Patients with
AL amyloidosis were treated with VAD (
vincristine,
doxorubicin and
dexamethasone) with or without high-dose
melphalan followed by auto-PBSCT according to eligibility criteria based on disease severity, and prospectively investigated the therapeutic benefits and complications. Thirty-one patients were enrolled in this study. VAD and subsequent high-dose
melphalan with auto-PBSCT were performed only in patients who met all of the eligibility criteria. Among patients ineligible for this treatment, VAD alone was performed in those with satisfactory general status. Eleven patients met the eligibility criteria, and of these, 7 were treated with VAD and subsequent high-dose
melphalan with auto-PBSCT. Seven patients received VAD alone, and the remaining 17 were treated with the supportive
therapy. Among the 14 patients treated with
chemotherapy, 9 (5 of the 7 treated with VAD and high-dose
melphalan, and 4 of the 7 treated with VAD alone) showed complete hematological response with apparent improvement of
amyloidosis-related clinical symptoms. Serious complications of
chemotherapy were
cytomegalovirus infection and
pneumocystis carinii pneumonia seen in 1 and 2 patients, respectively. These
chemotherapies may be effective for reduction of M-
protein and are also useful in improving of
amyloidosis-induced organ dysfunction. In patients who cannot tolerate high-dose
melphalan, VAD alone is a potent therapeutic option, although there are possible harmful effects on the heart and peripheral nerve.