Two patients with progressive primary amyloidosis, monoclonal serum and urinary proteins, multiple organ involvement, and nephrotic syndrome were treated with melphalan and prednisone for one year. In one patient, splenomegaly and nephrotic syndrome rapidly responded to therapy but massive hepatomegaly responded slowly, requiring 15 months' time for normalization of size. Results of liver function tests, although improved, remained abnormal, and amyloid deposits remained in the marrow. A second patient also demonstrated dramatic diminution in proteinuria and improvement in liver function abnormalities, but macroglossia persisted. These observations suggest that amyloid organ involvement may be reversible with differences in organ responsiveness to chemotherapy. An empirical trial of chemotherapy may be indicated in some patients with progressive primary amyloidosis, and therapy may need to be continued for a prolonged period of time before seeing an effect.
(Arch Intern Med 139:1144-1147, 1979)
Schwartz RS, Cohen JR, Schrier SL. Therapy of Primary Amyloidosis With Melphalan and Prednisone. Arch Intern Med. 1979;139(10):1144–1147. doi:10.1001/archinte.1979.03630470056018
Browse and subscribe to JAMA Network podcasts!
Customize your JAMA Network experience by selecting one or more topics from the list below.
Create a personal account or sign in to: