ArticleCardiology and therapy2026
Neurologic Stability During Tafamidis Treatment in Transthyretin Amyloidosis with a Mixed Phenotype: A Multicenter Real-World Study.
Article in Cardiology and therapy, 2026. The graph could read no effect estimate from its abstract, so it casts no vote on the map. It is linked to trial NCT06393465 (Real-World Effectiveness of High-Dose Tafamidis on Neurologic Disease Progression in Mixed-Phenotype Transthyretin Amyloid Cardiomyopathy), which is not on this map. Not yet cited in PubMed.
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Real-World Effectiveness of High-Dose Tafamidis on Neurologic Disease Progression in Mixed-Phenotype Transthyretin Amyloid Cardiomyopathy (ATTR-CM)
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9 authors.
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Abstract
introductionReal-world evidence on the effect of tafamidis in mixed-phenotype transthyretin (TTR) amyloidosis (ATTR amyloidosis) is limited. This study assessed changes in neurologic and other measures in patients with mixed-phenotype ATTR amyloidosis treated with tafamidis free acid 61 mg for ≥ 12 months in routine clinical practice.
methodsThis observational, non-interventional, retrospective multicenter cohort study used anonymized data from four centers. Adults with variant or wild-type mixed-phenotype ATTR amyloidosis treated with tafamidis 61 mg for ≥ 12 months with pre- and post-treatment neurologic assessments were eligible. The primary objective was to assess changes in neurologic measures after ≥ 12 months of treatment; secondary and exploratory objectives were to assess changes in cardiac and frailty measures, modified body mass index (mBMI), and TTR concentration.
resultsOverall, 67 patients were included (median age 77.0 years; 79.1% male patients; 55.2% with variant ATTR amyloidosis). Median duration of treatment with tafamidis 61 mg was 35.0 months. Neuropathy Impairment Score (NIS) motor subscale, NIS reflex subscale, and Medical Research Council Scale for Muscle Strength scores showed a median change of 0 from baseline after ≥ 12 months of treatment. Cardiac measures demonstrated minimal change, although interventricular septal thickness (median change, 0.1 cm; p = 0.017) and left atrial diameter (median change, 0.3 cm; p = 0.018) increased modestly. Median change in mBMI was 6.7 g/L × kg/m
conclusionsIn this real-world study of patients with variant or wild-type mixed-phenotype ATTR amyloidosis, neurologic, cardiac, mBMI, and frailty measures were largely maintained after ≥ 12 months of treatment with tafamidis 61 mg. TTR concentration significantly increased. These findings provide real-world evidence of maintenance of neurologic function during treatment with tafamidis in mixed-phenotype ATTR amyloidosis.
trial registrationClinicalTrials.gov, NCT06393465.
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