Transthyretin Amyloidosis Outcome Survey (THAOS)
Transthyretin Amyloidosis Outcome Survey (THAOS)
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.
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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.