Neurologic Stability During Tafamidis Treatment in Transthyretin Amyloidosis with a Mixed Phenotype: A Multicenter Real-World Study.
9 affiliations
- 1South Health Campus Hospital, 4448 Front Street SE, Calgary, AB, T3M 1M4, Canada. [email protected].
- 2Division of Cardiology, Department of Cardiac Sciences, Libin Cardiovascular Institute, Cumming School of Medicine, University of Calgary, Calgary, AB, Canada. [email protected].
- 3Department of Neurology, Georgetown University, Washington, DC, USA.
- 4ULS Santa Maria, MUHCRI, Faculdade de Medicina da Universidade de Lisboa, Lisbon, Portugal.
- 5Amyloidosis Research and Treatment Center, Foundation IRCCS Policlinico San Matteo, Pavia, Italy.
- 6Department of Molecular Medicine, University of Pavia, Pavia, Italy.
- 7Pfizer Inc, New York, NY, USA.
- 8MedStar Heart and Vascular Institute, Washington, DC, USA.
- 9Georgetown University School of Medicine, Washington, DC, USA.
Abstract
Introduction
Real-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.Methods
This 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.Results
Overall, 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/m² (p = 0.925), and 90% of patients with data remained within the same Essential Frailty Toolset score range. TTR concentration increased significantly (median change, 3.0 mg/dL; p < 0.001).Conclusions
In 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 registration
ClinicalTrials.gov, NCT06393465.
Related
- Impact of Tafamidis on Transthyretin Concentration and Its Relation to All-Cause Mortality in Patients with Transthyretin Amyloid Cardiomyopathy: An Analysis from ATTR-ACT
- Stage-Specific Cost-Effectiveness of Early vs. Delayed Tafamidis for Transthyretin Amyloid Cardiomyopathy in Japan
- Effect of Transthyretin Stabilizer in Transthyretin Cardiac Amyloidosis
- When Earlier Is Better But Still Not Cost-Effective ― Reframing the Value of Tafamidis in Transthyretin Amyloid Cardiomyopathy ―
- Gender and Racial Distribution of Transthyretin Stabilizer Therapy in Patients With Transthyretin Cardiac Amyloidosis.
BibTeX
@article{fine2026neurologic,
title = {{Neurologic Stability During Tafamidis Treatment in Transthyretin Amyloidosis with a Mixed Phenotype: A Multicenter Real-World Study.}},
author = {Nowell M Fine and Nicholas S Streicher and Isabel Conceição and João Agostinho and Laura Obici and Giovanni Palladini and Martin Carlsson and Leslie Amass and Farooq H Sheikh},
journal = {Cardiology and therapy},
year = {2026},
month = oct,
doi = {10.1007/s40119-026-00479-z},
url = {https://europepmc.org/article/MED/42844536},
note = {CC BY-NC},
}
RIS
TY - JOUR
TI - Neurologic Stability During Tafamidis Treatment in Transthyretin Amyloidosis with a Mixed Phenotype: A Multicenter Real-World Study.
AU - Nowell M Fine
AU - Nicholas S Streicher
AU - Isabel Conceição
AU - João Agostinho
AU - Laura Obici
AU - Giovanni Palladini
AU - Martin Carlsson
AU - Leslie Amass
AU - Farooq H Sheikh
PY - 2026
DA - 2026/10/07
JO - Cardiology and therapy
DO - 10.1007/s40119-026-00479-z
UR - https://europepmc.org/article/MED/42844536
AB - Introduction Real-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.Methods This 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.Results Overall, 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/m² (p = 0.925), and 90% of patients with data remained within the same Essential Frailty Toolset score range. TTR concentration increased significantly (median change, 3.0 mg/dL; p < 0.001).Conclusions In 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 registration ClinicalTrials.gov, NCT06393465.
SN - 2193-8261
SN - 2193-6544
ER -