Therapeutic strategies in patients with systemic AL-amyloidosis and renal involvement

Background and objective: Despite significant efficacy of melphalan and pre dnisone in the therapy of systemic AL(light chain amyloid)amyloidosis the p rognosis of the disease is poor. In patients with severe renal manifestatio n the reported results of low-dose melphalan therapy are inconclusive with respect to relief of clinical symptoms and overall prognosis. Patients and methods: We report our results of therapy in a group of 22 pat ients (8 women, 14 men, mean age 60 years) with renal involvement as the ma in manifestation of systemic AL-amyloidosis without overt myeloma. Results: Ten patients were treated with low doses of melphalan and predniso ne. No significant clinical improvement was observed in any case: the patie nts died an average of 12 months after diagnosis of the disease. Three pati ents were treated with high doses of melphalan followed by autologous stem cell transplantation. One patient died due to septicaemia after high-dose c hemotherapy. Two of the patients experienced significant re mission and liv e virtually free of clinical symptoms 12 and 18 months after therapy. Nine patients were treated only symptomatically: four of them were alive an aver age of 30 months after diagnosis of systemic AL-amyloidosis. Conclusion: Only high-dose melphalan therapy offered a realistic chance of amelioration of clinical symptoms in our group of patients, although therap y-associated risks seem to be high. In patients with severe renal amyloidos is, who are not considered for high-dose therapy, particularly careful cons ideration of potential benefits and possible risks of conventional melphala n therapy is necessary, because the results of this approach are in doubt.