Articles: transthyretin.
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Clinical Trial
Safety and efficacy of a TTR specific antisense oligonucleotide in patients with transthyretin amyloid cardiomyopathy.
Cardiomyopathy is a major cause of death in both the hereditary form of transthyretin (TTR) amyloidosis and the sporadic late-age-onset transthyretin amyloidosis (ATTR wild-type (ATTRwt)). Clinically disease progression from time of diagnosis to death is usually quoted as 5- to 15-years. In prior studies, significant progression of cardiac parameters in patients with moderate to severe cardiomyopathy has been noted within a 12-month time span. ⋯ ASO treatment of patients with moderate to advanced ATTR cardiomyopathy shows indication of stabilization of disease progression and may therefore contribute to enhanced life expectancy.
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Multicenter Study Clinical Trial Observational Study
How your ears can tell what is hidden in your heart: wild-type transthyretin amyloidosis as potential cause of sensorineural hearing loss inelderly-AmyloDEAFNESS pilot study.
Wild-type transthyretin amyloidosis (ATTRwt) is an age-related life-threatening condition. Prognosis is mainly dependent on cardiac involvement. Other organs and tissues may be affected. Their early recognition may increase awareness of physicians and positively affects the prognosis. Presbycusis is another age-related disorder. Whether this disease is associated to ATTRwt amyloidosis is unknown. ⋯ These findings suggest that amyloid deposits could infiltrate the various anatomical structures of the inner ear. Description of specific audiologic pattern of ATTRwt amyloidosis might be proposed as a "red flag" and could help for early identification of patients who may be at high risk of ATTRwt amyloidosis as specific treatments are available.
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Randomized Controlled Trial
Tafamidis delays disease progression in patients with early stage transthyretin familial amyloid polyneuropathy: additional supportive analyses from the pivotal trial.
Tafamidis, a non-NSAID highly specific transthyretin stabilizer, delayed neurologic disease progression as measured by Neuropathy Impairment Score-Lower Limbs (NIS-LL) in an 18-month, double-blind, placebo-controlled randomized trial in 128 patients with early-stage transthyretin V30M familial amyloid polyneuropathy (ATTRV30M-FAP). The current post hoc analyses aimed to further evaluate the effects of tafamidis in delaying ATTRV30M-FAP progression in this trial. ⋯ These supportive analyses confirm that tafamidis delays neurologic progression in early-stage ATTRV30M-FAP.
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J. Am. Coll. Cardiol. · Jul 2016
Comparative StudyCardiac Amyloid Load: A Prognostic and Predictive Biomarker in Patients With Light-Chain Amyloidosis.
Cardiac amyloid load has not been analyzed for its effect on mortality in patients with amyloid light-chain (AL) cardiac amyloidosis. ⋯ Early identification of subjects with AL amyloid is essential given that in late-stage disease with extensive amyloid load, our data suggested that outcomes are not affected by administration of chemotherapy.