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Abstract
Bortezomib combination chemotherapy appears to be active in light chain (AL) amyloidosis with high rates of hematologic and organ response. We report a retrospective evaluation of the clinical outcome of treatment with bortezomib, melphalan, and prednisolone (VMP) as first-line chemotherapy in patients with AL amyloidosis who were ineligible for autologous stem cell transplant. Among the 19 patients included in this study, 90% had two or more involved organs and most of the patients had advanced stage AL amyloidosis (84% with 2004 Mayo Stage III and 92% with 2012 Mayo Stage III or IV). Sixteen (84%) patients had a hematologic response, including seven (37%) with complete response, with time to response of 1–3 months. Cardiac and renal responses were observed in 44% and 33% of patients, respectively. Estimated 2-year survival is 39%, and 5 patients (26%) died during therapy. The common grade 3–4 adverse events were thrombocytopenia, diarrhea and pneumonia. A once-weekly bortezomib is more feasible than twice-weekly regimen. Our results suggest that triplet regimen of VMP appears to be an effective regimen in advanced AL amyloidosis ,but benefits in the patients with multi-organ dysfunction remain to be proven.