ArticleNeurology and therapy2015
A Review of Tafamidis for the Treatment of Transthyretin-Related Amyloidosis.
Article in Neurology and therapy, 2015. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 33 papers, 1 of them a synthesis that pooled it.
What it found
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The abstract states no effect estimate the extractor could read, or names no intervention and outcome on the map, so this paper lights no cell and moves no belief. It is still indexed, cited and linked below.
The trial behind it
Trials whose registry record cites this paper, or whose number appears in the abstract. A trial that started after this paper was published is citing it as background, not reporting it.
Neither the registry nor the abstract names a trial number. If this is a trial report, that itself is worth knowing.
Who cites it
33 citing papers in PubMed, 1 synthesis or guideline pooled it, 78 citations in OpenAlex.
- Chemistry, Biological Properties, and Bio-analysis of Tafamidis, a New Transthyretin Stabilizer: A Systematic Review.Cardiovascular & hematological agents in medicinal chemistry · 2024Pooled it
- A phase II, open-label, extension study of long-term patisiran treatment in patients with hereditary transthyretin-mediated (hATTR) amyloidosis.Orphanet journal of rare diseases · 2020Trial
- Early intervention with tafamidis provides long-term (5.5-year) delay of neurologic progression in transthyretin hereditary amyloid polyneuropathy.Amyloid : the international journal of experimental and clinical investigation : the official journal of the International Society of Amyloidosis · 2016Trial
- Central Nervous System Manifestations Associated with Hereditary Transthyretin Amyloidosis: a Narrative Review.Arquivos de neuro-psiquiatria · 2026Review
- Recovery of right ventricular function in patients with transthyretin cardiac amyloidosis after one-year tafamidis administration.The international journal of cardiovascular imaging · 2025Article
- Real-world tafamidis experience in hereditary transthyretin amyloidosis with peripheral neuropathy in Brazil.Arquivos de neuro-psiquiatria · 2025Article
- Optimal practices for the management of hereditary transthyretin amyloidosis: real-world experience from Japan, Brazil, and Portugal.Orphanet journal of rare diseases · 2023Review
- Brazilian consensus for diagnosis, management and treatment of hereditary transthyretin amyloidosis with peripheral neuropathy: second edition.Arquivos de neuro-psiquiatria · 2023Article
- Article
- Updated Evaluation of the Safety, Efficacy and Tolerability of Tafamidis in the Treatment of Hereditary Transthyretin Amyloid Polyneuropathy.Drug, healthcare and patient safety · 2023Review
- Long-Term Survival With Tafamidis in Patients With Transthyretin Amyloid Cardiomyopathy.Circulation. Heart failure · 2022Article
- Preparative Scale Production of Recombinant Human Transthyretin for Biophysical Studies of Protein-Ligand and Protein-Protein Interactions.International journal of molecular sciences · 2020Article
- Diagnosis and treatment of transthyretin-related amyloidosis cardiomyopathy.Clinical cardiology · 2020Review
- Poor Yield of Routine Transthyretin Screening in Patients with Idiopathic Neuropathy.The Canadian journal of neurological sciences. Le journal canadien des sciences neurologiques · 2020Article
- Evaluation of Mortality During Long-Term Treatment with Tafamidis for Transthyretin Amyloidosis with Polyneuropathy: Clinical Trial Results up to 8.5 Years.Neurology and therapy · 2020Article
- Diagnosis and Screening of Patients with Hereditary Transthyretin Amyloidosis (hATTR): Current Strategies and Guidelines.Therapeutics and clinical risk management · 2020Review
- Review on the Structures and Activities of Transthyretin Amyloidogenesis Inhibitors.Drug design, development and therapy · 2020Review
- A pair of peptides inhibits seeding of the hormone transporter transthyretin into amyloid fibrils.The Journal of biological chemistry · 2019Article
- Autonomic involvement in hereditary transthyretin amyloidosis (hATTR amyloidosis).Clinical autonomic research : official journal of the Clinical Autonomic Research Society · 2019Review
- Tafamidis, a Noninvasive Therapy for Delaying Transthyretin Familial Amyloid Polyneuropathy: Systematic Review and Meta-Analysis.Journal of clinical neurology (Seoul, Korea) · 2019Article
Corrections and comments
PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.
Authors and funding
2 authors at 2 institutions in 2 countries.
Funding
No grant is acknowledged in the PubMed record.
Abstract
Transthyretin (TTR)-related amyloidosis (ATTR) is a devastating disease which affects a combination of organs including the heart and the peripheral nerves, and which has a fatal outcome if not treated within a average of 10 years. Tafamidis, or 2-(3,5-dichloro-phenyl)-benzoxazole-6-carboxylic acid, selectively binds to TTR with negative cooperativity and kinetically stabilizes wild-type native TTR and mutant TTR; tafamidis therefore has the potential to halt the amyloidogenic cascade initiated by TTR tetramer dissociation, monomer misfolding, and aggregation. The first tafamidis trial, Fx-005, evaluated the effect of 18 months of tafamidis treatment (20 mg once daily) on disease progression, as well as assessing its safety in TTR-FAP Val30Met patients. The secondary objective of this trial was to study the pharmacodynamic stabilization of mutated TTR. Tafamidis proved effective in reducing the progress of neuropathy, and in maintaining the nutritional status and quality of life of stage 1 (able to walk without support) Val3OMet TTR-FAP patients. Furthermore, TTR stabilization was achieved in more than 90% of patients. An extension study, Fx-006, was conducted to determine the long-term safety and tolerability of tafamidis and to assess the efficacy of the drug on slowing disease progression. No significant safety or tolerability issues were noticed. Taken together, the results from both trials indicated that the beneficial effects of tafamidis were sustained over a 30-month period and that starting treatment early is desirable. Results are expected from an extended open-label study but data that have already been presented show that long-term use of tafamidis in Val30Met patients is associated with reduced progression in polyneuropathy. Tafamidis was initially approved for commercial use in Europe in 2011 and has since been approved for use in Japan, Mexico, and Argentina where it is used as a first-line treatment option for patients with early-stage TTR-FAP. Patients should be carefully followed at referral centers to ascertain the individual response to treatment. In cases of discontinuation, liver transplantation and enrollment in clinical trials of novel drugs aimed mostly toward suppression of TTR production are options.
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