Cover Image

Effect of eplontersen on cardiac structure and function in patients with hereditary transthyretin amyloidosis

Background - Hereditary transthyretin amyloidosis (ATTRv) is associated with polyneuropathy, cardiomyopathy, or both. The effects of eplontersen on cardiac structure and function were assessed. - Methods and Results - NEURO-TTRansform was an open-label trial involving 144 adults with ATTRv polyneuro...

Full description

Saved in:
Bibliographic Details
Main Authors: Masri, Ahmad (Author) , Maurer, Mathew S. (Author) , Claggett, Brian L. (Author) , Kulac, Ian (Author) , Waddington Cruz, Marcia (Author) , Conceição, Isabel (Author) , Weiler, Markus (Author) , Berk, John L. (Author) , Gertz, Morie (Author) , Gillmore, Julian D. (Author) , Rush, Stephen (Author) , Chen, Jersey (Author) , Zhou, Wunan (Author) , Kwoh, Jesse (Author) , Duran, Jason M. (Author) , Tsimikas, Sotirios (Author) , Solomon, Scott D. (Author)
Format: Article (Journal)
Language:English
Published: August 2024
In: Journal of cardiac failure
Year: 2024, Volume: 30, Issue: 8, Pages: 973-980
ISSN:1532-8414
DOI:10.1016/j.cardfail.2023.11.016
Online Access:Verlag, lizenzpflichtig, Volltext: https://doi.org/10.1016/j.cardfail.2023.11.016
Verlag, lizenzpflichtig, Volltext: https://www.sciencedirect.com/science/article/pii/S1071916423008941
Get full text
Author Notes:Ahmad Masri, Mathew S. Maurer, Brian L. Claggett, Ian Kulac, Marcia Waddington Cruz, Isabel Conceição, Markus Weiler, John L. Berk, Morie Gertz, Julian D. Gillmore, Stephen Rush, Jersey Chen, Wunan Zhou, Jesse Kwoh, Jason M. Duran, Sotirios Tsimikas and Scott D. Solomon

MARC

LEADER 00000naa a2200000 c 4500
001 193015335X
003 DE-627
005 20250710111457.0
007 cr uuu---uuuuu
008 250710s2024 xx |||||o 00| ||eng c
024 7 |a 10.1016/j.cardfail.2023.11.016  |2 doi 
035 |a (DE-627)193015335X 
035 |a (DE-599)KXP193015335X 
040 |a DE-627  |b ger  |c DE-627  |e rda 
041 |a eng 
084 |a 33  |2 sdnb 
100 1 |a Masri, Ahmad  |e VerfasserIn  |0 (DE-588)1371128537  |0 (DE-627)1930153864  |4 aut 
245 1 0 |a Effect of eplontersen on cardiac structure and function in patients with hereditary transthyretin amyloidosis  |c Ahmad Masri, Mathew S. Maurer, Brian L. Claggett, Ian Kulac, Marcia Waddington Cruz, Isabel Conceição, Markus Weiler, John L. Berk, Morie Gertz, Julian D. Gillmore, Stephen Rush, Jersey Chen, Wunan Zhou, Jesse Kwoh, Jason M. Duran, Sotirios Tsimikas and Scott D. Solomon 
264 1 |c August 2024 
300 |b Illustrationen 
300 |a 8 
336 |a Text  |b txt  |2 rdacontent 
337 |a Computermedien  |b c  |2 rdamedia 
338 |a Online-Ressource  |b cr  |2 rdacarrier 
500 |a Online verfügbar: 7. Dezember 2023, Artikelversion: 13. August 2024 
500 |a Gesehen am 10.07.2025 
520 |a Background - Hereditary transthyretin amyloidosis (ATTRv) is associated with polyneuropathy, cardiomyopathy, or both. The effects of eplontersen on cardiac structure and function were assessed. - Methods and Results - NEURO-TTRansform was an open-label trial involving 144 adults with ATTRv polyneuropathy (49 patients [34%] with cardiomyopathy) receiving eplontersen throughout and compared with a historical placebo group (n=60; 30 patients [50%] with cardiomyopathy) from the NEURO-TTR trial at week 65. Treatment effect (eplontersen vs placebo), presented as mean difference (95% confidence interval) was analyzed after adjusting for age, sex, region, baseline value, ATTRv disease stage, previous ATTRv treatment, and V30M transthyretin variant. There were notable differences at baseline between the eplontersen group and historical placebo. In the cardiomyopathy subgroup, 65 weeks of eplontersen treatment was associated with improvement from baseline relative to placebo in left ventricular ejection fraction of 4.3% (95% confidence interval 1.40-21.01; P=.049) and stroke volume 10.64 mL (95% confidence interval 3.99-17.29; P=.002) while the remainder of echocardiographic parameters remained stable. - Conclusions - Eplontersen was associated with stable or improved measures of cardiac structure and function vs historical placebo in patients with ATTRv polyneuropathy and cardiomyopathy. Further investigation into eplontersen's effect on transthyretin amyloid cardiomyopathy is being conducted in the CARDIO-TTRansform trial. 
650 4 |a Amyloidosis 
650 4 |a clinical trial 
650 4 |a echo 
650 4 |a eplontersen 
650 4 |a polyneuropathy 
650 4 |a transthyretin 
700 1 |a Maurer, Mathew S.  |e VerfasserIn  |4 aut 
700 1 |a Claggett, Brian L.  |e VerfasserIn  |4 aut 
700 1 |a Kulac, Ian  |e VerfasserIn  |4 aut 
700 1 |a Waddington Cruz, Marcia  |e VerfasserIn  |4 aut 
700 1 |a Conceição, Isabel  |e VerfasserIn  |4 aut 
700 1 |a Weiler, Markus  |d 1972-  |e VerfasserIn  |0 (DE-588)123920256  |0 (DE-627)706474236  |0 (DE-576)293943079  |4 aut 
700 1 |a Berk, John L.  |e VerfasserIn  |4 aut 
700 1 |a Gertz, Morie  |e VerfasserIn  |4 aut 
700 1 |a Gillmore, Julian D.  |e VerfasserIn  |4 aut 
700 1 |a Rush, Stephen  |e VerfasserIn  |4 aut 
700 1 |a Chen, Jersey  |e VerfasserIn  |4 aut 
700 1 |a Zhou, Wunan  |e VerfasserIn  |4 aut 
700 1 |a Kwoh, Jesse  |e VerfasserIn  |4 aut 
700 1 |a Duran, Jason M.  |e VerfasserIn  |4 aut 
700 1 |a Tsimikas, Sotirios  |e VerfasserIn  |4 aut 
700 1 |a Solomon, Scott D.  |e VerfasserIn  |4 aut 
773 0 8 |i Enthalten in  |t Journal of cardiac failure  |d New York, NY : Elsevier, 1994  |g 30(2024), 8 vom: Aug., Seite 973-980  |h Online-Ressource  |w (DE-627)330078410  |w (DE-600)2048826-9  |w (DE-576)264629159  |x 1532-8414  |7 nnas  |a Effect of eplontersen on cardiac structure and function in patients with hereditary transthyretin amyloidosis 
773 1 8 |g volume:30  |g year:2024  |g number:8  |g month:08  |g pages:973-980  |g extent:8  |a Effect of eplontersen on cardiac structure and function in patients with hereditary transthyretin amyloidosis 
856 4 0 |u https://doi.org/10.1016/j.cardfail.2023.11.016  |x Verlag  |x Resolving-System  |z lizenzpflichtig  |3 Volltext 
856 4 0 |u https://www.sciencedirect.com/science/article/pii/S1071916423008941  |x Verlag  |z lizenzpflichtig  |3 Volltext 
951 |a AR 
992 |a 20250710 
993 |a Article 
994 |a 2024 
998 |g 123920256  |a Weiler, Markus  |m 123920256:Weiler, Markus  |d 910000  |d 911100  |d 50000  |e 910000PW123920256  |e 911100PW123920256  |e 50000PW123920256  |k 0/910000/  |k 1/910000/911100/  |k 0/50000/  |p 7 
999 |a KXP-PPN193015335X  |e 4744537278 
BIB |a Y 
SER |a journal 
JSO |a {"physDesc":[{"noteIll":"Illustrationen","extent":"8 S."}],"recId":"193015335X","note":["Online verfügbar: 7. Dezember 2023, Artikelversion: 13. August 2024","Gesehen am 10.07.2025"],"type":{"media":"Online-Ressource","bibl":"article-journal"},"language":["eng"],"title":[{"title":"Effect of eplontersen on cardiac structure and function in patients with hereditary transthyretin amyloidosis","title_sort":"Effect of eplontersen on cardiac structure and function in patients with hereditary transthyretin amyloidosis"}],"person":[{"display":"Masri, Ahmad","role":"aut","given":"Ahmad","family":"Masri"},{"display":"Maurer, Mathew S.","role":"aut","given":"Mathew S.","family":"Maurer"},{"display":"Claggett, Brian L.","role":"aut","given":"Brian L.","family":"Claggett"},{"display":"Kulac, Ian","role":"aut","given":"Ian","family":"Kulac"},{"role":"aut","given":"Marcia","family":"Waddington Cruz","display":"Waddington Cruz, Marcia"},{"display":"Conceição, Isabel","role":"aut","given":"Isabel","family":"Conceição"},{"given":"Markus","role":"aut","family":"Weiler","display":"Weiler, Markus"},{"display":"Berk, John L.","role":"aut","given":"John L.","family":"Berk"},{"display":"Gertz, Morie","family":"Gertz","given":"Morie","role":"aut"},{"family":"Gillmore","given":"Julian D.","role":"aut","display":"Gillmore, Julian D."},{"display":"Rush, Stephen","role":"aut","given":"Stephen","family":"Rush"},{"family":"Chen","role":"aut","given":"Jersey","display":"Chen, Jersey"},{"display":"Zhou, Wunan","role":"aut","given":"Wunan","family":"Zhou"},{"role":"aut","given":"Jesse","family":"Kwoh","display":"Kwoh, Jesse"},{"display":"Duran, Jason M.","given":"Jason M.","role":"aut","family":"Duran"},{"family":"Tsimikas","given":"Sotirios","role":"aut","display":"Tsimikas, Sotirios"},{"family":"Solomon","role":"aut","given":"Scott D.","display":"Solomon, Scott D."}],"relHost":[{"physDesc":[{"extent":"Online-Ressource"}],"recId":"330078410","disp":"Effect of eplontersen on cardiac structure and function in patients with hereditary transthyretin amyloidosisJournal of cardiac failure","pubHistory":["1.1994 -"],"origin":[{"publisher":"Elsevier ; Harcourt ; Churchill Livingstone","dateIssuedKey":"1994","dateIssuedDisp":"1994-","publisherPlace":"New York, NY ; London ; New York"}],"note":["Gesehen am 25.10.21"],"language":["eng"],"type":{"media":"Online-Ressource","bibl":"periodical"},"part":{"pages":"973-980","text":"30(2024), 8 vom: Aug., Seite 973-980","extent":"8","issue":"8","year":"2024","volume":"30"},"title":[{"title":"Journal of cardiac failure","title_sort":"Journal of cardiac failure","subtitle":"official journal of the Heart Failure Society of America and the Japanese Heart Failure Society"}],"id":{"eki":["330078410"],"issn":["1532-8414"],"zdb":["2048826-9"]}}],"origin":[{"dateIssuedDisp":"August 2024","dateIssuedKey":"2024"}],"id":{"eki":["193015335X"],"doi":["10.1016/j.cardfail.2023.11.016"]},"name":{"displayForm":["Ahmad Masri, Mathew S. Maurer, Brian L. Claggett, Ian Kulac, Marcia Waddington Cruz, Isabel Conceição, Markus Weiler, John L. Berk, Morie Gertz, Julian D. Gillmore, Stephen Rush, Jersey Chen, Wunan Zhou, Jesse Kwoh, Jason M. Duran, Sotirios Tsimikas and Scott D. Solomon"]}} 
SRT |a MASRIAHMADEFFECTOFEP2024 

Similar Items