Cardiac Transplantation in Danon Disease.
J Card Fail
; 28(4): 664-669, 2022 04.
Article
em En
| MEDLINE
| ID: mdl-34775111
ABSTRACT
BACKGROUND:
Danon disease (DD) is a rare X-linked dominant cardioskeletal myopathy caused by mutations in the lysosome-associated membrane protein-2 (LAMP-2) gene that is usually lethal without cardiac transplantation. The purpose of this study was to characterize post-transplant outcomes in a large cohort of patients with DD who underwent cardiac transplantation.METHODS:
The clinical phenotype and outcome data of patients with DD who underwent cardiac transplantation (nâ¯=â¯38; 19 males and 19 females) were obtained from 8 centers. Study outcomes included graft survival, defined as death or retransplantation, and episodes of acute cellular and antibody-mediated rejection and cardiac allograft vasculopathy at 1 year.RESULTS:
Median follow-up time after transplantation for the entire cohort was 4.4 years (IQR 1.5-12.8 years). The median age at transplant for the cohort was 20.2 years (15.8-27.9 years), with no difference in age between sexes. Median pretransplant left-ventricular ejection fraction for the entire cohort was 30% (range 11%-84%). Males had higher pretransplant aspartate aminotransferase, alanine aminotransferase and creatine phosphokinase levels than females (P < 0.001). There were 2 deaths in the entire cohort and 2 retransplants. There was no difference in actuarial graft survival between males and females (Pâ¯=â¯0.8965); the estimated graft survival was 87.1% (95%CI 63.6%-95.9%) at 5 years. One episode (2.7%) of antibody-mediated rejection, grade 2, and 7 episodes (19%) of acute cellular rejection, grade 2 or 3, were reported in patients who survived to discharge (6 females and 1 male; Pâ¯=â¯0.172).CONCLUSIONS:
Heart transplantation outcomes are acceptable in DD with high probabilities of 5-year graft survival for males and females suggesting that cardiac transplantation is an effective treatment option for DD patients.Palavras-chave
Texto completo:
1
Coleções:
01-internacional
Base de dados:
MEDLINE
Assunto principal:
Transplante de Coração
/
Doença de Depósito de Glicogênio Tipo IIb
/
Insuficiência Cardíaca
Tipo de estudo:
Diagnostic_studies
/
Observational_studies
Limite:
Female
/
Humans
/
Male
Idioma:
En
Revista:
J Card Fail
Assunto da revista:
CARDIOLOGIA
Ano de publicação:
2022
Tipo de documento:
Article