Prospective randomized trial of melphalan and prednisone versus vincristine, carmustine, melphalan, cyclophosphamide, and prednisone in the treatmentof primary systemic amyloidosis
Ma. Gertz et al., Prospective randomized trial of melphalan and prednisone versus vincristine, carmustine, melphalan, cyclophosphamide, and prednisone in the treatmentof primary systemic amyloidosis, J CL ONCOL, 17(1), 1999, pp. 262-267
Purpose: primary systemic amyloidosis is an immunoglobulin deposition disor
der in which insoluble light chains cause organ dysfunction and death. The
established conventional therapy is treatment with melphalan and prednisone
. We investigated whether treatment with multiple alkylating agents improve
d the response rare or survival time, compared with melphalan and prednison
e therapy.
Patients and Methods: We treated 101 patients with biopsy-proven primary am
yloidosis. The patients were randomly assigned to receive melphalan and pre
dnisone (52 patients) or vincristine, carmustine, melphalan, cyclophosphami
de, and prednisone (49 patients). patients were stratified according to the
presence of cardiac involvement; time from diagnosis to randomization, ser
um beta(2)-microglobulin level, and whether peripheral neuropathy was the m
ajor manifestation of the disease.
Results: The median duration of survival after randomization was 29 months,
with no differences in survival time between the two groups. There were 29
patients who fulfilled the response criteria: 15 in the vincristine, carmu
stine, melphalan, cyclophosphamide, and prednisone arm and 14 in the melpha
lan and prednisone arm.
Conclusion: Therapy with multiple alkylating agents did not result in a hig
her response rate or longer survival time, compared with standard melphalan
and prednisone treatment in patients with primary systemic amyloidosis.
J Clin Oncol 17:262-267. (C) 1999 by American Society of Clinical Oncology.