Vyndaqel (tafamidis meglumine) / Pfizer - LARVOL DELTA

Hereditary Transthyretin Amyloidosis in Brazilian Amazon Caused by a Globally Rare Variant Predominant in Asia. (PubMed, JACC Case Rep) - "p.Asp58Ala is a rare ATTR variant, previously described mainly in Asian populations. Its identification in the Amazon region suggests a possible previously unreported regional clustering in Brazil."

Journal • Amyloidosis • Cardiac Amyloidosis • Cardiomyopathy • Cardiovascular • Congestive Heart Failure • Genetic Disorders • Heart Failure

Hereditary Transthyretin Cardiac Amyloidosis With Severe Left Ventricular Outflow Tract Obstruction Mimicking Hypertrophic Obstructive Cardiomyopathy. (PubMed, JACC Case Rep) - "Transthyretin-cardiac amyloidosis may mimic hypertrophic obstructive cardiomyopathy with severe LVOT obstruction. Certain hereditary ATTR variants may demonstrate low-grade tracer uptake on bone scintigraphy. Multimodality imaging, genetic testing, and tissue confirmation are essential when clinical suspicion persists."

Journal • Amyloidosis • Cardiac Amyloidosis • Cardiomyopathy • Cardiovascular • Hypertrophic Cardiomyopathy • Obstructive Hypertrophic Cardiomyopathy • Pulmonary Disease

Transthyretin Amyloidosis Due to a Rare TTR Val142del Deletion With Relatively Early Clinical Manifestations. (PubMed, JACC Case Rep) - "Rare transthyretin deletions may cause aggressive hereditary transthyretin amyloidosis. Early recognition and family screening are essential."

Journal • Amyloidosis • Cardiac Amyloidosis • Cardiovascular • Congestive Heart Failure • Heart Failure • Hypotension • Monoclonal Gammopathy • Pain

Neurohormonal Therapy in Patients With Transthyretin Cardiac Amyloidosis: Is it an Option? (PubMed, JACC Case Rep) - "Guideline-directed medical therapy may be considered on an individualized basis in patients with wtATTR-CA and reduced LVEF. Some patients may tolerate treatment and experience improvement."

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

Donut Morphology of Cardiac Amyloidosis: A Glazed Over Diagnosis. (PubMed, JACC Case Rep) - "When clinical suspicion for ATTR-CM is high, negative CARI should prompt further evaluation rather than premature exclusion of the diagnosis."

Journal • Amyloidosis • Atrial Fibrillation • Cardiac Amyloidosis • Cardiovascular • Congestive Heart Failure • Heart Failure • Musculoskeletal Pain

Comparative Effectiveness of TTR Stabilizers for the Treatment of ATTR-CM Using Real-World Evidence. (PubMed, Cardiol Ther) - "In this first real-world comparative effectiveness study of newly treated patients, acoramidis had a significantly lower risk of DI events and composite events of DI, HFH, and mortality than tafamidis, potentially supporting improved clinical stability with acoramidis initiation. Additional evaluation with longer follow-up, larger cohorts, and/or prospective clinical outcomes is warranted. Graphical abstract available for this article."

HEOR • Journal • Real-world evidence • Amyloidosis • Cardiac Amyloidosis • Cardiomyopathy • Cardiovascular • Congestive Heart Failure • Heart Failure • Hematological Malignancies • Multiple Myeloma • Oncology

Case Report: Hereditary ATTR-CA presenting with recurrent severe abdominal cramps: a diagnostic challenge. (PubMed, Front Cardiovasc Med) - "She was treated with tafamidis but died suddenly only two months after diagnosis...In patients with ventricular hypertrophy, ATTR-CA may manifest with atypical symptoms such as severe, recurrent abdominal pain, which can result from autonomic neuropathy or gastrointestinal amyloid deposition. When the clinical diagnosis remains uncertain, multidisciplinary team consultation is essential to synthesize all available findings."

Journal • Amyloidosis • Cardiac Amyloidosis • Cardiovascular • Gastroenterology • Gastrointestinal Disorder • Hepatology • Pain • Psychosomatic Disorders

Specific treatment of ATTR-CM in Germany-expert consensus of the German Society of Amyloid Diseases (DGAK) and German Cardiac Society (DGK). (PubMed, Clin Res Cardiol) - "This consensus provides a practical framework for the specific treatment of ATTR-CM in Germany, aiming for early treatment initiation. Local standards and shared decision-making remain essential for the establishment of ATTR-CM therapy in an individual patient."

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

Case series of heart transplantation in advanced amyloid cardiomyopathy at a single centre (TTS 2026) - "A 54-year-old male with AL amyloidosis/myeloma was in complete remission with bortezomib/cyclophosphamide/daratumumab...Both patients 3 and 4 received compassionate access to a TTR stabiliser (Tafamidis) within 6 months post-HTx... We report a small case series of patients who received a heart transplant for advanced amyloid cardiomyopathy. This represents a heterogenous cohort and heart transplantation remains a possibility in carefully selected individuals. Characteristics of patients both pre and post heart transplantation"

Clinical • Metastases • Amyloidosis • Cardiac Amyloidosis • Cardiomyopathy • Cardiovascular • CNS Disorders • Congestive Heart Failure • Cytomegalovirus Infection • Heart Failure • Hematological Malignancies • Infectious Disease • Lymphoma • Multiple Myeloma • Septic Shock • Transplant Rejection • Transplantation

Neuropathy Progression in Variant Transthyretin Amyloidosis: Comparison Between TTR Stabilizers and Gene Silencer Therapies. (PubMed, Muscle Nerve) - "In this cohort, patisiran attenuated neuropathy progression in patients who had deteriorated despite tafamidis. These findings highlight the importance of ongoing clinical monitoring to guide timely treatment optimization."

Journal • Amyloidosis • Cardiac Amyloidosis • Pain

A trial emulation study indirectly comparing tafamidis and acoramidis in transthyretin amyloid cardiomyopathy: the ReplicATTR study. (PubMed, J Comp Eff Res) - " Acoramidis was linked to a lower estimated ACM risk in this emulated comparative analysis. These findings should be considered as hypothesis-generating, requiring confirmation in head-to-head studies."

Journal • Amyloidosis • Cardiac Amyloidosis • Cardiomyopathy • Cardiovascular

Molecular imaging of TTR amyloidosis: New data. (PubMed, Hell J Nucl Med) - "This narrative review synthesizes recent data on molecular imaging, emphasizing the central role of nuclear medicine physicians in applying multimodality diagnostic algorithms, recognizing typical and equivocal scan patterns. It further explores emerging roles for quantitative SPECT/CT and amyloid PET tracers in monitoring response to disease modifying therapies, including tetramer stabilizers (tafamidis, acoramidis), RNA interference silencers (vutrisiran, patisiran), and investigational fibril targeting antibodies and gene editing strategies, underscoring a shift from purely diagnostic imaging toward dynamic treatment monitoring in ATTR CM."

Journal • Review • Amyloidosis • Cardiac Amyloidosis • Cardiovascular • Congestive Heart Failure • Gastrointestinal Disorder • Heart Failure

Phenotypic Considerations for Tafamidis Use in a Real-World Cohort of Older Patients with Transthyretin Amyloid Cardiomyopathy. (PubMed, Cardiol Ther) - P, P3 | "Patients aged ≥ 80 years who were treated with tafamidis had outcomes comparable to those of younger patients when clinical vulnerability markers were considered. These findings support individualized treatment decisions based on patient's overall clinical profile and multidimensional assessment rather than age alone."

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

Proteomics Profiling Reveals Molecular Subtypes of Transthyretin Cardiac Amyloidosis. (PubMed, Circ Heart Fail) - "Subtype A had dysregulation of Ras/MAPK (mitogen-activated protein kinase) pathways, pathways implicated in ATTR-CA pathogenesis, and metabolic/inflammatory pathways. Our prospective cohort study using comprehensive plasma proteomics profiling not only uncovered molecular ATTR-CA subtypes but also identified pathogenetic mechanisms associated with differential prognosis and estimated the treatment effect of tafamidis."

Journal • Amyloidosis • Cardiac Amyloidosis • Cardiomyopathy • Cardiovascular

Clinical Factors Related to Cardiac Conduction Disturbance in Patients With Wild-Type Transthyretin Amyloid Cardiomyopathy Undergoing Tafamidis Treatment. (PubMed, Circ Rep) - "In patients with wild-type ATTR-CM undergoing tafamidis treatment, AVCD and IVCD progress over 1 year. Baseline T1native and hs-cTnT are predictors of progression of IVCD under tafamidis treatment, and T1native is associated with AVCD and IVCD in disease-modifying therapy-naïve conditions."

Journal • Amyloidosis • Cardiac Amyloidosis • Cardiomyopathy • Cardiovascular

Frailty is a mortality risk factor in tafamidis-treated patients with transthyretin cardiac amyloidosis. (PubMed, Amyloid) - "Frailty assessed using the SEGA independently predicts mortality in ATTR-CM patients treated with tafamidis and improves prognostic stratification. It may also help identify potentially modifiable vulnerabilities warranting personalized multidisciplinary care."

Journal • Amyloidosis • Cardiac Amyloidosis • Cardiovascular • Geriatric Disorders

Discordance of 99mTc-PYP Uptake in the Myocardium and Breast Both Before and After Treatment in Transthyretin Cardiac Amyloidosis. (PubMed, Clin Nucl Med) - "99mTc-PYP scintigraphy also reveals deposits of wild-type amyloid transthyretin (ATTRwt) in the myocardium and extracardiac sites, such as subcutaneous fat, skeletal trunk muscles, and alimentary tract, of patients with ATTRwt cardiomyopathy (CM). We describe an 87-year-old man with ATTRwt-CM in whom myocardial 99mTc-PYP uptake was reduced and mammary tracer uptake was increased due to gynecomastia after treatment with a disease-modifying drug for ATTRwt-CM, tafamidis, and heart failure medications, including spironolactone."

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

Diagnostic Delay In Transthyretin Cardiac Amyloidosis Is Common And Associated With Increased Mortality And Missed Diagnostic Opportunity (HFSA 2026) - "The clinical consequences and system-level contributors to delayed diagnosis are incompletely defined.Hypothesis: Diagnostic delay from heart failure symptom onset to Tc-PYP-confirmed ATTR-CM is associated with worse survival and reflects missed opportunities for earlier recognition. We performed a single-center retrospective study of 161 patients with ATTR-CM treated with tafamidis... Diagnostic delay in ATTR-CM is common and independently associated with mortality and increased hospitalization. A high burden of preceding red-flag symptoms, limited echocardiographic recognition, and infrequent strain utilization despite documented suspicion, are consistent with delayed clinical recognition and a missed window, rather than treatment access as a rate-limiting step. Structured echocardiographic and workflow-based identification strategies may represent targets to improve outcomes."

Amyloidosis • Cardiac Amyloidosis • Cardiovascular • Congestive Heart Failure • Heart Failure • Musculoskeletal Pain

Long-Term Vutrisiran Reduces Cardiovascular Events And Hospitalizations In Patients With Transthyretin Cardiac Amyloidosis In The HELIOS-B One-Year Open Label Extension (HFSA 2026) - "Patients were assessed as the overall population (all patients), the monotherapy population (patients not on tafamidis at baseline; tafamidis drop-in therapy was allowed at any time) and the baseline tafamidis subgroup (patients receiving tafamidis at baseline). At rollover into OLE, patients in the monotherapy population receiving placebo during the DB (placebo-to-vutrisiran) had more advanced disease than those initially randomized to vutrisiran (long-term vutrisiran) or those in the baseline tafamidis subgroup (Table). These data demonstrate that long-term vutrisiran continues to reduce the risk of CV events and hospitalizations relative to patients originally randomized to placebo. Reduction in non-CV hospitalizations may suggest a systemic effect of therapy. Many treatment effects at 48M were larger than those previously reported at 36M, highlighting the benefits of early diagnosis and treatment."

Clinical • Amyloidosis • Cardiac Amyloidosis • Cardiomyopathy • Cardiovascular • Congestive Heart Failure • Heart Failure

Beyond The Heart: Extra-cardiac Manifestations Enabling Early Diagnosis Of Transthyretin Cardiomyopathy Using 1-124 Evuzamitide Imaging (HFSA 2026) - "TTR gene sequencing showed no pathogenic mutations. He was started on tafamidis, and after 6 months of follow-up, remains clinically stable."

Amyloidosis • Cardiac Amyloidosis • Cardiomyopathy • Cardiovascular • Musculoskeletal Diseases • Musculoskeletal Pain • Orthopedics

Comparative Outcomes Of Tafamidis Plus Vutrisiran Versus Tafamidis Monotherapy In Transthyretin Amyloid Cardiomyopathy [Poster no. 570] (HFSA 2026) - No abstract available

Monotherapy • Amyloidosis • Cardiac Amyloidosis • Cardiomyopathy • Cardiovascular

Real-world Burden Of Transthyretin Amyloid Cardiomyopathy Disease And Outcomes In Patients On Tafamidis Before Switching To Acoramidis [Poster no. 484] (HFSA 2026) - No abstract available

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

Progressive Myocardial Dysfunction And Systemic Disease Burden Drive Outcomes In Octogenarian Transthyretin Cardiac Amyloidosis Treated With Tafamidis [Poster no. 424] (HFSA 2026) - No abstract available

Amyloidosis • Cardiac Amyloidosis • Cardiovascular

Sex Differences in a Contemporary, Real-World Cohort of Patients with Transthyretin Amyloid Cardiomyopathy Treated with Tafamidis. (PubMed, Cardiol Ther) - P, P3 | "In this analysis of contemporary patients with ATTR-CM treated with tafamidis, women represented a small proportion of the overall population, and a greater proportion had ATTRv-CM compared with men. Although women appeared more symptomatic at presentation, men had a higher risk of all-cause mortality."

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

Rising Diagnoses, Falling Mortality: A National Analysis of Cardiac Amyloidosis Hospitalizations Across the Tafamidis Era (AHA 2026) - "Abstract is embargoed at this time."

Clinical • Amyloidosis • Cardiac Amyloidosis • Cardiovascular