Bortezomib, melphalan, and prednisolone combination chemotherapy for newly diagnosed light chain (AL) amyloidosis

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.

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