Temporal Trends of Wild-Type Transthyretin Amyloid Cardiomyopathy in the Transthyretin Amyloidosis Outcomes Survey
José Nativi-Nicolau, Alfonso Siu, Angela Dispenzieri, Mathew S. Maurer, Claudio Rapezzi, Arnt V. Kristen, Pablo García‐Pavía, Samantha LoRusso, Márcia Waddington‐Cruz, Olivier Lairez, Ronald Witteles, Doug Chapman, Leslie Amass, Martha Grogan, Fábio Barroso, Johan Van Cleemput, Nowell M. Fine, Hartmut Schmidt, Burkhard Gess, H. Moelgaard, Violaine Planté‐Bordeneuve, David Adams, Jocelyn Inamo, Giuseppe Vita, Calogero Lino Cirami, Marco Luigetti, Michele Emdin, Yoshiki Sekijima, Taro Yamashita, Eun‐Seok Jeon, Maria Alejandra Gonzalez Duarte Briseno, Hans Nienhuis, Olga Azevedo, Josep Maria Campistol Plana, Juan González Moreno, José González‐Costello, Jonas Wixner, Yeşim Parman, Sanjiv J. Shah, Dianna Quan, Tessa Marburger, Michael Polydefkis, Stephen S. Gottlieb, Jeffrey Ralph, Nitasha Sarswat, Jin Luo, Srinivas Murali, William Cotts, Brian Drachman, D. Eric Steidley, Scott L. Hummel, David Slosky, Hector Ventura, Daniel Jacoby, James E. Hoffman, James M. Tauras, Saša Živković, José Tallaj, Daniel J. Lenihan, Christopher R. Mueller
Abstract
BACKGROUND: Transthyretin amyloid cardiomyopathy results from the accumulation of wild-type (ATTRwt) or variant (ATTRv) transthyretin amyloid fibrils in the myocardium. THAOS (Transthyretin Amyloidosis Outcomes Survey) is a global, longitudinal, observational survey of patients with ATTRv and ATTRwt amyloidosis and asymptomatic patients with transthyretin mutations. OBJECTIVES: This study explored temporal trends in ATTRwt amyloidosis diagnoses using data from THAOS. METHODS: Using THAOS data from December 2007 to January 2020, the following comparisons were made according to year: ATTRwt amyloidosis diagnoses in the United States versus rest of the world, ATTRwt versus ATTRv amyloidosis with cardiac-associated mutations diagnoses, and ATTRwt amyloidosis diagnoses by tissue biopsy versus bone scintigraphy. RESULTS: There were 1,069 patients with ATTRwt amyloidosis and 525 with ATTRv amyloidosis with cardiac mutations enrolled in THAOS. The median time from symptom onset to ATTRwt amyloidosis diagnosis did not change over the past 5 years (>60 months from 2015-2019). ATTRwt amyloidosis diagnoses increased from 2 in 2005 to >100 per year from 2016, with a more pronounced increase in the United States compared with the rest of the world. Diagnoses of ATTRwt amyloidosis by tissue biopsy increased yearly and peaked in 2014 before declining, whereas diagnoses by bone scintigraphy increased markedly since 2011. ATTRv amyloidosis with cardiac mutation diagnoses increased from 3 in 2005 to 37 in 2011, then plateaued. The proportion of patients with ATTRwt amyloidosis diagnosed with New York Heart Association functional class III/IV heart failure decreased from 2012 (46.4%) to 2019 (16.0%). CONCLUSIONS: In the past decade, ATTRwt amyloidosis diagnoses increased worldwide. Despite the growing utilization of bone scintigraphy, patients are diagnosed several years after symptom onset. (Transthyretin Amyloidosis Outcomes Survey [THAOS]; NCT00628745).