JACC: Advances (Aug 2024)
Age- and Sex-Related Differences in Patients With Wild-Type Transthyretin Amyloidosis
- Nerea Mora-Ayestaran, MD,
- Angela Dispenzieri, MD,
- Arnt V. Kristen, MD,
- Mathew S. Maurer, MD,
- Igor Diemberger, MD,
- Brian M. Drachman, MD,
- Martha Grogan, MD,
- Pritam Gupta, PhD,
- Oliver Glass, PhD,
- Leslie Amass, PhD,
- Pablo Garcia-Pavia, MD, PhD,
- Michele Emdin,
- Mazen Hanna,
- Olga Azevedo,
- Calogero Lino Cirami,
- Jose Gonzalez Costello,
- David Slosky,
- Henning Moelgaard,
- Jose Nativi Nicolau,
- Scott Hummel,
- Eun-Seok Jeon,
- Nowell Fine,
- Srinivas Murali,
- Edward Miller,
- Sanjiv Shah,
- Ronald Witteles,
- Marcia Waddington-Cruz,
- Daniel Lenihan,
- Yoshiki Sekijima,
- Johan Van Cleemput,
- Edileide de Barros Correia,
- Eve Cariou,
- Dianna Quan,
- Miriam Freimer,
- David Steidley,
- Anna Hüsing-Kabar,
- Violaine Plante-Bordeneuve,
- Hans Nienhuis,
- Jonas Wixner,
- Jeffrey Ralph,
- Hector Ventura,
- Sasa Zivkovic,
- Diego Delgado,
- Roberto Fernandéz Torrón,
- Stephen Gottlieb,
- William Cotts,
- Jose Tallaj,
- Robert Brunkhorst,
- Michael Polydefkis,
- Christopher Mueller,
- Carsten Tschoepe,
- Juan Gonzalez Moreno,
- Nitasha Sarswat,
- Jin Luo,
- James Tauras,
- Alberta Warner
Affiliations
- Nerea Mora-Ayestaran, MD
- Heart Failure and Inherited Cardiac Diseases Unit, Department of Cardiology, Hospital, Universitario Puerta de Hierro, IDIPHISA, CIBERCV, Madrid, Spain
- Angela Dispenzieri, MD
- Division of Hematology, Mayo Clinic, Rochester, Minnesota, USA
- Arnt V. Kristen, MD
- Department of Cardiology, Angiology, and Respiratory Medicine, Medical University of Heidelberg, Heidelberg, Germany
- Mathew S. Maurer, MD
- Division of Cardiology, Columbia University College of Physicians and Surgeons, New York, New York, USA
- Igor Diemberger, MD
- Department of Medical and Surgical Sciences, DIMEC, University of Bologna, Bologna, Italy; Cardiology Unit, IRCCS Policlinico di S. Orsola, Bologna, Italy
- Brian M. Drachman, MD
- Division of Cardiology, University of Pennsylvania Health System, Philadelphia, Pennsylvania, USA
- Martha Grogan, MD
- Department of Cardiovascular Diseases, Mayo Clinic, Rochester, Minnesota, USA
- Pritam Gupta, PhD
- Pfizer Healthcare India Pvt. Ltd, Chennai, India
- Oliver Glass, PhD
- Pfizer Inc, New York, New York, USA
- Leslie Amass, PhD
- Pfizer Inc, New York, New York, USA
- Pablo Garcia-Pavia, MD, PhD
- Heart Failure and Inherited Cardiac Diseases Unit, Department of Cardiology, Hospital, Universitario Puerta de Hierro, IDIPHISA, CIBERCV, Madrid, Spain; Universidad Francisco de Vitoria (UFV), Pozuelo de Alarcon, Spain; Centro Nacional de Investigaciones Cardiovasculares (CNIC), Madrid, Spain; Address for correspondence: Dr Pablo Garcia-Pavia, Heart Failure and Inherited Cardiac Diseases Unit, Department of Cardiology, Hospital Universitario Puerta de Hierro Majadahonda, Manuel de Falla, 1, Majadahonda, Madrid 28222, Spain.
- Michele Emdin
- Mazen Hanna
- Olga Azevedo
- Calogero Lino Cirami
- Jose Gonzalez Costello
- David Slosky
- Henning Moelgaard
- Jose Nativi Nicolau
- Scott Hummel
- Eun-Seok Jeon
- Nowell Fine
- Srinivas Murali
- Edward Miller
- Sanjiv Shah
- Ronald Witteles
- Marcia Waddington-Cruz
- Daniel Lenihan
- Yoshiki Sekijima
- Johan Van Cleemput
- Edileide de Barros Correia
- Eve Cariou
- Dianna Quan
- Miriam Freimer
- David Steidley
- Anna Hüsing-Kabar
- Violaine Plante-Bordeneuve
- Hans Nienhuis
- Jonas Wixner
- Jeffrey Ralph
- Hector Ventura
- Sasa Zivkovic
- Diego Delgado
- Roberto Fernandéz Torrón
- Stephen Gottlieb
- William Cotts
- Jose Tallaj
- Robert Brunkhorst
- Michael Polydefkis
- Christopher Mueller
- Carsten Tschoepe
- Juan Gonzalez Moreno
- Nitasha Sarswat
- Jin Luo
- James Tauras
- Alberta Warner
- Journal volume & issue
-
Vol. 3,
no. 8
p. 101086
Abstract
Background: Wild-type transthyretin amyloidosis (ATTRwt amyloidosis) is primarily diagnosed in elderly men but diagnoses in younger patients and women have recently increased. Objectives: The purpose of this study was to examine age- and sex-related differences in patients with ATTRwt amyloidosis enrolled in the THAOS (Transthyretin Amyloidosis Outcomes Survey). Methods: THAOS was a global, longitudinal, observational survey of patients with transthyretin amyloidosis, including both hereditary and wild-type disease, and asymptomatic carriers of pathogenic transthyretin gene variants. Patient characteristics at enrollment were analyzed by age at enrollment and sex (data cutoff date: August 1, 2022). Results: Of 1,251 patients with ATTRwt amyloidosis, 13.7%, 49.1%, 34.5%, and 2.8% were aged <70 years, 70 to 79 years, 80 to 89 years, and ≥90 years, respectively. The proportion of women increased with age, from 4.1% in patients aged <70 years to 14.3% in patients aged ≥90 years. In the respective age groups, median time from symptom onset to diagnosis overall (male, female) was 1.7 (1.3, 5.2), 2.0 (2.0, 2.2), 1.8 (1.9, 0.8), and 0.7 (0.6, 2.5) years. A Karnofsky Performance Status score ≤70 was observed in 17.1%, 30.1%, 46.1%, and 44.4% of patients aged <70 years, 70 to 79 years, 80 to 89 years, and ≥90 years, respectively. Conclusions: In this THAOS analysis of patients with ATTRwt amyloidosis, patients were diagnosed an average of 2 years after symptom onset, with the greatest diagnostic delay in women aged <70 years at 5 years. Patients were predominantly men, but the proportion of women increased with age. A substantial proportion of patients had significant functional impairment regardless of age. (Transthyretin Amyloidosis Outcome Survey [THAOS]; NCT00628745)
