High-dose melphalan with autologous stem cell transplantation after VAD induction chemotherapy for treatment of amyloid light chain amyloidosis: a single centre prospective phase II study.

摘要: Amyloid light chain (AL) amyloidosis is the result of a clonal plasma cell expansion, in which monoclonal chains transform to amyloid deposit various tissues and can lead organ dysfunction failure. The median survival patients with AL without therapy 10-14 months. With high-dose melphalan (HDM) autologous stem transplantation (ASCT), haematological clinical remission rates up 50% treated have been reported from phase II studies. HDM followed by ASCT appears prolong patients, if be reached. In this study, we evaluated vincristine, adriamycin dexamethasone (VAD) as induction chemotherapy prior mobilization ASCT. regimen was, general, feasible amyloidosis, but VAD had considerable World Health Organization (WHO) grade III-IV toxicity (25%) mortality (7%) rate. pretreatment did not interfere was possible 86% patients. overall treatment efficacy comparable results preceding chemotherapy. We could show an additional benefit terms increasing response rate; however 13% rate after our series lower than previous report.