From the Guidelines
Management of Amyloidosis Episodes Requires a Multidisciplinary Approach. The primary goals of care for patients with cardiac amyloidosis are improved survival and maximized quality of life, which can be achieved through a team-based approach involving physicians and advanced-practice providers across various specialties, including cardiology, nephrology, hematology, neurology, gastroenterology, and palliative care 1.
Key Considerations in Management
- Diagnosis: Accurate diagnosis of cardiac amyloidosis is crucial, and testing for ATTR amyloidosis has identified unrecognized cases of AL amyloidosis 1.
- Treatment: Treatment is largely supportive and includes management of heart failure, arrhythmias, and conduction system problems. However, standard heart failure treatment regimens can be challenging in patients with cardiac amyloidosis, and caution should be exercised with the use of diuretics, vasodilators, beta-blockers, and ACE inhibitors due to the risk of hypotension and worsening heart failure symptoms 1.
- Disease-Modifying Therapies: Disease-modifying therapies, such as TTR silencers (e.g., inotersen and patisiran), TTR stabilizers (e.g., diflunisal and tafamidis), and TTR disruptors (e.g., doxycycline and tauroursodeoxycholic acid), may be beneficial in slowing the progression of amyloidosis-related polyneuropathy and improving cardiovascular outcomes 1.
- Anticoagulation: Anticoagulation is a reasonable treatment strategy to reduce the risk of stroke in patients with cardiac amyloidosis and atrial fibrillation 1.
Specific Treatment Strategies
- AL Amyloidosis: Treatment of AL amyloidosis involves chemotherapy and transplant, and patients with cardiac involvement should be promptly referred to hematology-oncology specialists for timely treatment 1.
- ATTR Amyloidosis: Treatment of ATTR amyloidosis involves targeted therapy, such as tafamidis, and disease-modifying therapies, such as TTR silencers and stabilizers 1.
Important Considerations in Clinical Practice
- Autonomic Dysfunction: Evaluation and management of autonomic dysfunction are important in patients with cardiac amyloidosis 1.
- Volume Status: Judicious diuresis remains the mainstay of heart failure therapy in patients with cardiac amyloidosis, and careful management of volume status is crucial to avoid hypotension and worsening heart failure symptoms 1.
- Arrhythmia: Management of arrhythmias, including atrial fibrillation, is important in patients with cardiac amyloidosis, and anticoagulation may be necessary to reduce the risk of stroke 1.
From the Research
Management of Amyloidosis Episodes
The management of amyloidosis episodes involves various approaches, including:
- Diagnosis and identification of the amyloid subtype 2, 3, 4
- Treatment of the underlying disease, adequate organ support, and symptom management 4
- Use of specific therapies, such as proteosome inhibitors (bortezomib) and monoclonal antibodies (daratumumab) for AL amyloidosis 2, 5
- Emerging treatments for ATTR amyloidosis that slow disease progression, decrease hospitalizations, and improve quality of life and survival 2
- Consideration of autologous stem cell transplant in eligible patients, especially those who do not attain a satisfactory response to initial treatment 5
Treatment Options
Treatment options for amyloidosis depend on the subtype and may include:
- High-dose chemotherapy and stem cell transplant or bortezomib-based chemotherapy for AL amyloidosis 3
- Three therapies approved by the US Food and Drug Administration for managing ATTR amyloidosis, depending on clinical phenotype 3
- Novel therapeutic strategies targeting the formation of amyloid fibrils and amyloid deposition for AA amyloidosis 4
- Combination of daratumumab, cyclophosphamide, bortezomib, and dexamethasone (dara-CyBorD) as the current standard of care for AL amyloidosis 5
Diagnostic Techniques
Diagnostic techniques for amyloidosis include:
- Histological evidence of amyloid deposits 4
- (123)I-labeled serum amyloid P component scintigraphy, a safe and noninvasive technique that can identify anatomical patterns of amyloid deposition throughout the body 4
- Technetium scintigraphy, which has made noninvasive diagnosis of ATTR amyloidosis much easier 2
- Blood testing for staging and monitoring of the disease 3