dc.contributor.author | Gonzalez-Lopez, Esther | |
dc.contributor.author | Escobar-Lopez, Luis | |
dc.contributor.author | Obici, Laura | |
dc.contributor.author | Saturi, Giulia | |
dc.contributor.author | Bezard, Mélanie | |
dc.contributor.author | Saith, Sunil E | |
dc.contributor.author | AbouEzzeddine, Omar F | |
dc.contributor.author | Mussinelli, Roberta | |
dc.contributor.author | Gagliardi, Christian | |
dc.contributor.author | Kharoubi, Mounira | |
dc.contributor.author | Griffin, Jan M | |
dc.contributor.author | Dispenzieri, Angela | |
dc.contributor.author | Vilches, Silvia | |
dc.contributor.author | Perlini, Stefano | |
dc.contributor.author | Longhi, Simone | |
dc.contributor.author | Oghina, Silvia | |
dc.contributor.author | Rivas, Adrian | |
dc.contributor.author | Grogan, Martha | |
dc.contributor.author | Maurer, Mathew S | |
dc.contributor.author | Damy, Thibaud | |
dc.contributor.author | Palladini, Giovanni | |
dc.contributor.author | Rapezzi, Claudio | |
dc.contributor.author | Garcia-Pavia, Pablo | |
dc.date.accessioned | 2023-09-15T11:39:52Z | |
dc.date.available | 2023-09-15T11:39:52Z | |
dc.date.issued | 2022-11 | |
dc.identifier.citation | JACC CardioOncol. 2022 Nov 15;4(4):442-454. | es_ES |
dc.identifier.uri | http://hdl.handle.net/20.500.12105/16472 | |
dc.description.abstract | BACKGROUND
Transthyretin amyloid cardiomyopathy (ATTR-CM) is increasingly recognized as a treatable cause of heart failure (HF). Advances in diagnosis and therapy have increased the number of patients diagnosed at early stages, but prognostic data on patients without HF symptoms are lacking. Moreover, it is unknown whether asymptomatic patients benefit from early initiation of transthyretin (TTR) stabilizers.
OBJECTIVES
The aim of this study was to describe the natural history and prognosis of ATTR-CM in patients without HF symptoms.
METHODS
Clinical characteristics and outcomes of patients with ATTR-CM without HF symptoms were retrospectively collected at 6 international amyloidosis centers.
RESULTS
A total of 118 patients (78.8% men, median age 66 years [IQR: 53.8-75 years], 68 [57.6%] with variant transthyretin amyloidosis, mean left ventricular ejection fraction 60.5% ± 9.9%, mean left ventricular wall thickness 15.4 ± 3.1 mm, and 53 [45%] treated with TTR stabilizers at baseline or during follow-up) were included. During a median follow-up period of 3.7 years (IQR: 1-6 years), 38 patients developed HF symptoms (23 New York Heart Association functional class II and 14 functional class III or IV), 32 died, and 2 required cardiac transplantation. Additionally, 20 patients received pacemakers, 13 developed AF, and 1 had a stroke. Overall survival was 96.5% (95% CI: 91%-99%), 90.4% (95% CI: 82%-95%), and 82% (95% CI: 71%-89%) at 1, 3, and 5 years, respectively. Treatment with TTR stabilizers was associated with improved survival (HR: 0.31; 95% CI: 0.12-0.82; P = 0.019) and remained significant after adjusting for sex, age, ATTR-CM type, and estimated glomerular filtration rate (HR: 0.18; 95% CI: 0.06-0.55; P = 0.002).
CONCLUSIONS
After a median follow-up period of 3.7 years, 1 in 3 patients with asymptomatic ATTR-CM developed HF symptoms, and nearly as many died or required cardiac transplantation. Treatment with TTR stabilizers was associated with improved prognosis. | es_ES |
dc.description.sponsorship | This work was supported by grants from Instituto de Salud Carlos III
(PI18/0765 and PI20/01379). Dr Gonzalez-Lopez has received speaker
fees from Pfizer and Alnylam; has received consulting fees from Pfizer
and Proclara; and has received research and educational support to
her institution from Pfizer, BridgeBio, and Alnylam. Dr Obici has
received speaker and consulting fees from Pfizer, Alnylam, and
Akcea. Dr AbouEzzeddine has received research grant support from
Pfizer. Dr Mussinelli has received speaker fees from Pfizer and Akcea.
Dr Dispenzieri has received consulting fees from Janssen and Akcea;
and has received research support from Pfizer, Alnylam, Celgene, and
Takeda. Dr Perlini has received speaker and consulting fees from
Pfizer, Alnylam, and Akcea. Dr Palladini has received speaker fees
from Janssen-Cilag, Pfizer, and Siemens; and has participated on an
advisory board for Janssen Cilag. Dr Damy has received research
grants or consulting fees from Alnylam, Akcea, Pfizer, and Prothena.
Dr Grogan has received research grant support and consulting fees to
her institution from Alnylam, Eidos, Pfizer, and Prothena. Dr Maurer
has received grant support from National Institutes of Health
(R01HL139671-01, R21AG058348, and K24AG036778); has received
consulting income from Pfizer, GlaxoSmithKline, Eidos, Prothena,
Akcea, and Alnylam; and has received clinical trial funding to his
institution from Pfizer, Prothena, Eidos, and Alnylam. Dr Garcia-Pavia
has received speaker fees from Pfizer, BridgeBio, Alnylam, and Ionis;
has received consulting fees from Pfizer, BridgeBio, AstraZeneca,
NovoNordisk, Neuroimmune, Alnylam, Alexion, and Attralus; and has
received research and educational support to his institution from
Pfizer, BridgeBio, and Alnylam. All other authors have reported that
they have no relationships relevant to the contents of this paper to
disclose. | es_ES |
dc.language.iso | eng | es_ES |
dc.publisher | Elsevier | es_ES |
dc.type.hasVersion | VoR | es_ES |
dc.rights.uri | http://creativecommons.org/licenses/by/4.0/ | * |
dc.title | Prognosis of Transthyretin Cardiac Amyloidosis Without Heart Failure Symptoms. | es_ES |
dc.type | journal article | es_ES |
dc.rights.license | Atribución 4.0 Internacional | * |
dc.identifier.pubmedID | 36444226 | es_ES |
dc.format.volume | 4 | es_ES |
dc.format.number | 4 | es_ES |
dc.format.page | 442 | es_ES |
dc.identifier.doi | 10.1016/j.jaccao.2022.07.007 | es_ES |
dc.contributor.funder | Instituto de Salud Carlos III | es_ES |
dc.contributor.funder | Pfizer | es_ES |
dc.contributor.funder | Akcea Therapeutics | es_ES |
dc.contributor.funder | Janssen Cilag | es_ES |
dc.contributor.funder | National Institutes of Health (Estados Unidos) | es_ES |
dc.description.peerreviewed | Sí | es_ES |
dc.identifier.e-issn | 2666-0873 | es_ES |
dc.relation.publisherversion | 10.1016/j.jaccao.2022.07.007 | es_ES |
dc.identifier.journal | JACC. CardioOncology | es_ES |
dc.repisalud.orgCNIC | CNIC::Grupos de investigación::Miocardiopatías Hereditarias | es_ES |
dc.repisalud.institucion | CNIC | es_ES |
dc.rights.accessRights | open access | es_ES |
dc.relation.projectFECYT | info:eu-repo/grantAgreement/ES/PI18/0765 | es_ES |
dc.relation.projectFECYT | info:eu-repo/grantAgreement/ES/PI20/01379 | es_ES |