ABSTRACT
Amyloid light chain (AL) amyloidosis is a rare disease with an incidence of 5–13 patients/million/year. The disease is the result of a clonal plasma cell expansion, in which monoclonal light chains transformed to amyloid can be deposited in various tissues leading to organ dysfunction and organ failure. The median survival without therapy is 10–14 months from diagnosis. With high-dose melphalan (HDM) and autologous stem cell transplantation (ASCT) haematological and clinical remission rates in approximately 50% of treated patients could be reported in several phase II studies. HDM followed by ASCT appears to prolong survival in patients, in whom haematological remission can be reached. The treatment toxicity of HDM is on the other hand known to be severe with mortality rates of 15–40%. Nephrotic syndrome is a frequent manifestation of AL amyloidosis. Untreated amyloidosis is in almost all cases a progressive and fatal disease.
