Primary Systemic Amyloidosis Treatment & Management
- Author: Judit Nyirady, MD, MBA; Chief Editor: Dirk M Elston, MD more...
The treatment of primary systemic amyloidosis (PSA) is directed toward the affected organ and the specific type of the disease. In studies of different regimens of intermittent oral melphalan and prednisone, Skinner et al and Kyle et al reported that the response rates were low, with an increased survival from a median of approximately 7-9 months in patients who did not receive chemotherapy to approximately 12-18 months in those receiving chemotherapy.[12, 22]
Shimojima et al reported a patient with primary systemic amyloidosis who achieved partial hematological response after 2 courses of the VAD (vincristine, doxorubicin [Adriamycin], and dexamethasone) chemotherapy regimen and subsequent high-dose melphalan followed by autologous peripheral blood stem cell transplantation despite involvement of multiple organs, including the heart. When amyloidosis-related dysfunction is seen in multiple organs, intensive chemotherapy might be a possible therapeutic option, although several modifications in the regimen and careful management are necessary.
The nephrotic syndrome requires supportive therapy and diuretics, and renal failure can be successfully treated with dialysis.
Congestive heart failure may respond to diuretics, but larger doses are often required as the disease progresses. The use of calcium channel blockers, beta-blockers, and digoxin are contraindicated in cardiac amyloidosis, because they may cause toxicity at therapeutic levels.
Gastrointestinal involvement and neuropathy are treated symptomatically.
The presence of diffuse skin infiltrates and the loss of scalp hair can be distressing.
Macroglossia may be associated with painful dysphagia.
Cardiac involvement may cause angina, infarction, arrhythmias, or orthostatic hypotension.[24, 25]
Congestive heart failure or arrhythmias account for death in about 40% of the cases of primary systemic amyloidosis.
Blood vessel infiltration may lead to claudication of the legs or jaw. A case of temporal arteritis also has been described.
Amyloid infiltration of the gastrointestinal tract may result in hemorrhage that can cause malabsorption or even death.
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