Patients receiving eplontersen demonstrated a significantly greater reduction in volumetric heart and lung ratio compared with the control group (-20.7% versus -3.4%, P=0.007).The volumetric heart and lung ratio used to quantify technetium-99m-pyrophosphate uptake showed a significant reduction subsequent to eplontersen treatment in individuals diagnosed with hATTR-CM. These findings suggest the potential efficacy of eplontersen in treating hATTR-CM and highlight the value of technetium-99m-pyrophosphate single-photon emission computed tomography/computed tomography as a tool for monitoring therapeutic effectiveness.CONCLUSIONSThe volumetric heart and lung ratio used to quantify technetium-99m-pyrophosphate uptake showed a significant reduction subsequent to eplontersen treatment in individuals diagnosed with hATTR-CM. These findings suggest the potential efficacy of eplontersen in treating hATTR-CM and highlight the value of technetium-99m-pyrophosphate single-photon emission computed tomography/computed tomography as a tool for monitoring therapeutic effectiveness. Hereditary transthyretin amyloid cardiomyopathy (hATTR-CM) is a progressive and fatal disease. Recent evidence indicates that bone scintigraphy may serve as a tool to monitor the effectiveness of hATTR-CM treatment. The objective of this study was to examine how eplontersen therapy influences the semiquantitative uptake of technetium-99m-pyrophosphate in individuals diagnosed with hATTR-CM. We retrospectively analyzed a prospective cohort from the NEURO-TTRansform trial, including patients with hATTR-CM receiving eplontersen (45 mg/4 weeks). A control group comprised patients with hATTR-CM who had not received eplontersen, inotersen, tafamidis, or patisiran. Technetium-99m-pyrophosphate single-photon emission computed tomography/computed tomography was conducted at baseline and during follow-up. Thirteen patients with hATTR-CM were enrolled, with 6 receiving eplontersen and 7 serving as the control group. The median follow-up time was 544 days. The eplontersen group exhibited a significant decrease in volumetric heart and lung ratio (3.774 to 2.979, =0.028), whereas the control group showed no significant change (4.079 to 3.915, =0.237). Patients receiving eplontersen demonstrated a significantly greater reduction in volumetric heart and lung ratio compared with the control group (-20.7% versus -3.4%, =0.007). The volumetric heart and lung ratio used to quantify technetium-99m-pyrophosphate uptake showed a significant reduction subsequent to eplontersen treatment in individuals diagnosed with hATTR-CM. These findings suggest the potential efficacy of eplontersen in treating hATTR-CM and highlight the value of technetium-99m-pyrophosphate single-photon emission computed tomography/computed tomography as a tool for monitoring therapeutic effectiveness. Background Hereditary transthyretin amyloid cardiomyopathy (hATTR‐CM) is a progressive and fatal disease. Recent evidence indicates that bone scintigraphy may serve as a tool to monitor the effectiveness of hATTR‐CM treatment. The objective of this study was to examine how eplontersen therapy influences the semiquantitative uptake of technetium‐99m‐pyrophosphate in individuals diagnosed with hATTR‐CM. Methods and Results We retrospectively analyzed a prospective cohort from the NEURO‐TTRansform trial, including patients with hATTR‐CM receiving eplontersen (45 mg/4 weeks). A control group comprised patients with hATTR‐CM who had not received eplontersen, inotersen, tafamidis, or patisiran. Technetium‐99m‐pyrophosphate single‐photon emission computed tomography/computed tomography was conducted at baseline and during follow‐up. Thirteen patients with hATTR‐CM were enrolled, with 6 receiving eplontersen and 7 serving as the control group. The median follow‐up time was 544 days. The eplontersen group exhibited a significant decrease in volumetric heart and lung ratio (3.774 to 2.979, P=0.028), whereas the control group showed no significant change (4.079 to 3.915, P=0.237). Patients receiving eplontersen demonstrated a significantly greater reduction in volumetric heart and lung ratio compared with the control group (−20.7% versus −3.4%, P=0.007). Conclusions The volumetric heart and lung ratio used to quantify technetium‐99m‐pyrophosphate uptake showed a significant reduction subsequent to eplontersen treatment in individuals diagnosed with hATTR‐CM. These findings suggest the potential efficacy of eplontersen in treating hATTR‐CM and highlight the value of technetium‐99m‐pyrophosphate single‐photon emission computed tomography/computed tomography as a tool for monitoring therapeutic effectiveness. |
Author | Chen, Yi‐Chieh Chao, Chi‐Chao Lin, Yen‐Hung Hsueh, Hsueh‐Wen Tseng, Ping‐Huei Shun, Chia‐Tung Juang, Jimmy Jyh‐Ming Wu, Yuan‐Kun Aden Hsieh, Sung‐Tsang Cheng, Mei‐Fang Yu, An‐Li Chou, Chia‐Hung Hsu, Chia‐Hua Ko, Chi‐Lun Lee, Ming‐Jen Tsai, Cheng‐Hsuan Su, Mao‐Yuan |
AuthorAffiliation | 11 Institute of Environmental and Occupational Health Sciences National Taiwan University Taipei Taiwan 6 Department of Medical Imaging and Radiological Technology Yuanpei University of Medical Technology Hsinchu Taiwan 8 Department of Forensic Medicine and Pathology National Taiwan University Hospital Taipei Taiwan 2 Department of Internal Medicine, Division of Cardiology National Taiwan University Hospital Hsin‐Chu Branch Hsinchu Taiwan 4 National Taiwan University College of Medicine Graduate Institute of Clinical Medicine Taipei Taiwan 9 Department of Neurology National Taiwan University Hospital and National Taiwan University College of Medicine Taipei Taiwan 7 Department of Obstetrics and Gynecology National Taiwan University Hospital and National Taiwan University College of Medicine Taipei Taiwan 5 Department of Medical Imaging National Taiwan University Hospital Taipei Taiwan 10 Department of Internal Medicine, Division of Gastroenterology National Taiwan University Hospital and Natio |
AuthorAffiliation_xml | – name: 6 Department of Medical Imaging and Radiological Technology Yuanpei University of Medical Technology Hsinchu Taiwan – name: 7 Department of Obstetrics and Gynecology National Taiwan University Hospital and National Taiwan University College of Medicine Taipei Taiwan – name: 4 National Taiwan University College of Medicine Graduate Institute of Clinical Medicine Taipei Taiwan – name: 11 Institute of Environmental and Occupational Health Sciences National Taiwan University Taipei Taiwan – name: 1 Department of Internal Medicine, Division of Cardiology National Taiwan University Hospital and National Taiwan University College of Medicine Taipei Taiwan – name: 8 Department of Forensic Medicine and Pathology National Taiwan University Hospital Taipei Taiwan – name: 10 Department of Internal Medicine, Division of Gastroenterology National Taiwan University Hospital and National Taiwan University College of Medicine Taipei Taiwan – name: 2 Department of Internal Medicine, Division of Cardiology National Taiwan University Hospital Hsin‐Chu Branch Hsinchu Taiwan – name: 5 Department of Medical Imaging National Taiwan University Hospital Taipei Taiwan – name: 3 Department of Nuclear Medicine National Taiwan University Hospital and National Taiwan University College of Medicine Taipei Taiwan – name: 9 Department of Neurology National Taiwan University Hospital and National Taiwan University College of Medicine Taipei Taiwan |
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BackLink | https://www.ncbi.nlm.nih.gov/pubmed/38214277$$D View this record in MEDLINE/PubMed |
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Copyright | 2024 The Authors. Published on behalf of the American Heart Association, Inc., by Wiley. |
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DocumentTitleAlternate | Monitoring Effectiveness of Eplontersen by Tc‐PYP |
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Keywords | 99mTc‐pyrophosphate hereditary transthyretin amyloidosis eplontersen single‐photon emission computed tomography/computed tomography |
Language | English |
License | This is an open access article under the terms of the http://creativecommons.org/licenses/by-nc-nd/4.0/ License, which permits use and distribution in any medium, provided the original work is properly cited, the use is non‐commercial and no modifications or adaptations are made. |
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Notes | ObjectType-Article-1 SourceType-Scholarly Journals-1 ObjectType-Feature-2 content type line 23 This article was sent to Barry London, MD, PhD, Senior Guest Editor, for review by expert referees, editorial decision, and final disposition. For Sources of Funding and Disclosures, see page 9. M.‐F. Cheng and C.‐C. Chao contributed equally. |
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Snippet | Hereditary transthyretin amyloid cardiomyopathy (hATTR-CM) is a progressive and fatal disease. Recent evidence indicates that bone scintigraphy may serve as a... Background Hereditary transthyretin amyloid cardiomyopathy (hATTR‐CM) is a progressive and fatal disease. Recent evidence indicates that bone scintigraphy may... |
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SubjectTerms | 99mTc‐pyrophosphate eplontersen hereditary transthyretin amyloidosis Original Research single‐photon emission computed tomography/computed tomography |
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Title | Use of Technetium‐99m‐Pyrophosphate Single‐Photon Emission Computed Tomography/Computed Tomography in Monitoring Therapeutic Changes of Eplontersen in Patients With Hereditary Transthyretin Amyloid Cardiomyopathy |
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