Clinical trial NCT00628745: Transthyretin Amyloidosis Outcome Survey (THAOS)
Official title: Transthyretin Amyloidosis Outcomes Survey (THAOS): A Global, Multi-Center, Longitudinal, Observational Survey of Patients With Documented Transthyretin Gene Mutations or Wild-Type Transthyretin Amyloidosis.
Study type: OBSERVATIONAL
Overall status: COMPLETED
Conditions: Transthyretin Gene Mutations
Transthyretin Amyloidosis
Keywords: TRANSTHYRETIN
AMYLOIDOSIS
TRANSTHYRETIN AMYLOIDOSIS
Transthyretin amyloid cardiomyopathy
Lead sponsor: Pfizer
Sponsor class: INDUSTRY
First posted: 2008-03-05
Enrollment: 6718
Sex: ALL
Minimum age (years): 18
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Observational model: OTHER
Time perspective: PROSPECTIVE
Sampling method: NON_PROBABILITY_SAMPLE
Study population: The study population includes patients with confirmed hereditary or wild-type ATTR amyloidosis (inclusive of ATTR-PN and ATTR-CM) and those with TTR gene mutations without a diagnosis of ATTR amyloidosis.

Brief summary: THAOS is a global, multi-center, longitudinal observational survey open to all patients with transthyretin amyloidosis (ATTR), including ATTR-PN (polyneuropathy), ATTR-CM (cardiomyopathy) and wild-type ATTR-CM. It is open-ended with a minimum duration of 10 years. Patients will be followed as long as they are able to participate. The principal aims of this outcome survey are to better understand and characterize the natural history of the disease by studying a large and heterogenous patient population. Survey data may be used to develop new treatment guidelines and recommendations, and to inform and educate clinicians about the management of this disease.

Detailed description: n/a NA

Eligibility criteria: Inclusion Criteria: Patients must meet all of the following inclusion criteria to be eligible for inclusion into THAOS:

1. Evidence of a personally signed and dated informed consent document indicating that the participant (or a legally acceptable representative) has been informed of all pertinent aspects of the study.
2. Males and females greater than or equal to 18 years of age.
3. Confirmed genotyped TTR mutation with or without a diagnosis of hereditary or wild-type ATTR amyloidosis. Confirmation of ATTRwt amyloidosis will be determined by genotyped confirmation that patient does not possess a known mutation in TTR gene (ie, is a carrier of wild-type allele only) via genetic testing and one of the following set of criteria (a, b, or c):

   1. Presence of amyloid in cardiac biopsy tissue confirmed as TTR amyloid by mass spectrometry or immunohistochemistry; or
   2. Evidence of cardiac involvement by echocardiogram as defined by left ventricle wall thickness of \>12 mm, and presence of amyloid in non-cardiac tissue confirmed as TTR amyloid by mass spectrometry or immunohistochemistry; or
   3. Evidence of cardiac involvement by echocardiogram as defined by left ventricle wall thickness of \>12 mm, and presence of amyloid in cardiac tissue indirectly confirmed by scintigraphy with a "bone seeking tracer" eg, 99mTC-DPD \[99mTC-3,3-diphosphono-1,2-propano-dicarboxylic acid\], 99mTC- PYP \[Pyrophosphate\], and 99mTC-HMDP \[hydroxymethylene diphosphonate\] with Perugini grade greater than or equal to 2.

Exclusion Criteria

Patients meeting any of the following will not be included in the study:

1. Patient has evidence of primary (light chain) or secondary amyloidosis.

Primary outcomes:
measure: Number of Participants With Treatment-Emergent Adverse Events (AEs) and Serious Adverse Events (SAEs)
timeFrame: From the start of data collection at Baseline (Day 1) to the end of data collection (Up to 14.4 years)
measure: Number of Participants With Treatment Emergent Treatment Related AEs and SAEs
timeFrame: From the start of data collection at Baseline (Day 1) to the end of data collection (Up to 14.4 years)
measure: Number of All-Cause Deaths
timeFrame: From the start of data collection at Baseline (Day 1) to the end of data collection (Up to 14.4 years)
Secondary outcomes:
measure: Number of Participants With Modified Polyneuropathy Disability (mPND) Scores at Baseline
timeFrame: At the start of data collection at Baseline (Day 1)
measure: Number of Participants With Coutinho Disease Stages at Baseline
timeFrame: At the start of data collection at Baseline (Day 1)
measure: Number of Participants With Karnofsky Performance Index at Baseline
timeFrame: At the start of data collection at Baseline (Day 1)
measure: Number of Participants With Heart Failure at Baseline
timeFrame: At the start of data collection at Baseline (Day 1)
measure: Number of Participants With New York Heart Association (NYHA) Classifications at Baseline
timeFrame: At the start of data collection at Baseline (Day 1)
measure: Number of Participants Diagnosed With ATTR at Baseline
timeFrame: At the start of data collection at Baseline (Day 1)
measure: Number of Participants With Prior Misdiagnosis at Baseline
timeFrame: At the start of data collection at Baseline (Day 1)
measure: Number of Participants With ATTR Genotypes at Baseline
timeFrame: At the start of data collection at Baseline (Day 1)
measure: Number of Participants With Past or Current Clinical Trial Participation at Baseline
timeFrame: At the start of data collection at Baseline (Day 1)
measure: Number of Participants With Past or Current Tafamidis Compassionate Use/Early Access or Other Non-commercial Program at Baseline
timeFrame: At the start of data collection at Baseline (Day 1)
measure: Number of Participants With Known Family History of Symptomatic ATTR at Baseline
timeFrame: At the start of data collection at Baseline (Day 1)
measure: Number of Affected Generations at Baseline
timeFrame: At the start of data collection at Baseline (Day 1)
measure: Derived Neuropathy Impairment Score-Lower Limb (NIS-LL) at Baseline
timeFrame: At the start of data collection at Baseline (Day 1)
measure: Body Mass Index (BMI) at Baseline
timeFrame: At the start of data collection at Baseline (Day 1)
measure: Modified Body Mass Index (mBMI) at Baseline
timeFrame: At the start of data collection at Baseline (Day 1)
measure: Sitting Systolic and Diastolic Blood Pressures (SBP and DBP) at Baseline
timeFrame: At the start of data collection at Baseline (Day 1)
measure: Left Ventricular (LV) Septum Thickness at Baseline
timeFrame: At the start of data collection at Baseline (Day 1)
measure: Left Ventricular (LV) Ejection Fraction at Baseline
timeFrame: At the start of data collection at Baseline (Day 1)
measure: Euro Quality of Life-5 Dimensions-3 Level (EQ-5D-3L): Visual Analog Scale (VAS) Overall Health Score at Baseline
timeFrame: At the start of data collection at Baseline (Day 1)
measure: EQ-5D-3L: VAS Derived Index at Baseline
timeFrame: At the start of data collection at Baseline (Day 1)
measure: Norfolk Total Quality of Life (QoL) Score at Baseline
timeFrame: At the start of data collection at Baseline (Day 1)
measure: Number of Participants With Abnormal Electrocardiogram (ECG) at Baseline
timeFrame: At the start of data collection at Baseline (Day 1)
measure: Number of Participants With Atrial Fibrillation/Flutter, Pacemaker Implanted, and Implantable Cardioverter/Defibrillator (ICD) at Baseline
timeFrame: At the start of data collection at Baseline (Day 1)
Other outcomes:
Locations:
city: Birmingham
country: United States
facility: University of Alabama at Birmingham
state: Alabama
city: Phoenix
country: United States
facility: Mayo Clinic Arizona
state: Arizona
city: Los Angeles
country: United States
facility: VA Greater Los Angeles Healthcare System
state: California
city: Orange
country: United States
facility: University of California, Irvine
state: California
city: San Francisco
country: United States
facility: University of California - San Francisco, UCSF Department of Neurology
state: California
city: San Francisco
country: United States
facility: Office of Sponsored Research
state: California
city: Stanford
country: United States
facility: Stanford University School of Medicine
state: California
city: Aurora
country: United States
facility: UC Denver
state: Colorado
city: New Haven
country: United States
facility: Smilow Cancer Hospital at Yale-New Haven
state: Connecticut
city: Deerfield Beach
country: United States
facility: UHealth Deerfield Beach
state: Florida
city: Miami
country: United States
facility: University of Miami Hospital & Clinics
state: Florida
city: Plantation
country: United States
facility: UHealth Plantation
state: Florida
city: Chicago
country: United States
facility: Clinical Trials Unit
state: Illinois
city: Chicago
country: United States
facility: Northwestern University
state: Illinois
city: Chicago
country: United States
facility: University of Chicago Medical Center IRB
state: Illinois
city: Chicago
country: United States
facility: University of Chicago Medical Center
state: Illinois
city: Oak Lawn
country: United States
facility: Advocate Christ Medical Centre
state: Illinois
city: New Orleans
country: United States
facility: John Ochsner Heart & Vascular Institute
state: Louisiana
city: Baltimore
country: United States
facility: University of Maryland School of Medicine
state: Maryland
city: Baltimore
country: United States
facility: University of Maryland
state: Maryland
city: Baltimore
country: United States
facility: Johns Hopkins Hospital
state: Maryland
city: Boston
country: United States
facility: Harvard Vanguard Medical Associates
state: Massachusetts
city: Ann Arbor
country: United States
facility: University of Michigan
state: Michigan
city: Rochester
country: United States
facility: Mayo Clinic
state: Minnesota
city: Saint Louis
country: United States
facility: Washington University School of Medicine
state: Missouri
city: Bronx
country: United States
facility: Cardiology Clinic at Montefiore Hutchinson Campus
state: New York
city: Bronx
country: United States
facility: Montefiore Medical Center-Jack D. Weiler Hospital
state: New York
city: Bronx
country: United States
facility: Congestive Heart Failure Clinic
state: New York
city: Bronx
country: United States
facility: Montefiore Moses Division
state: New York
city: New York
country: United States
facility: NYU Medical Center
state: New York
city: New York
country: United States
facility: Columbia University Medical Center
state: New York
city: Cleveland
country: United States
facility: Cleveland Clinic Foundation
state: Ohio
city: Columbus
country: United States
facility: The Ohio University College of Medicine
state: Ohio
city: Columbus
country: United States
facility: OSU Wexner Medical Center
state: Ohio
city: Portland
country: United States
facility: Oregon Health and Science University
state: Oregon
city: McMurray
country: United States
facility: Peters Township Health and Wellness Pavillion
state: Pennsylvania
city: Philadelphia
country: United States
facility: Penn Presbyterian Medical Center-University of Pennsylvania
state: Pennsylvania
city: Philadelphia
country: United States
facility: University of Pennsylvania - Perelman Center for Advanced Medicine
state: Pennsylvania
city: Philadelphia
country: United States
facility: Temple University School of Medicine
state: Pennsylvania
city: Pittsburgh
country: United States
facility: Allegheny General Hospital
state: Pennsylvania
city: Pittsburgh
country: United States
facility: Admin
state: Pennsylvania
city: Pittsburgh
country: United States
facility: University of Pittsburgh Medical Center (UPMC)
state: Pennsylvania
city: Wexford
country: United States
facility: Wexford Health and Wellness Pavillion
state: Pennsylvania
city: Nashville
country: United States
facility: IRB
state: Tennessee
city: Nashville
country: United States
facility: Vanderbilt University School of Medicine
state: Tennessee
city: Salt Lake City
country: United States
facility: The University of Utah Health Sciences Center
state: Utah
city: Milwaukee
country: United States
facility: Medical College of Wisconsin
state: Wisconsin
city: Buenos Aires
country: Argentina
facility: Hospital Italiano de Buenos Aires (HIBA)
city: Ciudad Autonoma de Buenos aires
country: Argentina
facility: FLENI
city: Ciudad Autonoma De Buenos Aires
country: Argentina
facility: Instituto De Investigaciones Medicas Dr Alfredo Lanari
city: Leuven
country: Belgium
facility: Afdeling Klinische Cardiologie, O&N I
city: Rio de Janeiro
country: Brazil
facility: Hospital Universitário Clementino Fraga Filho -HUCFF Universidade Federal do Rio de Janeiro
city: Sao Paulo
country: Brazil
facility: Instituto Dante Pazzanese De Cardiologia
city: Sofia
country: Bulgaria
facility: Alexandrovska University Hospital Clinic of Neurology
city: Calgary
country: Canada
facility: University of Alberta Foothills Medical Centre
state: Alberta
city: Toronto
country: Canada
facility: Toronto General Hospital-University Health Network
state: Ontario
city: Toronto
country: Canada
facility: Toronto General Hospital
state: Ontario
city: Nicosia
country: Cyprus
facility: Cyprus Institute of Neurology and Genetics
city: Aarhus
country: Denmark
facility: Aarhus University Hospital, Skejby
city: Cedex
country: France
facility: CHU de Bicetre
city: Colmar
country: France
facility: Hopital Louis Pasteur
city: Créteil
country: France
facility: CHU Henri Mondor
city: Fort De France
country: France
facility: Chu De Fort De France
city: Lille
country: France
facility: CHRU de Lille, Hopital Claude Huriez
city: Lille
country: France
facility: Hopital Salengro - CHRU de Lille
city: Toulouse cedex 09
country: France
facility: CHU de Toulouse - Hopital Rangueil
city: Aachen
country: Germany
facility: University Hospital of RWTH Aachen
city: Berlin
country: Germany
facility: Charite CAmpus Rudolf-Virchow-Klinikum
city: Heidelberg
country: Germany
facility: Medical University of Heidelberg
city: Mainz
country: Germany
facility: Johann-Gutenberg-Universität
city: Mainz
country: Germany
facility: University Medical Center, Johannes Gutenberg-University Mainz
city: Mainz
country: Germany
facility: Universitätsmedizin der Johannes Gutenberg Universität Mainz KöR
city: Muenster
country: Germany
facility: Universitatsklinikum Muenster - Transplant Hepatology
city: Holon
country: Israel
facility: Wolfson Medical Center
city: Ramat Gan
country: Israel
facility: Sheba Medical Center
city: Bologna
country: Italy
facility: Comitato Etico Indipendente dell Azienda
city: Firenze
country: Italy
facility: Azienda Ospedaliero-Universitaria di Careggi
city: Messina
country: Italy
facility: AOU Policlinico G. Martino - Messina - Comitato Etico Scientifico
city: Messina
country: Italy
facility: AOU Policlinico G. Martino - Messina - Dr. Vita
city: Pavia
country: Italy
facility: Centro per lo Studio e la Cura delle Amiloidosi Sistemiche - Pavia - Prof. Merlini
city: Pavia
country: Italy
facility: Comitato di Bioetica della Fondazione IRCCS Policlinico S. Matteo
city: Pisa
country: Italy
facility: Fondazione Toscana Gabriele Monasterio Per La Ricerca Medica E Di Sanita Pubblica (Ftgm)
city: Roma
country: Italy
facility: Fondazione Policlinico Gemelli - Universita Cattolica del Sacro Cuore
city: Matsumoto
country: Japan
facility: Shinshu University School of Medicine
state: JP
city: Chiba
country: Japan
facility: Chiba University Hospital
city: Kumamoto
country: Japan
facility: Kumamoto University
city: Seoul
country: Korea, Republic of
facility: Samsung Medical Center, Sungkyunkwan University School of Medicine
city: Seoul
country: Korea, Republic of
facility: Konkuk University Medical Center
city: Kuala Lumpur
country: Malaysia
facility: University Malaya Medical Centre (UMMC)
state: Wilayah Persekutuan
city: Distrito Federal
country: Mexico
facility: Instituto Nacional de Ciencia Medicas y Nutricion Salvador Zubiran
city: Groningen
country: Netherlands
facility: University Medical Center Groningen
city: Guimaraes
country: Portugal
facility: Centro Hospitalar Do Alto Ave, Epe
city: Lisboa
country: Portugal
facility: Centro Hospitalar Lisboa Norte (CHLN) EPE - Hospital de Santa Maria
city: Lisboa
country: Portugal
facility: Hospital Santa Maria
city: Porto
country: Portugal
facility: Unidade Clinica de Paramiloidose-Centro Hospitalar Porto,EPE-Hospital Geral Santo Antonio
city: Bucuresti
country: Romania
facility: Institutul De Cardiologie Prof. Dr. C. C. Iliescu Spitalului Fundeni
city: Riyadh
country: Saudi Arabia
facility: King Faisal Specialist Hospital and Research Center
city: Gipuzkoa - SanSebastian
country: Spain
facility: Hospital Universitario Donostia
state: Donostia
city: Majadahonda
country: Spain
facility: Hospital Universitario Puerta de Hierro
state: Madrid
city: Barcelona
country: Spain
facility: Institut Clinic de Nefrologia i Urologia - ICNU, Hospital Clinic i Provincial de Barcelona
city: Barcelona
country: Spain
facility: Hospital Universitari de Bellvitge
city: Granada
country: Spain
facility: Hospital de Rehabilitacion y Traumatologia Virgen de las Nieves
city: Huelva
country: Spain
facility: Hospital Juan Ramon Jimenez
city: Madrid
country: Spain
facility: Hospital Gregorio Marañón
city: Madrid
country: Spain
facility: Hospital Clinico San Carlos
city: Palma de Mallorca
country: Spain
facility: Hospital Son Llàtzer
city: Piteå
country: Sweden
facility: Piteå Älvdals Hospital
city: Stockholm
country: Sweden
facility: Karolinska University Hospital, Huddinge
city: Umeå
country: Sweden
facility: Umeå University Hospital
city: Taipei
country: Taiwan
facility: National Taiwan University Hospital
city: Taipei
country: Taiwan
facility: National Taiwan University Hospital
city: Istanbul
country: Turkey
facility: Istanbul University,Istanbul Faculty of Medicine,Department of Neurology
city: Abu Dhabi
country: United Arab Emirates
facility: Cleveland Clinic Abu Dhabi LLC
Study officials:
affiliation: Pfizer
name: Pfizer CT.gov Call Center
role: STUDY_DIRECTOR
References:
citation: Coelho T, Conceicao I, Waddington-Cruz M, Keohane D, Sultan MB, Chapman D, Amass L; THAOS investigators. A natural history analysis of asymptomatic TTR gene carriers as they develop symptomatic transthyretin amyloidosis in the Transthyretin Amyloidosis Outcomes Survey (THAOS). Amyloid. 2022 Dec;29(4):228-236. doi: 10.1080/13506129.2022.2070470. Epub 2022 Jun 22.
citation: Dispenzieri A, Coelho T, Conceicao I, Waddington-Cruz M, Wixner J, Kristen AV, Rapezzi C, Plante-Bordeneuve V, Gonzalez-Moreno J, Maurer MS, Grogan M, Chapman D, Amass L; THAOS investigators. Clinical and genetic profile of patients enrolled in the Transthyretin Amyloidosis Outcomes Survey (THAOS): 14-year update. Orphanet J Rare Dis. 2022 Jun 18;17(1):236. doi: 10.1186/s13023-022-02359-w.
citation: Campbell CM, LoRusso S, Dispenzieri A, Kristen AV, Maurer MS, Rapezzi C, Lairez O, Drachman B, Garcia-Pavia P, Grogan M, Chapman D, Amass L; THAOS investigators. Sex Differences in Wild-Type Transthyretin Amyloidosis: An Analysis from the Transthyretin Amyloidosis Outcomes Survey (THAOS). Cardiol Ther. 2022 Sep;11(3):393-405. doi: 10.1007/s40119-022-00265-7. Epub 2022 May 18.
citation: Barroso FA, Coelho T, Dispenzieri A, Conceicao I, Waddington-Cruz M, Wixner J, Maurer MS, Rapezzi C, Plante-Bordeneuve V, Kristen AV, Gonzalez-Duarte A, Chapman D, Stewart M, Amass L; THAOS investigators. Characteristics of patients with autonomic dysfunction in the Transthyretin Amyloidosis Outcomes Survey (THAOS). Amyloid. 2022 Sep;29(3):175-183. doi: 10.1080/13506129.2022.2043270. Epub 2022 Apr 22.
citation: Nativi-Nicolau J, Siu A, Dispenzieri A, Maurer MS, Rapezzi C, Kristen AV, Garcia-Pavia P, LoRusso S, Waddington-Cruz M, Lairez O, Witteles R, Chapman D, Amass L, Grogan M; THAOS Investigators. Temporal Trends of Wild-Type Transthyretin Amyloid Cardiomyopathy in the Transthyretin Amyloidosis Outcomes Survey. JACC CardioOncol. 2021 Oct 19;3(4):537-546. doi: 10.1016/j.jaccao.2021.08.009. eCollection 2021 Oct.
citation: Gonzalez-Moreno J, Losada-Lopez I, Cisneros-Barroso E, Garcia-Pavia P, Gonzalez-Costello J, Munoz-Beamud F, Campistol JM, Fernandez-Torron R, Chapman D, Amass L. A Descriptive Analysis of ATTR Amyloidosis in Spain from the Transthyretin Amyloidosis Outcomes Survey. Neurol Ther. 2021 Dec;10(2):833-845. doi: 10.1007/s40120-021-00267-y. Epub 2021 Jul 30.
citation: Gentile L, Tournev I, Amass L, Chapman D, Mazzeo A; THAOS investigators. Phenotypic Differences of Glu89Gln Genotype in ATTR Amyloidosis From Endemic Loci: Update From THAOS. Cardiol Ther. 2021 Dec;10(2):481-490. doi: 10.1007/s40119-021-00226-6. Epub 2021 Jun 19.
citation: Waddington-Cruz M, Wixner J, Amass L, Kiszko J, Chapman D, Ando Y; THAOS investigators. Characteristics of Patients with Late- vs. Early-Onset Val30Met Transthyretin Amyloidosis from the Transthyretin Amyloidosis Outcomes Survey (THAOS). Neurol Ther. 2021 Dec;10(2):753-766. doi: 10.1007/s40120-021-00258-z. Epub 2021 May 22.
citation: Gonzalez-Duarte A, Barroso F, Mundayat R, Shapiro B. Blood pressure and orthostatic hypotension as measures of autonomic dysfunction in patients from the transthyretin amyloidosis outcomes survey (THAOS). Auton Neurosci. 2019 Dec;222:102590. doi: 10.1016/j.autneu.2019.102590. Epub 2019 Oct 23.
citation: Huber P, Flynn A, Sultan MB, Li H, Rill D, Ebede B, Gundapaneni B, Schwartz JH. A comprehensive safety profile of tafamidis in patients with transthyretin amyloid polyneuropathy. Amyloid. 2019 Dec;26(4):203-209. doi: 10.1080/13506129.2019.1643714. Epub 2019 Jul 27.
citation: Pinto MV, Pinto LF, Dias M, Rosa RS, Mundayat R, Pedrosa RC, Waddington-Cruz M. Late-onset hereditary ATTR V30M amyloidosis with polyneuropathy: Characterization of Brazilian subjects from the THAOS registry. J Neurol Sci. 2019 Aug 15;403:1-6. doi: 10.1016/j.jns.2019.05.030. Epub 2019 May 28.
citation: Damy T, Kristen AV, Suhr OB, Maurer MS, Plante-Bordeneuve V, Yu CR, Ong ML, Coelho T, Rapezzi C; THAOS Investigators. Transthyretin cardiac amyloidosis in continental Western Europe: an insight through the Transthyretin Amyloidosis Outcomes Survey (THAOS). Eur Heart J. 2022 Feb 3;43(5):391-400. doi: 10.1093/eurheartj/ehz173.
citation: Cruz MW, Pinto MV, Pinto LF, Gervais R, Dias M, Perez C, Mundayat R, Ong ML, Pedrosa RC, Foguel D. Baseline disease characteristics in Brazilian patients enrolled in Transthyretin Amyloidosis Outcome Survey (THAOS). Arq Neuropsiquiatr. 2019 Feb;77(2):96-100. doi: 10.1590/0004-282X20180156.
citation: Rubin J, Steidley DE, Carlsson M, Ong ML, Maurer MS. Myocardial Contraction Fraction by M-Mode Echocardiography Is Superior to Ejection Fraction in Predicting Mortality in Transthyretin Amyloidosis. J Card Fail. 2018 Aug;24(8):504-511. doi: 10.1016/j.cardfail.2018.07.001. Epub 2018 Aug 17.
citation: Mundayat R, Stewart M, Alvir J, Short S, Ong ML, Keohane D, Rill D, Sultan MB. Positive Effectiveness of Tafamidis in Delaying Disease Progression in Transthyretin Familial Amyloid Polyneuropathy up to 2 Years: An Analysis from the Transthyretin Amyloidosis Outcomes Survey (THAOS). Neurol Ther. 2018 Jun;7(1):87-101. doi: 10.1007/s40120-018-0097-9. Epub 2018 Apr 9.
citation: Kristen AV, Maurer MS, Rapezzi C, Mundayat R, Suhr OB, Damy T; THAOS investigators. Impact of genotype and phenotype on cardiac biomarkers in patients with transthyretin amyloidosis - Report from the Transthyretin Amyloidosis Outcome Survey (THAOS). PLoS One. 2017 Apr 6;12(4):e0173086. doi: 10.1371/journal.pone.0173086. eCollection 2017.
citation: Maurer MS, Hanna M, Grogan M, Dispenzieri A, Witteles R, Drachman B, Judge DP, Lenihan DJ, Gottlieb SS, Shah SJ, Steidley DE, Ventura H, Murali S, Silver MA, Jacoby D, Fedson S, Hummel SL, Kristen AV, Damy T, Plante-Bordeneuve V, Coelho T, Mundayat R, Suhr OB, Waddington Cruz M, Rapezzi C; THAOS Investigators. Genotype and Phenotype of Transthyretin Cardiac Amyloidosis: THAOS (Transthyretin Amyloid Outcome Survey). J Am Coll Cardiol. 2016 Jul 12;68(2):161-72. doi: 10.1016/j.jacc.2016.03.596.
citation: Damy T, Maurer MS, Rapezzi C, Plante-Bordeneuve V, Karayal ON, Mundayat R, Suhr OB, Kristen AV. Clinical, ECG and echocardiographic clues to the diagnosis of TTR-related cardiomyopathy. Open Heart. 2016 Feb 8;3(1):e000289. doi: 10.1136/openhrt-2015-000289. eCollection 2016.
citation: Coelho T, Maurer MS, Suhr OB. THAOS - The Transthyretin Amyloidosis Outcomes Survey: initial report on clinical manifestations in patients with hereditary and wild-type transthyretin amyloidosis. Curr Med Res Opin. 2013 Jan;29(1):63-76. doi: 10.1185/03007995.2012.754348. Epub 2012 Dec 13.