Age- and Sex-Related Differences in Patients With Wild-Type Transthyretin Amyloidosis
oleh: 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
| Format: | Article |
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| Diterbitkan: | Elsevier 2024-08-01 |
Deskripsi
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)