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Evidence For The Efficacy Of TTR Stabilizer Therapy To Improve Vascular Endothelial Function In Patients With Transthyretin Amyloidosis: 2146
Abstract   Peer reviewed

Evidence For The Efficacy Of TTR Stabilizer Therapy To Improve Vascular Endothelial Function In Patients With Transthyretin Amyloidosis: 2146

Jarred J. Iacovelli, Jeremy K. Alpenglow, Jill Waldron Stratford, Addison Concidine, Kanokwan Bunsawat, Jonah M. Simmons, Spencer Carter and D. Walter Wray
Medicine and science in sports and exercise, Vol.58(10S), 2146
10/2026
DOI: 10.1249/01.mss.0001265568.90941.9c

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Abstract

PURPOSE: Transthyretin amyloidosis (ATTR) is an infiltrative disease in which misfolded transthyretin (TTR) proteins are deposited in the extracellular space of organs such as the myocardium and blood vessel walls. While impairments in coronary microvascular function have been well characterized in patients with ATTR, less agreement exists regarding the degree of peripheral vascular dysfunction, which may be related to the impact of disease-modifying pharmacotherapy. Thus, this cross-sectional investigation sought to test the hypothesis that peripheral vascular responsiveness would be better in patients with ATTR treated with TTR stabilizer therapy (Treated; n=15) than in patients with ATTR who had not yet started this pharmacotherapy (Untreated; n=5). METHODS: Conduit artery endothelial function was determined by brachial artery flow-mediated dilation (FMD). Upper and lower limb microvascular function were determined by reactive hyperemia (RH) and passive limb movement (PLM) testing, respectively. Comparisons were made using Student’s unpaired t test. RESULTS: FMD was higher in the treated versus untreated ATTR group (Treated, 3.73 ± 1.60 %; Untreated, 1.79 ± 2.03 %; P=0.041). RH was not different between the treated and untreated ATTR groups (Treated, 439 ± 193 mL; Untreated, 368 ± 166; P=0.457). PLM leg blood flow area under-the-curve (LBFAUC) was higher in the treated versus untreated ATTR group (Treated, 168 ± 107 mL; Untreated, 76 ± 55 mL; P=0.007). CONCLUSIONS: These novel data provide that TTR stabilizer therapy may ameliorate endothelial-dependent peripheral vascular function in patients with ATTR, though longitudinal studies are warranted to ascertain these preliminary findings.

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