TREATMENT OF AL AMYLOiDOSIS WITH TANDEM CYCLES OF HIGH DOSE MELPHALAN AND AUTOLOGOUS STEM CELL TRANSPLANTATION: FINAL ANALYSIS OF A PROSPECTIVE TRIAL

摘要

AL or primary systemic amyloidosis is characterized by widespread deposition of amyloid fibrils, derived from monoclonal immunoglobulin light chains as a result of a clonal plasma cell dyscrasia. In the last decade, high-dose melphalan and autologous stem cell transplantation (HDM/SCT) has been shown to induce hematologic and clinical remissions and prolong survival in patients with AL amyloidosis (1), Moreover, the treatment outcomes following HDM/SCT are related to whether or not a complete hematologic response, defined as disappearance of the underlying monoclonal gammopathy from serum and urine by immunofixation electrophoresis and of clonal plasma cell dysrasia by bone marrow biopsy, is achieved,. The median survival of patients who achieve a hematologic complete response (CR) is greater than 8 years compared to 5,2 years for those who do not achieve a CR. Furthermore, clinical improvements in affected organ systems occur in 66% of patients achieving a hematologic CR compared to only 30% of those who do not. Similar differences with respect to improvements in performance status were observed in association with hematologic CR.