Response Rates and Survival in Primary Systemic Amyloidosis

Abstract

There were 153 patients with biopsy-proven primary systemic amyloidosis (AL) evaluated for their response to alkylating agent based chemotherapy. There were 27 patients (18 percent) who responded. The serum creatinine had an adverse effect on response rate (p = 0.05). In patients with nephrotic syndrome, a normal serum creatinine value, and no echocardiographic evidence of cardiac amyloidosis, the response rate was 39 percent (12 of 31). 0f 34 patients with amyloid cardiomyopathy, 5 responded. Two of these five are alive ten years after diagnosis. None of the 18 patients with amyloid peripheral neuropathy showed regression of disease. The median survival of the 27 patients was 89.4 months and 21 of 27 survived five years (78 percent). Eight patients remain alive with a minimum follow-up of 90 months. In the group of 126 patients who showed no response to therapy, the median survival was 14.7 months and 9 (7 percent) survived >5 years. All 126 patients have died. Alkylating agent based chemotherapy for AL is beneficial in a subset of patients.