Vyndaqel (tafamidis meglumine) / Pfizer - LARVOL DELTA

Novel Therapeutic Mechanisms for the Treatment of Transthyretin Amyloid Cardiomyopathy. (PubMed, Cardiol Rev) - "In this review, special attention is given to tafamidis and vutrisiran for the treatment of ATTR-CM, while exploring the therapeutic potential of leptin modulation and TIE kinase inhibition through a narrative review of preclinical and clinical evidence. On the other hand, leptin modulators and TIE kinase inhibition show promise in clinical trials, but further validation of these findings is necessary. Ultimately, these agents may become therapeutic targets for treating ATTR-CM in the future."

Journal • Amyloidosis • Cardiac Amyloidosis • Cardiomyopathy • Cardiovascular • LEP

To Evaluate the Efficacy and Safety of Tafamidis Meglumine Soft Capsules in the Treatment of Adult Patients With Transthyretin Amyloid Polyneuropathy (clinicaltrials.gov) - P4 | N=15 | Recruiting | Sponsor: Qilu Pharmaceutical Co., Ltd. | Not yet recruiting ➔ Recruiting

Enrollment open • Amyloidosis • CNS Disorders • Pain

Transthyretin Kinetic Stabilizers for ATTR Amyloidosis: A Narrative Review of Mechanisms and Therapeutic Benefits. (PubMed, Cardiol Ther) - "Current availability of two kinetic stabilizers has increased interest in their pharmacological properties and clinical effects, including potential similarities and disparities. In this review, the mechanisms involved in TTR kinetic stabilization are summarized with preclinical and clinical study findings on the use of the kinetic stabilizers tafamidis and acoramidis."

Clinical • Journal • Review • Amyloidosis • Cardiac Amyloidosis • Cardiomyopathy • Cardiovascular • Pain

Proteomic Analysis of Serum in Cardiac Transthyretin Amyloidosis: Diagnostic and Prognostic Implications for Biomarker Discovery. (PubMed, Biomedicines) - "Controls exhibited elevated levels of transthyretin and prothrombin. This study identified candidate serum biomarkers for diagnosing ATTR-CA and monitoring the effectiveness of tafamidis treatment."

Biomarker • Journal • Amyloidosis • Cardiac Amyloidosis • Cardiomyopathy • Cardiovascular • A2M • APOA2 • APOE • CD14 • CLU • GSN • PON1 • SERPINA1 • SERPINA3 • VCAM1

Relative efficacy of tafamidis, acoramidis, patisiran and vutrisiran in patients with transthyretin cardiac amyloidosis: a network meta-analysis (ESC-WCC 2025) - No abstract available

Retrospective data • Cardiac Amyloidosis • Cardiovascular

Tafamidis in patients with amyloid cardiomyopathy: a systematic review and meta-analysis of 14 studies. (ESC-WCC 2025) - No abstract available

Retrospective data • Review • Cardiac Amyloidosis • Cardiomyopathy • Cardiovascular

Plasma transthyretin fractions in transthyretin cardiac amyloidosis patients undergoing tafamidis treatment (ESC-WCC 2025) - No abstract available

Clinical • Cardiac Amyloidosis • Cardiovascular

Combination therapy with sglt2-inhibitors and tafamidis in transthyretin cardiomyopathy (ESC-WCC 2025) - No abstract available

Combination therapy • Cardiomyopathy • Cardiovascular

Tafamidis treatment significantly reduces the risk of recurrent cardiovascular-related hospitalisation in patients with early stage transthyretin amyloid cardiomyopathy (ESC-WCC 2025) - No abstract available

Clinical • Cardiac Amyloidosis • Cardiomyopathy • Cardiovascular

Survival in a contemporary, real-world, matched cohort of patients with transthyretin amyloid cardiomyopathy treated vs not treated with tafamidis (ESC-WCC 2025) - No abstract available

Clinical • Real-world • Real-world evidence • Cardiac Amyloidosis • Cardiomyopathy • Cardiovascular

Impact of Tafamidis on survival in elderly patients in a real-world setting: insights from the Healthcare European Amyloidosis Registry (ESC-WCC 2025) - No abstract available

Clinical • Real-world • Real-world evidence • Cardiac Amyloidosis • Cardiovascular

Dose dependent tafamidis response in transthyretin amyloid cardiomyopathy: a four-case series with serial bone scintigraphy [WITHDRAWN] (ESC-WCC 2025) - No abstract available

Clinical • Cardiac Amyloidosis • Cardiomyopathy • Cardiovascular

A case of concurrent light chain and transthyretin cardiac amyloidosis treated with chemotherapy and tafamidis (ESC-WCC 2025) - No abstract available

Clinical • Cardiac Amyloidosis • Cardiovascular

Potential association between tafamidis and aortic stenosis progression in cardiac amyloidosis (ESC-WCC 2025) - No abstract available

Cardiac Amyloidosis • Cardiovascular

Prognostic value of baseline cardiopulmonary exercise testing in tafamidis-treated wild-type transthyretin amyloid cardiomyopathy (ESC-WCC 2025) - No abstract available

Cardiac Amyloidosis • Cardiomyopathy • Cardiovascular

Impact of tafamidis on sleep-disordered breathing in transthyretin cardiomyopathy (ESC-WCC 2025) - No abstract available

Cardiomyopathy • Cardiovascular

Real-world effectiveness of tafamidis in a contemporary, matched cohort of patients with variant and wild-type transthyretin amyloid cardiomyopathy (ESC-WCC 2025) - No abstract available

Clinical • Real-world • Real-world effectiveness • Real-world evidence • Cardiac Amyloidosis • Cardiomyopathy • Cardiovascular

Transthyretin Amyloid Cardiomyopathy-2025 Update: Current Diagnostic Approaches and Emerging Therapeutic Options. (PubMed, J Clin Med) - "First-generation therapies such as tafamidis have demonstrated survival benefits in ATTR-CM. More recently, second-generation agents-such as the TTR stabilizer acoramidis and RNA silencers including vutrisiran and eplontersen-have shown promising efficacy in clinical trials...This review outlines current diagnostic strategies and therapeutic options for ATTR amyloidosis, emphasizing the need for early detection and individualized treatment approaches. The expanding therapeutic landscape highlights the importance of accurate phenotyping and timely intervention to optimize clinical outcomes."

Journal • Review • Amyloidosis • Cardiac Amyloidosis • Cardiomyopathy • Cardiovascular • Congestive Heart Failure • Heart Failure • Pain

Current and Future Treatment Landscape of Transthyretin Amyloid Cardiomyopathy. (PubMed, Cardiol Ther) - "Therapeutic advances have significantly increased treatment possibilities, selection of appropriate therapy, switching between therapies, combination strategies, and how to monitor treatment response over time. This review summarizes available and investigational therapies for ATTR-CM and considers practical questions that guide clinical decision-making, with the goal of helping clinicians navigate the evolving therapeutic landscape."

Journal • Review • Amyloidosis • Cardiac Amyloidosis • Cardiomyopathy • Cardiovascular • Congestive Heart Failure • Heart Failure • Transplantation

Impact of retinol-binding protein changes on tafamidis treatment response in patients with transthyretin cardiac amyloidosis (PubMed, Zhonghua Xin Xue Guan Bing Za Zhi) - "Decline in RBP during treatment (ΔRBP<0) is associated with poorer response to tafamidis treatment. Monitoring RBP dynamics may assist clinicians in assessing disease severity and therapeutic response in ATTR-CA patients."

Journal • Retrospective data • Amyloidosis • Cardiac Amyloidosis • Cardiovascular • Rare Diseases

Tafamidis: A Game Changer in Transthyretin Cardiomyopathy? A Systematic Review and Meta-Analysis of Safety and Efficacy. (PubMed, Curr Probl Cardiol) - "Tafamidis treatment in ATTR-CM is associated with reduced all-cause mortality and a lower incidence of CHF exacerbations. These observations are consistent with the ATTRACT trial, which supports the efficacy of tafamidis in treating ATTR-CM."

Journal • Retrospective data • Review • Amyloidosis • Cardiomyopathy • Cardiovascular • Congestive Heart Failure • Heart Failure

Description of peripheral nervous system involvement in wild-type transthyretin amyloidosis: A clinical and electrophysiological study. (PubMed, Rev Neurol (Paris)) - "In our cohort, a large majority of ATTRwt patients showed peripheral nerve involvement, generally expressed as the combination of different neurological syndromes (entrapment syndromes, large and/or small fiber polyneuropathy, radicular impairment). Polyneuropathy in ATTRwt seemed stable over our follow-up period. Clinical and EDX screening should be conducted in ATTRwt patients with symptoms suggestive of neurological involvement in order not to oversee these frequent complications which could affect therapeutic decision making."

Journal • Amyloidosis • Cardiac Amyloidosis • CNS Disorders • Musculoskeletal Pain • Pain

Serial extracellular volume quantification using cardiac magnetic resonance imaging in transthyretin amyloidosis patients treated with tafamidis. (PubMed, Eur Radiol) - "Question What is the prognostic significance of serial ECV quantification in transthyretin amyloidosis patients on tafamidis treatment? Findings Change of ECV is a strong predictor of a combined endpoint consisting of all-cause mortality, cardiac transplantation, or hospitalization due to HF. Clinical relevance Patients with increasing ECV might potentially benefit from a change in amyloid-specific treatment to a different agent."

Journal • Amyloidosis • Cardiac Amyloidosis • Cardiovascular • Congestive Heart Failure • Heart Failure • Transplantation

Comparing Effects of Tafamidis in Controlling Left Ventricular and Left Atrial Strains in Patients With Wild-Type Transthyretin Amyloid Cardiomyopathy. (PubMed, Circ Rep) - "However, significant deterioration of GLS (-9.3 [-11, -7.4] to -8.0 [-9, -6.7]; P=0.0381) and LASr (11 [6, 16] to 6 [5, 11]; P=0.0074) were observed in patients with ATTRwt-CM without tafamidis. The LA and LV functions of patients with ATTRwt-CM treated with tafamidis were more favorable than those of untreated patients."

Journal • Amyloidosis • Cardiac Amyloidosis • Cardiomyopathy • Cardiovascular

Transthyretin-related amyloid cardiomyopathy: A single-center experience in southern Poland, an endemic area. (PubMed, Kardiol Pol) - P=N/A | "A comprehensive diagnostic approach is crucial for timely initiation of disease-modifying therapies. Our study suggests that, in this region, there is a high rate of the Phe53Leu TTR variant; however, further research is needed to validate these findings (NCT05814380)."

Journal • Amyloidosis • Cardiac Amyloidosis • Cardiomyopathy • Cardiovascular • Pain