The purpose of this study is to:

  -  Describe epidemiological and clinical characteristics, natural history and
     real-world clinical management of ATTR amyloidosis patients

  -  Characterize the safety and effectiveness of patisiran and vutrisiran as part of
     routine clinical practice in the real-world clinical setting

  -  Describe disease emergence/progression in pre-symptomatic carriers of a known
     disease-causing transthyretin (TTR) variant

ConTTRibute: A Global Observational Study of Patients With Transthyretin (TTR)-Mediated Amyloidosis (ATTR Amyloidosis)

Transthyretin Amyloidosis (ATTR)

Patisiran

Clinical Trial Site

ConTTRibute: A Global Observational Multicenter Long-Term Study of Patients With Transthyretin (TTR)-Mediated Amyloidosis (ATTR Amyloidosis)

Selected events of interest are defined as hepatic events, cardiovascular events, renal
events, ocular events and infusion-related reactions, hypersensitivity, and other events
in patients diagnosed with hATTR amyloidosis.

PND Scores: Stage 0=No symptoms; Stage I=Sensory disturbances but preserved walking
capabilities; Stage II=Impaired walking capacity, but ability to walk without a stick or
crutches; Stage IIIA=Walking with help of 1 stick or crutch; Stage IIIB=Walking with the
help of 2 sticks or crutches; Stage IV=confined to wheel chair or bedridden.

FAP Scores: Stage 0=No symptoms; Stage I=Unimpaired ambulation; mostly mild sensory,
motor and autonomic neuropathy in the lower limbs; Stage II=Assistance with ambulation
required, mostly moderate impairment progression to the lower limbs, upper limbs, and
trunk; Stage III=Wheelchair-bound or bedridden; severe sensory, motor, and autonomic
involvement of all limbs.

NIS : 74 items, assess muscle weakness, reflexes and sensation; scored separately for
left, right limbs (37 items for each side). Components of muscle weakness (hip and knee
flexion, hip and knee extension, ankle dorsiflexors, ankle plantar flexors, toe
extensors, toe flexors) scored on scale 0 (normal) to 4 (paralysis), higher score=greater
weakness. Components of reflexes (quadriceps femoris, triceps surae) and sensation (touch
pressure, pin-prick, vibration, joint position) scored 0 = normal, 1= decreased, or 2 =
absent. Total possible NIS score range 0-244, higher score=greater impairment.

Cardiomyopathy will be assessed using New York Heart Association (NYHA) Class: I=No
symptoms; II=Symptoms with ordinary physical activity; III=Symptoms with less than
ordinary physical activity; IV=Symptoms at rest.

Norfolk-QoL-DN: The Norfolk QOL-DN questionnaire is a standardized 35-item
patient-reported outcomes measure that assesses 6 domains: physical function, large-fiber
neuropathy, activities of daily living, symptoms, small-fiber neuropathy, and autonomic
neuropathy. The total score ranges from -4 points (best possible quality of life) to 136
points (worst possible quality of life).

The KCCQ is a 23-item self-administered questionnaire developed to independently measure
the patient's perception of health status, which includes heart failure symptoms, impact
on physical and social function, and how their heart failure impacts their quality of
life within a 2-week recall period. The KCCQ quantifies 6 domains (symptoms, physical
function, quality of life, social limitation, self-efficacy, and symptom stability) and 2
summary scores (clinical and overall summary [OS] scores).

The R-ODS is a 24-item self-administered questionnaire for assessment of the disability a
patient experiences. It uses a linearly weighted categorical rating scale that
specifically captures domains of activity and social participation limitations in
patients.

Patients with a diagnosis of ATTR amyloidosis, hereditary or wild type, will be eligible
for the study and will follow routine clinical care.

Pre-symptomatic carriers with a known disease-causing TTR variant will be eligible for
the study and will follow routine clinical care.