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Trusted Resources: Education
Scientific literature and patient education texts
157 resources available:
Consensus recommendations on holistic care in hereditary ATTR amyloidosis: an international Delphi survey of patient advocates and multidisciplinary healthcare professionals
Https://bmjopen.bmj.com Year: 2024
Full Article hATTR Holistic Care Consensus Reccomendations BMJ
Abstract
Background Hereditary transthyretin-mediated amyloidosis is a rare, progressive and potentially lif...
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myBinderTreatment of AA Amyloidosis
Amyloidosis.org.uk: Amyloidosis Patient Information Site
Principles of treatment
Treatment of all types of amyloidosis is currently based on the following principles:
Reducing the supply of amyloid forming precursor proteins.
Supporting the function of organs containing amyloid.
SAP scans in thousands of patients with various forms of amyloidosis have shown that when amyloid precursor protein supply is controlled:
existing amyloid...
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myBinderAA Amyloidosis FAQs
Amyloidosis Foundation
Why is it called AA amyloidosis?
In the past, AA amyloidosis was referred to as “Secondary” or “Inflammatory” amyloidosis. These are no longer accepted names for this form of amyloidosis, which is usually caused by a complication of chronic inflammation or chronic infection. If a patient has chronic inflammation or chronic infection due to a number of possible conditions or diseases, this inflammation can often trigger an inc...
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myBinderAA amyloidosis, or secondary amyloidosis, is one type of the rare disorder amyloidosis. This disorder happens when proteins in your body mutate, changing form and gathering on your organs and tissues. AA amyloidosis happens because you have a chronic inflammatory condition or disease. Healthcare providers treat AA amyloidosis by controlling the underlying disease or condition.
myBinderLong-term survival in people with transthyretin amyloid cardiomyopathy who took tafamidis: a plain language summary
Future Cardiology Year: 2023
What is this plain language summary about?
This summary presents the results from an ongoing, long-term extension study that followed an earlier study called ATTR-ACT. People who took part in this extension study an...
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myBinderIn this edition we hear from John Hicks and his experience of being a carer to his wife Joyce for the last 6 years. We also have an update from Gary Mines, who was previously featured in AL amyloidosis Matters in Summer 2017. He updates us with his experience of daratumumab (Darzalex®). Our living well section covers emotional support and its importance in AL amyloidosis.
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219 resources available:
From older to younger: intergenerational promotion of health behaviours in Portuguese families affected by familial amyloid polyneuropathy
European Journal of Human Genetics Year: 2017
The role of older generations in families with hereditary diseases has been recognised and associated to their function as guardians of the family’s medical history. However, research is scarce in examining the roles that older generations play in terms of health promotion and risk management towards younger generations, which is particularly evident with incurable genetically inherited disorders such as familial amyloid polyneuropathy (FAP)...
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myBinderLong-Term Survival With Tafamidis in Patients With Transthyretin Amyloid Cardiomyopathy
Circulation: Heart Failure Year: 2021
Background: Tafamidis is approved in many countries for the treatment of transthyretin amyloid cardiomyopathy. This study reports data on the long-term efficacy of tafamidis from an ongoing long-term extension (LTE) to the pivotal ATTR-ACT (Tafamidis in Transthyretin Cardiomyopathy Clinical Trial).
Methods: Patients with transthyretin amyloid cardiomyopathy who com...
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myBinderImpact of Earlier Diagnosis in Cardiac ATTR Amyloidosis Over the Course of 20 Years
Circulation Year: 2022
Background: Diagnostic and therapeutic advances have led to much greater awareness of transthyretin cardiac amyloidosis (ATTR-CA). We aimed to characterize changes in the clinical phenotype of patients diagnosed with ATTR-CA over the past 20 years.
Methods: This is a retrospective observational cohort study of all patients referred to the National Amyloidosis Centre (2002-2021) in whom ATTR-CA was a di...
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myBinderPredictive Modeling to Assess Pretest Probability of Transthyretin Gene Variants Based on Demographic Information
Circulation: Heart Failure Year: 2023
Background:
Transthyretin amyloid cardiomyopathy (ATTR-CM) is a morbid condition, though recent advances in diagnosis and therapy stand to change its natural history. Patients’ TTR genotype may guide family screening as more treatments and preventive strategies become available. An efficient, intuitive means of determining pretest genetic risk may better inform patients/clinicians when pursuing genetic testing.
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myBinderCurrent Approaches to the Diagnosis and Management of Amyloidosis
Internal Medicine Journal Year: 2022
Amyloidosis is a collection of diseases caused by the misfolding of proteins that aggregate into insoluble amyloid fibrils and deposit in tissues. While these fibrils may aggregate to form insignificant localised deposits, they can also accumulate in multiple organs to the extent that amyloidosis can be an immediately life-threatening disease, requiring urgent treatment. Recent advances in diagnostic techniques and therapies are dramatically c...
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myBinderEfficacy and Safety of Vutrisiran for Patients With Hereditary Transthyretin-Mediated Amyloidosis With Polyneuropathy: A Randomized Clinical Trial
Amyloid: The Official Journal of the International Society of Amyloidosis Year: 2022
Background: The study objective was to assess the effect of vutrisiran, an RNA interference therapeutic that reduces transthyretin (TTR) production, in patients with hereditary transthyretin (ATTRv) amyloidosis with polyneuropathy.
Methods: HELIOS-A was a phase 3, global, open-label study comparing the efficacy and safety of vutrisiran with an external placebo group (APOLLO study). Patients were random...
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11 resources available:
Comprehensive Review of AL Amyloidosis: Some Practical Recommendations
Springer Nature Limited Year: 2021
Amyloid light chain (AL) amyloidosis is among the more common and more severe of the amyloidoses usually involving the slow proliferation of a bone-marrow-residing plasma cell (PC) clone and the secretion of unstable immunoglobulin-free light chains (FLC) that infiltrate peripheral tissues and result in detrimental end-organ damage. Disease presentation is rather vague, and the hallmark of treatment is early diagnosis before irreversible end-o...
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These guidelines were written after an extensive literature search using MedLine publications from January 1980 to April 2021, under the terms “ATTR” AND “transthyretin” AND “amyloidosis”. Only full articles in English were considered. Following the literature search and review, the steering committee developed a summary of relevant statements that were evaluated by all the participants. Consensus was established using online, sing...
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myBinderGuidelines for High Dose Chemotherapy and Stem Cell Transplantation for Systemic AL Amyloidosis: EHA-ISA Working Group Guidelines
Amyloid: The Official Journal of the International Society of Amyloidosis Year: 2021
AL amyloidosis is a systemic amyloidosis and is associated with an underlying plasma cell dyscrasia. High dose intravenous melphalan and autologous stem cell transplantation was developed for the treatment of AL amyloidosis in the early 1990s and was prompted by its success in multiple myeloma. This application has evolved significantly over the past three decades. These guidelines provide a comprehensive assessment of eligibility criteria, st...
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myBinderGuidelines and New Directions in the Therapy and Monitoring of ATTRv Amyloidosis
Amyloid: The Official Journal of the International Society of Amyloidosis Year: 2022
The recent approval of three drugs for the treatment of amyloid transthyretin (ATTR) amyloidosis, both hereditary and wild-type, has opened a new era in the care of these diseases. ATTR amyloidosis is embedded in its pathophysiology, and the drugs target critical steps of the amyloid cascade. In addition to liver transplant, which removes the pathogenic variants, the introduction of gene silencers has allowed the suppression of both wild type...
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myBinderNutrition Guidelines for Patients: Hereditary Transthyretin Amyloidosis (Spanish)
Alnylam Pharmaceuticals, Inc. Year: 2020
Adopting simple, healthy habits is one of the most influential factors in developing a balanced lifestyle. Nutrition affects overall physical and mental health and gains further importance when suffering from a disease. Promoting good nutrition may improve the consequences that illness or medication has on our bodies.
Food is an art in our culture. We can look after ourselves and play a socially active role around...
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myBinderASNC/AHA/ASE/EANM/HFSA/ISA/SCMR/SNMMI Expert Consensus Recommendations for Multimodality Imaging In Cardiac Amyloidosis: Part 1 of 2-Evidence Base and Standardized Methods of Imaging
Journal of Nuclear Cardiology Year: 2019
Cardiac amyloidosis is a form of restrictive infiltrative cardiomyopathy that confers significant mortality. Due to the relative rarity of cardiac amyloidosis, clinical and diagnostic expertise in the recognition and evaluation of individuals with suspected amyloidosis is mostly limited to a few expert centers. Electrocardiography, echocardiography, and radionuclide imaging have been used for the evaluation of cardiac amyloidosis for over 40...
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7 resources available:
RNA-Targeting and Gene Editing Therapies for Transthyretin Amyloidosis
Nature Reviews Cardiology Year: 2022
Transthyretin (TTR) is a tetrameric protein synthesized mostly by the liver and secreted into the plasma. TTR molecules can misfold and form amyloid fibrils in the heart and peripheral nerves, either as a result of gene variants in TTR or as an ageing-related phenomenon, which can lead to amyloid TTR (ATTR) amyloidosis. Some of the proposed strategies to treat ATTR amyloidosis include blocking TTR synthesis in the liver, stabilizing TTR tetram...
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myBinderThe Patient Voice: Development and Results of a Pilot Patient Experience Data Survey
International Studies Association Year: 2022
Background
Amyloidosis is a rare, systemic disease that is characterized by a variable pattern of nonspecific symptoms and affected organs. These characteristics of amyloidosis are common among rare diseases and make them difficult to diagnose and treat. The 2 main forms of cardiac amyloidosis are light-chain (AL) amyloidosis and transthyretin (ATTR) amyloidosis. One way to improve diagnosis is to increase awareness...
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myBinderReal-World Patient, Advocate, and Caregiver Perspectives on Amyloidosis: Awareness, Knowledge Gaps, and Psychosocial Impact
International Studies Association Year: 2022
Background
All types of amyloidosis are rare, progressive, and potentially fatal disorders that are difficult to diagnose and treat. Correct diagnosis of amyloidosis is often delayed due to the nonspecific nature of symptoms and misdiagnoses leading to delays in treatment initiation. This delay results in considerable medical and financial burden on patients and their caregivers. While standardized quality-of-life...
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myBinderSubcutaneous Daratumumab (DARA SC) Plus Cyclophosphamide, Bortezomib, and Dexamethasone (Cybord) in Patients (Pts) With Newly Diagnosed Amyloid Light Chain (AL) Amyloidosis: Safety Run-in Results of Andromeda
American Society of Clinical Oncology Year: 2018
Background:
Systemic AL amyloidosis is characterized by disposition of insoluble amyloid fibrils into tissues and organs via clonal expansion of CD38+ plasma cells. The safety run-in of DARA SC + CyBorD in ANDROMEDA (NCT03201965) is presented.
Methods:
Eligible pts had >=1 involved organs, ECOG score =<2, absolute neutrophil count >=1.0 × 109/L; hemoglobin >=8.0 g/dL; plate...
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myBinderDaratumumab-Based Therapies in Patients With AL Amyloidosis
American Society of Clinical Oncology Year: 2018
Background:
Treatment options for patients (pts) with relapsed/refractory (RR) AL amyloidosis are limited. Daratumumab (dara) has been approved as monotherapy (DMT) or combination therapy (DCT) for multiple myeloma (MM). Data for dara-based therapy (DBT) in AL are sparse.
Methods:
We studied pts with RR AL without coexisting MM seen at Mayo Clinic from 11/2015 to 02/2018 & treated w...
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myBinderImplanted Cardiac Defibrillator and Pacemaker Lead Function in Patient With Cardiac Amyloidosis
Journal of the American College of Cardiology Year: 2017
Introduction:
Cardiac amyloidosis (CA) results from myocardial infiltration of amyloid proteins that leads to restrictive cardiomyopathy, heart failure, and conduction abnormalities. Low QRS voltage on surface electrocardiography is characteristic of CA, raising concern for poor R-wave sensing and elevated pacing and defibrillation thresholds in CA patients with Implantable Cardioverter Defibrillator (ICD) or pacemak...
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15 resources available:
From older to younger: intergenerational promotion of health behaviours in Portuguese families affected by familial amyloid polyneuropathy – a plain language summary
Future Rare Diseases Year: 2023
What is this summary about?
This is a plain language summary of an article originally published in European Journal of Human Genetics. Transthyretin amyloidosis (ATTR) is a disease that affects the heart and nerves of those it afflicts. One inherited form of ATTR is particularly common among people of Portuguese descent and presents primarily as a neurologic disease termed familial amyloid polyneuropathy (FAP)....
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myBinderAmyloid Nomenclature 2022: Update, Novel Proteins, and Recommendations by the International Society of Amyloidosis (ISA) Nomenclature Committee
Amyloid: The Official Journal of the International Society of Amyloidosis Year: 2022
The Nomenclature Committee of the International Society of Amyloidosis met at the XVIII International Symposium on Amyloidosis in September and virtually in October 2022, with discussions resulting in this upgraded nomenclature recommendation. The nomenclature principles remain unchanged, but there is an ongoing discussion regarding the importance an...
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myBinderExpert Analysis and Opinion—Understanding Cardiac Amyloidosis
American College of Cardiology Foundation Year: 2021
Cardiac amyloidosis (CA) is a protein-folding disorder nearly exclusively caused by misfolded amyloid transthyretin (ATTR) and amyloid light chain (AL) proteins.
• Name derives from Latin “amylum” (starch like).
• Nomenclature: “A” for amyloid, followed by precursor protein abbreviation (e.g., AL = amyloid light chain amyloidosis).
• Histologic diagnosis: Aggregates of beta-sheets that sta...
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myBinderAmyloid Nomenclature 2020: Update and Recommendations by the International Society of Amyloidosis (ISA) Nomenclature Committee
Amyloid: The International Journal of Experimental and Clinical Investigation Year: 2020
The ISA Nomenclature Committee met electronically before and directly after the XVII ISA International Symposium on Amyloidosis, which, unfortunately, had to be virtual in September 2020 due to the ongoing COVID-19 pandemic instead of a planned meeting in Tarragona in March. In addition to confirmation of basic nomenclature, several additional concepts were discussed, which are used in scientific amyloid literature. Among such concepts are c...
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myBinderGenetic Testing and Counseling Program for Hereditary ATTR (hATTR) Amyloidosis Offered at No Charge – For Providers
Alnylam Pharmaceuticals, Inc. Year: 2020
Consider genetic testing and counseling for your patients; Alnylam Act® provides one option for eligible individuals.
What Is Alnylam Act®?
Alnylam Act® is a sponsored, no-charge, third-party genetic testing and counseling program for patients with a family history or suspected diagnosis of hATTR amyloidosis. The Alnylam Act® program was developed to reduce barriers to genetic testi...
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myBinderLay Summary of “TTR Gene Silencing Therapy in Post Liver Transplant Hereditary ATTR Amyloidosis Patients”
Amyloid: The International Journal of Experimental and Clinical Investigation Year: 2020
This is an article that was recently published in the journal “Amyloid” (June 2020). Researchers from different amyloid clinics across the US reported on their experience treating patients with hATTR amyloidosis, who already underwent liver transplant, with TTR “knock down” therapy.
The rationale is as follows:
1) some people with hATTR who undergo liver transplant continue to have disease pr...
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