Skip to Main Content (Press Enter)

Logo UNIME
  • ×
  • Home
  • Corsi
  • Insegnamenti
  • Professioni
  • Persone
  • Pubblicazioni
  • Strutture
  • Terza Missione
  • Competenze

Competenze e Professionalità
Logo UNIME

|

UNIFIND - Competenze e Professionalità

unime.it
  • ×
  • Home
  • Corsi
  • Insegnamenti
  • Professioni
  • Persone
  • Pubblicazioni
  • Strutture
  • Terza Missione
  • Competenze
  1. Persone

Use of Drugs for ATTRv Amyloidosis in the Real World: How Therapy Is Changing Survival in a Non-Endemic Area

Articolo
Data di Pubblicazione:
2021
Abstract:
Background: Over the past decade, three new drugs have been approved for the treatment of hereditary amyloid transthyretin (ATTRv) polyneuropathy. The aim of this work was to analyze whether current therapies prolong survival for patients affected by ATTRv amyloidosis. Methods: The study was conducted retrospectively, analyzing the medical records of 105 patients with genetic diagnoses of familial amyloidotic polyneuropathy followed at the two referral centers for the disease in Sicily, Italy. Of these, 71 received disease-modifying therapy, while 34 received only symptomatic treatment or no therapy. Results: The most used treatment in our patient cohort was tafamidis, followed by liver transplantation, patisiran, inotersen, and diflunisal. The median survival was significantly longer for treated vs. untreated patients (12 years vs. 8 years). In the 71 patients who received disease-modifying treatment, the presence of cardiac involvement, weight loss, or autonomic dysfunction at diagnosis was not related to survival. Conversely, patients diagnosed in the early stage of the disease (PND 1) had significantly longer survival than those diagnosed in the late stage (PND 2–4).
Tipologia CRIS:
14.a.1 Articolo su rivista
Keywords:
ATTRv; hereditary transthyretin amyloidosis; inotersen; non-V30M; patisiran; polyneuropathy; survival; tafamidis; patisiran; inotersen
Elenco autori:
Russo, Massimo; Gentile, Luca; Di Stefano, Vincenzo; Di Bella, Gianluca; Minutoli, Fabio; Toscano, Antonio; Brighina, Filippo; Vita, Giuseppe; Mazzeo, Anna
Autori di Ateneo:
DI BELLA Gianluca
GENTILE Luca
MAZZEO Anna
MINUTOLI Fabio
RUSSO Massimo
TOSCANO Antonio
VITA Giuseppe
Link alla scheda completa:
https://iris.unime.it/handle/11570/3203582
Pubblicato in:
BRAIN SCIENCES
Journal
  • Dati Generali

Dati Generali

URL

https://www.mdpi.com/2076-3425/11/5/545
  • Informazioni
  • Assistenza
  • Accessibilità
  • Privacy
  • Utilizzo dei cookie
  • Note legali

Realizzato con VIVO | Designed by Cineca | 25.10.4.0