Tafamidis is entering the clinical arena for the treatment of transthyretin‐related cardiomyopathy: certainties and unmet needs

Rapezzi, Claudio; Aimo, Alberto; Emdin, Michele

doi:10.1002/ejhf.2104

Cited by 1 publication

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References 27 publications

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“…This drug acts as a pharmacologic chaperone that stabilizes the properly folded tetramer of TTR protein by binding in one of the two thyroxine-binding sites [42]. The U.S. Food and Drug Administration (FDA) in 2019 and the EMA in 2020 approved tafamidis also for the treatment of transthyretin-mediated cardiomyopathy (ATT-CM) [43]. Although it is not always able to alter the disease, it is certainly very well tolerated and manageable [29].…”

Section: Treatment Usedmentioning

confidence: 99%

Use of Drugs for ATTRv Amyloidosis in the Real World: How Therapy Is Changing Survival in a Non-Endemic Area

et al. 2021

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Background: Over the past decade, three new drugs have been approved for the treatment of hereditary amyloid transthyretin (ATTRv) polyneuropathy. The aim of this work was to analyze whether current therapies prolong survival for patients affected by ATTRv amyloidosis. Methods: The study was conducted retrospectively, analyzing the medical records of 105 patients with genetic diagnoses of familial amyloidotic polyneuropathy followed at the two referral centers for the disease in Sicily, Italy. Of these, 71 received disease-modifying therapy, while 34 received only symptomatic treatment or no therapy. Results: The most used treatment in our patient cohort was tafamidis, followed by liver transplantation, patisiran, inotersen, and diflunisal. The median survival was significantly longer for treated vs. untreated patients (12 years vs. 8 years). In the 71 patients who received disease-modifying treatment, the presence of cardiac involvement, weight loss, or autonomic dysfunction at diagnosis was not related to survival. Conversely, patients diagnosed in the early stage of the disease (PND 1) had significantly longer survival than those diagnosed in the late stage (PND 2–4).

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Section: Treatment Usedmentioning

confidence: 99%