Real-World Effectiveness of High-Dose Tafamidis on Neurologic Disease Progression in Mixed-Phenotype Transthyretin Amyloid Cardiomyopathy (ATTR-CM)

B3461121

This study will examine the clinical effectiveness of Tafamidis in patients with Mixed Phenotype Transthyretin Amyloidosis using data that already exist in patients' medical records

2024-04-29

2024-06-30

2024-06-30

Not yet recruiting

50

Inclusion Criteria: * Age ≥18 years at diagnosis. * Diagnosed with ATTRv-CM or ATTRwt-CM, mixed phenotype. * Treated with tafamidis, as VYNDAMAX 61 mg \[one 61-mg tafamidis capsule\] orally once daily for ≥12 months. * Have had ≥1 pre- and ≥1 post-treatment neurologic assessments. Exclusion Criteria: * History of any organ transplant. * Individuals who are non-ambulatory. * Prior or current treatment with any disease-modifying therapy (investigational or approved) alone or in combination, except tafamidis, as VYNDAQEL 80 mg \[four 20-mg tafamidis meglumine capsules\] orally once daily or VYNDAMAX 61 mg \[one 61-mg tafamidis capsule\] orally once daily. * Peripheral neuropathy attributed to causes other than ATTR amyloidosis (eg, diabetes mellitus, B12 deficiency, hypothyroidism, shingles,Lyme disease, HIV infection, secondary to injury, chronic kidney disease). * Patient's data fails to pass data quality checks.

{'studyType': 'OBSERVATIONAL', 'patientRegistry': False, 'designInfo': {'observationalModel': 'COHORT', 'timePerspective': 'RETROSPECTIVE'}, 'enrollmentInfo': {'count': 50, 'type': 'ESTIMATED'}}

2024-05-01