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
Diagnosis of ATTR amyloidosis or documented known disease-causing TTR variant for the cohort of pre-symptomatic carriers
Germany Only: Patients must be treated per the summary of product characteristics (SmPC) for any approved treatment for ATTR amyloidosis
Current enrollment in a clinical trial for any investigational agent
Patients with a diagnosis of ATTR amyloidosis, hereditary or wild type, and pre-symptomatic carriers with a known disease-causing TTR variant will be eligible for the study.
Cohort
Prospective
Not listed
Incidence of Adverse Events
Selected Events of Interest in Patients with Hereditary Transthyretin-mediated (hATTR) or Wild-type Transthyretin-mediated (wtATTR) Amyloidosis (ATTRv 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 or wtATTR amyloidosis.
Health Care Provider (HCP)-Assessed Polyneuropathy (PND) Disability Score
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.
HCP-Assessed Familial Amyloidotic Polyneuropathy (FAP) Score
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.
Click on the lead sponsor to view all of their trials.
