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Tafamidis, sold under the brand names Vyndaqel and Vyndamax, is a medication used to delay disease progression in adults with certain forms of transthyretin amyloidosis. It can be used to treat both hereditary forms, familial amyloid cardiomyopathy and familial amyloid polyneuropathy, as well as wild-type transthyretin amyloidosis, which formerly was called senile systemic amyloidosis. It works by stabilizing the quaternary structure of the protein transthyretin. In people with transthyretin amyloidosis, transthyretin falls apart and forms clumps called amyloid that harm tissues including nerves and the heart.
Read the full article on WikipediaTransthyretin amyloid cardiomyopathy Clinical criteria: Treatment Phase: First PBS-subsidised prescription for this drug The condition must have documented evidence of transthyretin precursor protein present, AND Patient must have experienced at least one episode of hospitalisation that was a direct result of heart failure; OR Patient must have clinical evidence of heart failure without hospitalisation that required treatment with a diuretic for improvement, AND Patient must have/have had New York Heart Association class I heart failure at the time of commencing this drug; OR Patient must have/have had New York Heart Association class II heart failure at the time of commencing this drug, AND Patient must have an end-diastolic interventricular septal wall thickness of at least 12 mm on imaging, AND Patient must have an estimated glomerular filtration rate (eGFR) greater than 25 mL/minute/1.73 m2. Treatment criteria: Must be treated by a medical practitioner who is any of the following: (i) a cardiologist, (ii) a consultant physician with experience in the management of amyloid disorders; this authority application must be sought by the same medical practitioner providing treatment. Applications for authorisation of initial treatment must be made via the Online PBS Authorities System (real time assessment) or in writing via HPOS form upload or mail. If the application is submitted through HPOS form upload or mail, it must include: (a) details of the proposed prescription; and (b) a completed authority application form relevant to the indication and treatment phase (the latest version is located on the website specified in the Administrative Advice). Evidence of clinical findings to establish the diagnosis: In this authority application, confirm that there is documented evidence of transthyretin precursor protein through either (1) alone, or, both (2) and (3), from the list below: Confirm the following has been completed: (1) amyloid expert centre histology findings derived via immunohistochemistry or mass spectrometry; OR (2) bone scintigraphy with grade 2-3 finding AND (3) Confirm that there are negative results for monoclonal protein on each of the following three tests: (a) serum immunofixation (also known as protein electrophoresis) (b) urine immunofixation (c) serum free light chains blood test State which of (1) to (3) above has been completed, as well as the: (i) date of the finding, (ii) imaging/pathology report number/code that links the finding to the patient, (iii) name of the amyloid expert centre in this authority application (if applicable). For end-diastolic interventricular septal wall thickness (at least 12 mm), confirm that: (i) imaging (echocardiogram or magnetic resonance imaging) has been undertaken; and (ii) that the imaging report is stored in the patient's medical records. State the date that the imaging was performed and the thickness (in mm) in this authority application. Where this authority application is to transition a patient from non-PBS-subsidised to PBS-subsidised supply (i.e. a 'grandfathered' patient), confirm the following: (i) the patient's heart failure has not worsened to persistent New York Heart Association Class III/IV heart failure while taking this drug.
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