High dose melphalan therapy with stem-cell support in primary systemic amyloidosis

BACKGROUND: Primary systemic amyloidosis (AL) is a plasma cell dyscrasia ch aracterized by the extracellular deposition of immunoglobulines light chain s in different organs and systems. Median survival with current standard tr eatment is less than 2 years. Intensive chemotherapy followed by hematopoie tic stem cell rescue has been used in AL for the last five years. The repor ted results are encouraging, with a high response rate (65%). Nonetheless, this procedure is associated to an important toxicity, with high transplant related mortality (about 25%). In patients with advanced AL (more than two involved organs) and/or complicated cardiac disease, the mortality is part icularly high. In the current report we describe the outcome of the first f ive patients who received high dose therapy for AL at our institution. PATIENTS AND METHODS: This treatment was administered to patients up to the age of 70 years, who met the standard eligibility criteria for an autologo us bone marrow transplantation. Intensive treatment consisted of melphalan 200 mg/m(2) in all patients but one who received 140 mg/m(2). RESULTS: Two patients died during the first month after the transplant (arr ythmia: 1, multiorganic failure: 1). Both patients had a poor performance s tatus and advanced disease, with more than two organs involved. The respons e in the remaining three was of stabilization of the nephrotic syndrome in one and objective response in all the involved organs in the remaining two. CONCLUSIONS: High dose therapy (HDT) with stem cell rescue in AL produces a high response rate. Nevertheless, this treatment approach is associated to a high toxicity.