Gastrointestinal Amyloidosis: Approach to Treatment

The main treatment goals in amyloidosis are twofold: 1) to diagnose the underlying disease state accurately to guide effective primary therapy (if available) and 2) to ameliorate symptoms. The correct diagnosis is essential because disease-modifying therapies vary widely according to the underlying primary pathology. Primary treatment options remain limited. The best evidence is for high-dose chemotherapy, followed by autologous stem cell transplantation in patients with primary systemic amyloidosis. High-flux hemodialysis (HD) may prevent HD-related amyloidosis. Liver transplantation may be an option for patients with familial amyloidotic polyneuropathy. Several novel specific therapies are under investigation, including small molecule drugs and vaccines. Their efficacy and safety in humans remain to be demonstrated. In the absence of specific cures, symptom-directed therapy assumes a paramount role and can improve quality of life by mitigating diarrhea or pain, for example.