ABSTRACT
Primary systemic amyloidosis is an aggressive plasma cell dyscrasia whose prognosis is largely dependent on the extent of heart involvement and the number of clinically involved organs. Present therapy is aimed to the annihilation of the marrow plasma cell clone. Several groups experimented high-dose melphalan with autologous peripheral blood transplantation (HDCT) with high response rates and apparently prolonged survival. However, HDCT was characterized by severe toxicity and high transplant related death rates. Hematologic response was assessed by immunofixation of serum and urine using high-resolution agarose gel electrophoresis and by bone marrow morphological examination. Complete response was defined by negative immunofixation with plasma cell infiltration <5%. Partial response was characterized by more than 50% reduction in monoclonal component concentrations in serum and urine. However, the risks of death and transplant morbidity are still substantially higher than in other hematological malignancies, stressing the importance of accurate selection of candidates and the need for less toxic therapies.
