Etanercept therapy in patients with advanced primary amyloidosis.

No effective treatment exists for primary amyloidosis, a plasma cell dyscrasia characterized by deposition of amyloid fibrils consisting of monoclonal light chains in various organs. TNF-alpha has been implicated in other amyloid disorders; therefore, we used etanercept to treat patients with advanced amyloidosis who had failed other therapies or were ineligible for other treatment regimens. Sixteen patients with amyloidosis that included patients with severe cardiac or multiple organ involvement were treated with etanercept and evaluated every 4-6 wk for evidence of toxicity and clinical response. Patients were treated with etanercept for a median of 42 wk. Eight of 16 patients (50%) experienced objective improvements and 14 patients (88%) experienced subjective improvements in symptoms. Only one patient experienced an adverse effect attributable to etanercept. For the entire group, improvement in performance status was statistically significant (p = 0.001), estimated median survival is 24.2 mo, 8 of whom are still alive with a median survival is 26.6 mo. The 12 patients with any cardiac involvement had an estimated median survival of 24.2 mo. Six of those 12 patients are still alive, with a median survival is 26.6 mo. The group of eight patients with severe cardiac involvement showed an estimated median survival of 13.2 mo, three of whom are still alive with a median survival is 25.9 mo. The clinical observations in this group of advanced and relapsed/refractory patients are highly encouraging. For the group as a whole, median survival was 24.2 mo and improvement in performance status was highly significant. Median survival for the patients with severe cardiac involvement was 13.2 mo with 3/8 patients are alive with a median survival of 25+ mo. Moreover, there was a statistically significant improvement in patients' performance status. These results, even though in a small group of patients, suggest that etanercept may provide a new therapeutic option for the management of amyloidosis that should be studied further.