Effects of tafamidis: A Synthesis of Findings from 22 Studies

  1. Home
  2. Effects of tafamidis
This information is not medical advice and is not a substitute for diagnosis or treatment by a physician.Data sources and disclaimers (data limitations, copyright, etc.)The analysis on "Effects of tafamidis: A Synthesis of Findings from 22 Studies" on this page is based on PubMed data provided by the U.S. National Library of Medicine (NLM). However, NLM does not endorse or verify these analyses.

This analysis is based on research papers included in PubMed, but medical research is constantly evolving and may not fully reflect the latest findings. There may also be biases towards certain research areas.

This information is not medical advice and is not a substitute for diagnosis or treatment by a physician. If you have concerns about "Effects of tafamidis: A Synthesis of Findings from 22 Studies", please consult your doctor.

For NLM copyright information, please see Link to NLM Copyright Page
PubMed data is obtained via Hugging Face Datasets: Link to DatasetPlease check the disclaimer.
This page's analysis is based on PubMed data provided by the U.S. National Library of Medicine (NLM).
Original Abstract of the Article

Major Findings

Tafamidis, a transthyretin (TTR) kinetic stabilizer, has been shown to delay the progression of transthyretin familial amyloid polyneuropathy (TTR-FAP) in multiple studies. 14 , 12 , 20 , 8 , 10 , 2 , 4 , 5 , 15 , 18 , 21 , 22 , 11 , 17 , , 16 , 1 , 19 , 13 , 6 , , 7 , 3 , 9 It has been shown to stabilize TTR, preventing the formation of amyloid fibrils. 14 , 4 , 13 Early initiation of tafamidis treatment may lead to greater benefits in preserving neurological function. 14 While long-term effects of tafamidis on mortality are not yet fully understood, 8 studies suggest that it is generally well-tolerated. 14

Benefits and Risks

Benefits Summary

Tafamidis has the potential to slow the progression of TTR-FAP and may help to preserve neurological function. 14 , 12 , 4 , 5 , 1 , 19 , 7 It can also help to stabilize TTR. 14 , 4 , 13 Tafamidis is generally well-tolerated and suitable for long-term use. 14

Risks Summary

Potential side effects of tafamidis include gastrointestinal issues such as nausea, vomiting, and diarrhea, as well as skin reactions like rash. 14 Most of these side effects are mild and do not require discontinuation of treatment. 14 Long-term effects of tafamidis on mortality are not yet fully understood. 8

Comparison Between Studies

Similarities

Numerous studies have consistently demonstrated that tafamidis can effectively delay the progression of TTR-FAP. 14 , 12 , 4 , 5 , 1 , 19 , 7 It has also been shown to effectively stabilize TTR in numerous studies. 14 , 4 , 13 Most research indicates that tafamidis is well-tolerated. 14

Differences

The effectiveness of tafamidis can vary based on factors such as patient age, the type of TTR mutation present, and the severity of the disease. 5 , 10 The impact of long-term tafamidis treatment on mortality is still under investigation. 8

Consistency and Contradictions in Results

While multiple studies support the efficacy of tafamidis, the magnitude and duration of its effects can vary between studies. 14 , 5 , 7 Reports on the side effects of tafamidis also differ between studies. 14 Further research is needed to better understand the effects and side effects of tafamidis. 8

Considerations for Real-World Application

While tafamidis holds promise for slowing the progression of TTR-FAP, it may not be effective for all patients. 14 , 12 , 4 , 5 , 1 , 19 , 7 Potential side effects should also be considered. 14 Tafamidis should be used under the guidance of a healthcare professional. 14

Limitations of Current Research

The long-term effects and safety of tafamidis are not fully understood. 8 Further research is needed to investigate the long-term impact of tafamidis. 8

Future Directions for Research

Long-term studies evaluating the effects of tafamidis are needed. 8 Research should explore optimal dosage regimens and strategies to minimize side effects. 14

Conclusion

Tafamidis shows promise as a treatment for TTR-FAP, potentially slowing its progression. 14 , 12 , 4 , 5 , 1 , 19 , 7 However, the effectiveness of tafamidis can vary depending on individual patient factors. 5 , 10 It's crucial to consider potential side effects and use tafamidis under the guidance of a healthcare professional. 14 Further research is ongoing to better understand the long-term effects and optimize the use of tafamidis. 8

Literature analysis of 22 papers
Positive Content
21
Neutral Content
0
Negative Content
1
Article Type
4
0
1
8
22
1.

Tafamidis: a review of its use in familial amyloid polyneuropathy.

Author: ScottLesley J

Delay peripheral neurologic impairment
Treatment for TTR amyloidosis

Tafamidis is a drug that is used to treat transthyretin (TTR) amyloidosis, a rare disease that causes damage to the nerves and organs. This study found that tafamidis was effective at slowing the progression of nerve damage in patients with early-stage TTR amyloidosis. The study also found that tafamidis was well-tolerated.

Journal ArticleAn article published in an academic or professional journal that reports original research or scholarly analysis.
ReviewAn article that critically examines existing research on a particular topic and summarizes the current state of knowledge in the field.

Language : English

2.

Treatment of cardiac transthyretin amyloidosis: an update.

Author: EmdinMichele, AimoAlberto, RapezziClaudio, FontanaMarianna, PerfettoFederico, SeferovićPetar M, BarisonAndrea, CastiglioneVincenzo, VergaroGiuseppe, GiannoniAlberto, PassinoClaudio, MerliniGiampaolo

Treatment options
Cardiac involvement
Dyspnea

This study provides an update on the treatment of TTR-related amyloidosis (ATTR), an underdiagnosed disorder that can lead to heart failure. While liver transplantation was previously the only treatment option, several new pharmacological agents are now available to inhibit TTR synthesis, stabilize the tetramer, or disrupt fibrils.

Journal ArticleAn article published in an academic or professional journal that reports original research or scholarly analysis.
ReviewAn article that critically examines existing research on a particular topic and summarizes the current state of knowledge in the field.

Language : English

4.

Effects of tafamidis on transthyretin stabilization and clinical outcomes in patients with non-Val30Met transthyretin amyloidosis.

Author: MerliniGiampaolo, Planté-BordeneuveViolaine, JudgeDaniel P, SchmidtHartmut, ObiciLaura, PerliniStefano, PackmanJeff, TrippTara, GroganDonna R

TTR stabilization
Worsening neurological function

Tafamidis, a drug for the treatment of transthyretin (TTR) amyloidosis, effectively stabilized TTR associated with several non-Val30Met variants in a phase II study. Tafamidis 20 mg QD was well tolerated.

Clinical Trial, Phase IIA Phase II trial is a trial that further evaluates the efficacy and safety of a new drug or treatment, whose safety has been confirmed to some extent in a Phase I trial, in a small number of patients.
Journal ArticleAn article published in an academic or professional journal that reports original research or scholarly analysis.
Multicenter StudyResearch conducted in collaboration with multiple research institutions. It aims to collect a wide range of data and obtain more generalizable results.
Research Support, Non-U.S. Gov'tResearch funding provided by government agencies outside the United States.

Language : English

6.

[Newly developed drug therapies for familial amyloid polyneuropathy: diflunisal and tafamidis].

Author: SekijimaYoshiki

Treatment for FAP

Familial amyloid polyneuropathy (FAP) is a genetic disorder with systemic deposition of amyloid fibrils. Until recently, liver transplantation was the only effective treatment. However, tafamidis, a transthyretin tetramer stabilizer, has been approved for the treatment of FAP in Europe and Japan.

English AbstractA summary of a research paper/publication written in English for a wider audience.
Journal ArticleAn article published in an academic or professional journal that reports original research or scholarly analysis.

Language : Japanese

7.

Tafamidis delays disease progression in patients with early stage transthyretin familial amyloid polyneuropathy: additional supportive analyses from the pivotal trial.

Author: KeohaneDenis, SchwartzJeffrey, GundapaneniBalarama, StewartMichelle, AmassLeslie

ATTRV30M-FAP progression
Neurologic disease

This study conducted post hoc analyses to further evaluate the effects of tafamidis, a transthyretin stabilizer, in delaying the progression of transthyretin V30M familial amyloid polyneuropathy (ATTRV30M-FAP).

Journal ArticleAn article published in an academic or professional journal that reports original research or scholarly analysis.
Randomized Controlled TrialA clinical trial that randomly assigns participants to treatment and control groups to evaluate the effects of an intervention.

Language : English

8.

Evaluation of Mortality During Long-Term Treatment with Tafamidis for Transthyretin Amyloidosis with Polyneuropathy: Clinical Trial Results up to 8.5 Years.

Author: MerliniGiampaolo, CoelhoTeresa, Waddington CruzMárcia, LiHuihua, StewartMichelle, EbedeBen

Survival benefit

This study found that long-term tafamidis treatment may confer survival benefit in patients with transthyretin amyloidosis with polyneuropathy (ATTR-PN).

Journal ArticleAn article published in an academic or professional journal that reports original research or scholarly analysis.

Language : English

9.

Targeting transthyretin - Mechanism-based treatment approaches and future perspectives in hereditary amyloidosis.

Author: DohrnMaike F, IhneSandra, HegenbartUte, MedinaJessica, ZüchnerStephan L, CoelhoTeresa, HahnKatrin

Neuropathy improvement
Quality of life improvement
TTR knockdown
TTR stabilization
Application burden
Blood-brain barrier permeability
Side effects

Transthyretin (TTR) amyloidosis is a lethal disease caused by the liver-derived transport protein TTR. This systematic review summarizes the current literature on TTR pathophysiology and therapeutic implications, including approved therapies like tafamidis, patisiran, and inotersen, and discusses future directions in research.

Journal ArticleAn article published in an academic or professional journal that reports original research or scholarly analysis.
Research Support, Non-U.S. Gov'tResearch funding provided by government agencies outside the United States.
Systematic ReviewA review that systematically collects, evaluates, and integrates relevant research to answer a specific research question.

Language : English

10.

Influence of baseline neurologic severity on disease progression and the associated disease-modifying effects of tafamidis in patients with transthyretin amyloid polyneuropathy.

Author: AmassLeslie, LiHuihua, GundapaneniBalarama K, SchwartzJeffrey H, KeohaneDenis J

ATTR-PN treatment
neurological severity

This analysis used longitudinal data from patients with transthyretin amyloid polyneuropathy (ATTR-PN) to evaluate the impact of baseline neurologic severity on disease progression.

Journal ArticleAn article published in an academic or professional journal that reports original research or scholarly analysis.
Randomized Controlled TrialA clinical trial that randomly assigns participants to treatment and control groups to evaluate the effects of an intervention.
Research Support, Non-U.S. Gov'tResearch funding provided by government agencies outside the United States.

Language : English

11.

[Review of the recent literature on peripheral neuropathies: therapeutic advances].

Author: AdamsD

Peripheral neuropathy treatment

This review highlights the importance of multicentric clinical trials in improving therapeutic strategies for peripheral neuropathies. It discusses several studies on chronic inflammatory demyelinating polyradiculoneuropathy (CIDP), familial amyloidotic polyneuropathy, and other peripheral neuropathies, and emphasizes the need for more research to assess treatment effectiveness.

English AbstractA summary of a research paper/publication written in English for a wider audience.
Journal ArticleAn article published in an academic or professional journal that reports original research or scholarly analysis.
ReviewAn article that critically examines existing research on a particular topic and summarizes the current state of knowledge in the field.

Language : French

13.

Tafamidis in transthyretin amyloid cardiomyopathy: effects on transthyretin stabilization and clinical outcomes.

Author: MaurerMathew S, GroganDonna R, JudgeDaniel P, MundayatRajiv, PackmanJeff, LombardoIlise, QuyyumiArshed A, AartsJanske, FalkRodney H

TTR stabilization
Well tolerated
Cardiovascular events
Clinical progression
Diarrhea

A phase 2 open-label trial found that tafamidis treatment effectively stabilized transthyretin (TTR) tetramers in patients with TTR amyloid cardiomyopathy. While the treatment was well tolerated, further evaluation is needed to determine its long-term efficacy.

Clinical Trial, Phase IIA Phase II trial is a trial that further evaluates the efficacy and safety of a new drug or treatment, whose safety has been confirmed to some extent in a Phase I trial, in a small number of patients.
Journal ArticleAn article published in an academic or professional journal that reports original research or scholarly analysis.
Multicenter StudyResearch conducted in collaboration with multiple research institutions. It aims to collect a wide range of data and obtain more generalizable results.
Research Support, Non-U.S. Gov'tResearch funding provided by government agencies outside the United States.

Language : English

14.

Long-term effects of tafamidis for the treatment of transthyretin familial amyloid polyneuropathy.

Author: CoelhoTeresa, MaiaLuis F, da SilvaAna Martins, CruzMárcia W, Planté-BordeneuveViolaine, SuhrOle B, ConceiçaoIsabel, SchmidtHartmut H-J, TrigoPedro, KellyJeffery W, LabaudinièreRichard, ChanJason, PackmanJeff, GroganDonna R

Neurologic function
TTR stabilization

Long-term tafamidis treatment (30 months) was well tolerated and showed sustained benefits in patients with Val30Met TTR familial amyloid polyneuropathy (TTR-FAP). Earlier initiation of tafamidis led to greater preservation of neurologic function.

Journal ArticleAn article published in an academic or professional journal that reports original research or scholarly analysis.
Multicenter StudyResearch conducted in collaboration with multiple research institutions. It aims to collect a wide range of data and obtain more generalizable results.
Randomized Controlled TrialA clinical trial that randomly assigns participants to treatment and control groups to evaluate the effects of an intervention.
Research Support, Non-U.S. Gov'tResearch funding provided by government agencies outside the United States.

Language : English

15.

Tafamidis decreased cardiac amyloidosis deposition in patients with Ala97Ser hereditary transthyretin cardiomyopathy: a 12-month follow-up cohort study.

Author: TsaiCheng-Hsuan, ChaoChi-Chao, HsiehSung-Tsang, YuAn-Li, WuYuan-Kun Aden, ChengMei-Fang, LeeMing-Jen, ChouChia-Hung, ShunChia-Tung, HsuehHsueh-Wen, Jyh-Ming JuangJimmy, TsengPing-Huei, SuMao-Yuan, LinYen-Hung

ATTR-CM treatment

This study aimed to investigate the effects of tafamidis, an effective treatment for transthyretin cardiac cardiomyopathy (ATTR-CM), on cardiac remodeling and cardiac amyloid deposition in patients with hereditary A97S ATTR-CM using cardiac magnetic resonance (CMR).

Journal ArticleAn article published in an academic or professional journal that reports original research or scholarly analysis.

Language : English

16.

Advances in the treatment of hereditary transthyretin amyloidosis: A review.

Author: GertzMorie A, MauermannMichelle L, GroganMartha, CoelhoTeresa

Treat amyloidosis

Inotersen and patisiran are new therapies that have been approved for the treatment of amyloid transthyretin amyloidosis (ATTR), a progressive and often fatal disease.

Journal ArticleAn article published in an academic or professional journal that reports original research or scholarly analysis.
ReviewAn article that critically examines existing research on a particular topic and summarizes the current state of knowledge in the field.

Language : English

17.

The effect of tafamidis on the QTc interval in healthy subjects.

Author: KlamerusKaren J, WatskyEric, MollerRobert, WangRonnie, RileySteve

Tafamidis QTc safe
Tafamidis not prolong QTc

A supra-therapeutic dose of tafamidis did not prolong the QTc interval and was well-tolerated in healthy volunteers.

Journal ArticleAn article published in an academic or professional journal that reports original research or scholarly analysis.
Multicenter StudyResearch conducted in collaboration with multiple research institutions. It aims to collect a wide range of data and obtain more generalizable results.
Randomized Controlled TrialA clinical trial that randomly assigns participants to treatment and control groups to evaluate the effects of an intervention.
Research Support, Non-U.S. Gov'tResearch funding provided by government agencies outside the United States.

Language : English

18.

[Ocular involvement in familial amyloid polyneuropathy].

Author: RousseauA, KaswinG, AdamsD, CauquilC, ThéaudinM, MinchevaZ, M'garrechM, LabetoulleM, BarreauE

Improved FAP
Increased survival
Cardiac failure
Neurological symptoms
Ocular complications
Renal failure

Familial amyloid polyneuropathy (FAP) is a rare, inherited disorder that affects the nerves, heart, kidneys, and eyes. It is caused by a buildup of abnormal protein in the body. Liver transplantation can stop the progression of the disease in 70% of cases and doubles the median survival of patients. Tafamidis is a new drug that can help to delay the development of neurological complications. Ocular manifestations of FAP are common and include keratoconjunctivitis sicca, glaucoma, and vitreous deposits.

English AbstractA summary of a research paper/publication written in English for a wider audience.
Journal ArticleAn article published in an academic or professional journal that reports original research or scholarly analysis.
ReviewAn article that critically examines existing research on a particular topic and summarizes the current state of knowledge in the field.

Language : French

19.

Effects of tafamidis treatment on transthyretin (TTR) stabilization, efficacy, and safety in Japanese patients with familial amyloid polyneuropathy (TTR-FAP) with Val30Met and non-Val30Met: A phase III, open-label study.

Author: AndoYukio, SekijimaYoshiki, ObayashiKonen, YamashitaTaro, UedaMitsuharu, MisumiYohei, MoritaHiroshi, MachiiKatsuyuki, OhtaMakoto, TakataAmi, IkedaShû-Ichi

Slowed neurologic progression
TTR stabilization

Tafamidis stabilized transthyretin, was safe and well-tolerated, and was effective over 1.5 years in slowing neurologic progression and maintaining quality of life and nutrition status in transthyretin familial amyloid polyneuropathy.

Clinical Trial, Phase IIIA Phase III trial is a trial that further rigorously verifies the efficacy and safety of a new drug or treatment, whose efficacy and safety have been confirmed in a Phase II trial, in a large number of patients, and evaluates whether it is superior to existing treatments.
Journal ArticleAn article published in an academic or professional journal that reports original research or scholarly analysis.
Multicenter StudyResearch conducted in collaboration with multiple research institutions. It aims to collect a wide range of data and obtain more generalizable results.
Research Support, Non-U.S. Gov'tResearch funding provided by government agencies outside the United States.

Language : English

20.

Tetrabromobisphenol A Is an Efficient Stabilizer of the Transthyretin Tetramer.

Author: IakovlevaIrina, BegumAfshan, BrännströmKristoffer, WijsekeraAlexandra, NilssonLina, ZhangJin, AnderssonPatrik L, Sauer-ErikssonA Elisabeth, OlofssonAnders

Stabilizes the tetrameric structure of TTR
Therapeutic effects against FAP

Tetrabromobisphenol A (TBBPA), a commonly used flame retardant, effectively stabilizes the tetrameric structure of transthyretin (TTR) with high selectivity in human plasma, offering potential as a drug scaffold for treating amyloid-related disorders.

Journal ArticleAn article published in an academic or professional journal that reports original research or scholarly analysis.

Language : English

21.

Tafamidis: A Review in Transthyretin Amyloid Cardiomyopathy.

Author: LambYvette N

Amyloid cardiomyopathy treatment

This study examines the efficacy and safety of tafamidis, a TTR stabilizer, in treating transthyretin amyloid cardiomyopathy (ATTR-CM). Tafamidis significantly reduced all-cause mortality and cardiovascular-related hospitalizations compared to placebo, demonstrating its potential as a disease-modifying therapy for ATTR-CM.

Journal ArticleAn article published in an academic or professional journal that reports original research or scholarly analysis.
ReviewAn article that critically examines existing research on a particular topic and summarizes the current state of knowledge in the field.

Language : English

22.

Tafamidis for autonomic neuropathy in hereditary transthyretin (ATTR) amyloidosis: a review.

Author: CruzMárcia Waddington

Autonomic dysfunction

Autonomic dysfunction is a common and distressing symptom of hereditary transthyretin (ATTR) amyloidosis. This abstract explores the use of tafamidis, a drug approved for ATTR, to treat dysautonomia, although direct evidence is limited.

Journal ArticleAn article published in an academic or professional journal that reports original research or scholarly analysis.
ReviewAn article that critically examines existing research on a particular topic and summarizes the current state of knowledge in the field.

Language : English

This site uses cookies. Visit our privacy policy page or click the link in any footer for more information and to change your preferences.