TY - JOUR
T1 - Sex Differences in Wild-Type Transthyretin Amyloidosis
T2 - An Analysis from the Transthyretin Amyloidosis Outcomes Survey (THAOS)
AU - The THAOS investigators
AU - Campbell, Courtney M.
AU - LoRusso, Samantha
AU - Dispenzieri, Angela
AU - Kristen, Arnt V.
AU - Maurer, Mathew S.
AU - Rapezzi, Claudio
AU - Lairez, Olivier
AU - Drachman, Brian
AU - Garcia-Pavia, Pablo
AU - Grogan, Martha
AU - Chapman, Doug
AU - Amass, Leslie
AU - Emdin, Michele
AU - Hanna, Mazen
AU - Azevedo, Olga
AU - Cirami, Calogero Lino
AU - Jacoby, Daniel
AU - Costello, Jose Gonzalez
AU - Slosky, David
AU - Moelgaard, Henning
AU - Hummel, Scott
AU - Nativi-Nicolau, Jose
AU - Murali, Srinivas
AU - Fine, Nowell
AU - Jeon, Eun Seok
AU - Shah, Sanjiv
AU - Witteles, Ronald
AU - Lenihan, Daniel
AU - Waddington-Cruz, Marcia
AU - Sekijima, Yoshiki
AU - Tallaj, Jose
AU - Mueller, Christopher
AU - Van Cleemput, Johan
AU - Planté-Bordeneuve, Violaine
AU - Nienhuis, Hans
AU - Quan, Dianna
AU - Steidley, David
AU - Schmidt, Hartmut
AU - Wixner, Jonas
AU - Polydefkis, Michael
AU - Ralph, Jeffrey
AU - Ventura, Hector
AU - Zivkovic, Sasa
AU - Gess, Burkhard
AU - Torrón, Roberto Fernandéz
AU - Gottlieb, Stephen
AU - Cotts, William
AU - Tauras, James
AU - Sarswat, Nitasha
AU - Moreno, Juan González
N1 - Publisher Copyright:
© 2022, The Author(s).
PY - 2022/9
Y1 - 2022/9
N2 - Introduction: Wild-type transthyretin amyloidosis (ATTRwt amyloidosis) is a progressive disease resulting from the accumulation of wild-type transthyretin (TTR) amyloid fibrils, and is diagnosed primarily in males. This analysis examined sex differences in patients with ATTRwt amyloidosis from the Transthyretin Amyloidosis Outcomes Survey (THAOS). Methods: THAOS is an ongoing, global, longitudinal, observational survey of patients with transthyretin amyloidosis, including both inherited and wild-type disease, and asymptomatic carriers of TTR mutations. THAOS data were analyzed to identify potential differences in demographic and clinical characteristics between males and females with ATTRwt amyloidosis (data cutoff: August 1, 2021). Results: Of 1386 patients with ATTRwt amyloidosis, 84 (6%) were female and 1302 (94%) were male. Females had a higher median age at enrollment (80 vs. 78 years; p = 0.002) and symptom onset (75 vs. 73 years; p = 0.045) than males. Mean left ventricular (LV) ejection fraction was higher (53% vs. 48%; p = 0.001) and mean LV diastolic diameter lower (42 vs. 46 mm; p < 0.001) in females versus males, but sex was not identified as a predictor of LV mean wall thickness adjusted for height (beta coefficient − 0.22; p = 0.460) or a predominantly cardiac phenotype (odds ratio 1.60; p = 0.191). Modified polyneuropathy disability scores differed between groups (p < 0.001), with a larger proportion of scores ≥ IIIa among females (23% vs. 7%). Conclusions: Females with ATTRwt amyloidosis in THAOS tended to present at a later age and showed signs of less severe cardiac impairment and more severe walking impairment. Trial Registration: ClinicalTrials.gov: NCT00628745.
AB - Introduction: Wild-type transthyretin amyloidosis (ATTRwt amyloidosis) is a progressive disease resulting from the accumulation of wild-type transthyretin (TTR) amyloid fibrils, and is diagnosed primarily in males. This analysis examined sex differences in patients with ATTRwt amyloidosis from the Transthyretin Amyloidosis Outcomes Survey (THAOS). Methods: THAOS is an ongoing, global, longitudinal, observational survey of patients with transthyretin amyloidosis, including both inherited and wild-type disease, and asymptomatic carriers of TTR mutations. THAOS data were analyzed to identify potential differences in demographic and clinical characteristics between males and females with ATTRwt amyloidosis (data cutoff: August 1, 2021). Results: Of 1386 patients with ATTRwt amyloidosis, 84 (6%) were female and 1302 (94%) were male. Females had a higher median age at enrollment (80 vs. 78 years; p = 0.002) and symptom onset (75 vs. 73 years; p = 0.045) than males. Mean left ventricular (LV) ejection fraction was higher (53% vs. 48%; p = 0.001) and mean LV diastolic diameter lower (42 vs. 46 mm; p < 0.001) in females versus males, but sex was not identified as a predictor of LV mean wall thickness adjusted for height (beta coefficient − 0.22; p = 0.460) or a predominantly cardiac phenotype (odds ratio 1.60; p = 0.191). Modified polyneuropathy disability scores differed between groups (p < 0.001), with a larger proportion of scores ≥ IIIa among females (23% vs. 7%). Conclusions: Females with ATTRwt amyloidosis in THAOS tended to present at a later age and showed signs of less severe cardiac impairment and more severe walking impairment. Trial Registration: ClinicalTrials.gov: NCT00628745.
KW - ATTRwt amyloidosis
KW - Registry
KW - Sex
KW - Transthyretin Amyloidosis Outcomes Survey
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U2 - 10.1007/s40119-022-00265-7
DO - 10.1007/s40119-022-00265-7
M3 - Article
AN - SCOPUS:85131873620
SN - 2193-8261
VL - 11
SP - 393
EP - 405
JO - Cardiology and Therapy
JF - Cardiology and Therapy
IS - 3
ER -