Patisiran Wins Two Firsts With FDA Approval
(MedPage Today) -- RNAi drug OK'd to treat polyneuropathy from hereditary transthyretin amyloidosis
Amyloid transthyretin (ATTR) cardiac amyloidosis (CA) is an increasingly recognized cause of restrictive cardiomyopathy and associated heart failure with preserved ejection fraction (HFpEF). Despite improved diagnostic techniques to identify this condition, many patients experience delayed diagnosis. Interpretation of routine cardiac biomarkers (i.e. troponins and brain natriuretic peptide (BNP)) and echocardiography in ATTR-CA is limited, particularly in relation to pyrophosphate (PYP) scintigraphy.
Medical treatment for cardiac amyloidosis (CA) is evolving rapidly. Heart transplantation can be a valid option when followed by transplantation of bone marrow or liver, dependent on the type and origin of the amyloid protein. Thus, accurate typing of amyloidosis has implications for treatment, prognosis, and genetic counseling. Although non-invasive diagnostic techniques can type CA, endomyocardial biopsy (EMB) may be needed in the case of equivocal imaging findings or discordant data. We aimed to define the role of mass spectrometry (MS) for diagnosis and subtyping of CA.
After initial efforts at heart transplantation (HT) in patients with light chain amyloidosis were fraught with poor outcomes, emerging experience with modern chemotherapy regimens has led to a major improvement in outcomes in select centers. Given the systemic nature of the disease and the added immunosuppression in the form of chemotherapy, this study sought to analyze if AL patients undergoing HT had a higher burden of readmissions and infections non-amyloid heart transplant patients.
Advanced amyloid cardiomyopathy (ACM) patients have high waitlist (WL) mortality. Given the greater emphasis and clarity for status exceptions for ACM patients in the new allocation system, we sought to assess whether this change in the allocation policy would affect the WL and post-transplant outcomes in ACM pts.
Limited data exists on the use continuous-flow left ventricular assist device (CF-LVAD) in patients with cardiac amyloidosis (CA). The largest single center analysis on CF-LVAD support in patients with CA reports a 50% mortality at 6-months. Our primary aim was to examine the survival and morbidity of our patients with diagnosed CA and supported by CF-LVAD.
Advances in amyloidosis management have improved patient longevity. Coupling potential increased transplantation for amyloid patients with graft scarcity, we intend to assess the use of extended-donor criteria (EDC) hearts for amyloidosis.
AbstractTafamidis, a non ‐nonsteroidal anti‐inflammatory benzoxazole derivative, acts as a transthyretin (TTR) stabilizer to slow progression of TTR amyloidosis (ATTR). Tafamidis meglumine, available as 20‐mg capsules, is approved in more than 40 countries worldwide for the treatment of adults with early‐stage sympt omatic ATTR polyneuropathy. This agent, administered as an 80‐mg, once‐daily dose (4 × 20‐mg capsules), is approved in the United States, Japan, Canada, and Brazil for the treatment of hereditary and wild‐type ATTR cardiomyopathy in adults. An alternative single solid oral dosage formulati...
European Journal of Heart Failure, Volume 22, Issue 3, Page 389-390, March 2020.
ConclusionSurvival in patients with ATTR-PN is highly variable and affected by non-cardiac baseline characteristics, such as autonomic dysfunction, large fiber involvement, late-onset disease, and non-Val30Met mutation. Careful interpretation of these findings is warranted given that this synthesis did not control for differences between studies. Survival in patients with ATTR-PN remains poor among those who are untreated or with delayed diagnosis.
ONPATTRO is a drug for the treatment of nerve damage in adult patients with hereditary transthyretin-mediated amyloidosis.