From the Guidelines
The management plan for a patient with AL (lambda-type) amyloidosis diagnosed from a colonoscopy tissue biopsy should begin with a comprehensive evaluation to determine disease extent and organ involvement, and treatment typically involves chemotherapy targeting the underlying plasma cell dyscrasia, with standard first-line therapy being a combination of bortezomib, cyclophosphamide, and dexamethasone (CyBorD regimen) 1.
Initial Assessment
Initial assessment should include:
- Serum and urine protein electrophoresis
- Free light chain assay
- Cardiac biomarkers (troponin, BNP)
- Echocardiogram
- 24-hour urine protein
- Complete blood count with renal function tests
Treatment
Treatment typically involves chemotherapy targeting the underlying plasma cell dyscrasia, with standard first-line therapy being a combination of:
- Bortezomib (Velcade) at 1.3 mg/m² subcutaneously weekly
- Cyclophosphamide at 300 mg/m² orally weekly
- Dexamethasone 20-40 mg weekly for cycles of 28 days Alternative regimens may include daratumumab-based combinations or lenalidomide with dexamethasone 1.
Supportive Care
Supportive care is crucial and should address specific organ dysfunction, such as:
- Diuretics for heart failure
- Dialysis for renal failure if needed
- Nutritional support for GI involvement
Treatment Response
Treatment response should be monitored with serial free light chain assays, with the goal of achieving a complete hematologic response 1.
Prognosis and Treatment Approach
The prognosis and treatment approach depend on the extent of organ involvement, particularly cardiac involvement, which significantly impacts survival 1. Regular follow-up every 3-6 months is essential to monitor disease progression and treatment response.
From the Research
Management Plan for AL Amyloidosis
The management plan for a patient with amyloidosis AL (lambda)-type deposition diagnosed from a colonoscopy tissue biopsy involves a multidisciplinary approach.
- The initial therapeutic aim is to rapidly eliminate the clonal plasma cell that produces the circulating amyloid precursor and achieve a complete hematologic response, with minimal toxicity 2.
- Treatment is tailored to the initial risk assessment of the patients, and regimens adapted from the expanding options available for multiple myeloma patients are used 3, 2.
- The combination of daratumumab, cyclophosphamide, bortezomib, and dexamethasone (dara-CyBorD) is the current standard of care 4, 5.
- Autologous stem cell transplant can be considered in selected cases, although there is no robust evidence of superiority over chemotherapy alone 6, 5.
- In the relapsed/refractory setting, numerous promising therapies are still under investigation, including venetoclax and chimeric antigen receptor T-cell therapy (CART) targeting B-cell maturation antigen (BCMA) 5.
Diagnosis and Staging
- The diagnosis requires demonstration in a tissue biopsy of amyloid deposits formed by immunoglobulin light chains 4.
- Staging is based on cardiac and renal biomarkers and guides the choice of treatment 4.
- The extent of organ involvement is established by various staging and biomarkers testing 6.
Treatment Outcomes
- Response to therapy is assessed based on validated criteria, with a goal of achieving a very good partial response (VGPR) after 4-6 cycles of treatment 5.
- Organ response rates are strongly associated with deeper hematologic response but usually lag behind hematological response and are also dependent on the initial organ function reserve 2.
- Treatment outcomes have improved with the use of newer drugs, and survival has been prolonged from months to years 3.