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Sydney 2005:
Amyloidosis
By Philip Hawkins, MD
04.12.05
Dr. Hawkins’ presentation focused on AL amyloidosis, which is a disorder of protein folding. This disease is challenging to diagnose and treat. Diagnosis is made through a biopsy and histological analysis. Underlying monoclonal gammopathies are subtle and some patients present with concurrent MM or other B-cell malignancy. Median survival for untreated patients is less than 2 years. Symptoms of AL amyloidosis include renal dysfunction, neuropathy, and hepatomegaly; cardiac involvement is often missed. There are very few clinical trials on AL amyloidosis; however, various studies suggest that chemotherapy is effective. Treatment options include the use of infusion and stem cell transplant to suppress free light chain (FLC) production. FLC levels correlate with the course and outcome of AL amyloidosis and treatment should be guided based on the measurements of serum FLC. Suppression of FLC by just 50% leads to a better outcome. UK guidelines support the use of VAD as front-line therapy with peripheral blood stem cell transplantation (PBSCT) considered for select eligible patients. Other treatment options include the use of novel agents, which are currently being studied in patients.


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