Myeloablative chemotherapy with stem cell rescue for the treatment of primary systemic amyloidosis: a status report

Stem cell transplantation has been incorporated in the treatment of primary systemic amyloidosis for 5 years. Results reported to date suggest Chat th e response rates are substantially better than those for patients treated w ith low-dose traditional melphalan and prednisone chemotherapy, Unexpectedl y high mortality rates have, however, been reported with stem cell transpla ntation, reaching 40% in some series, This unexpectedly high mortality appe ars to be related to multiorgan failure of tissues infiltrated with amyloid deposits. Deaths have been reported from gastrointestinal tract hemorrhage , gastrointestinal tract perforation, sudden cardiac death, and renal failu re. The best patient for transplantation appears to have single organ invol vement, an age <55 years, the absence of renal insufficiency, and no sympto matic cardiac dysfunction, Patients eligible to receive stem cell transplan t represent a highly selected population, and before conclusions about the efficacy of transplantation are drawn, comparison with a matched control gr oup is necessary. Amyloidosis should be considered an indication for stem c ell transplantation in the context of a clinical trial so that results can be compiled and reported for an accurate assessment of response rate, survi val, relapse rates and treatment-related toxicities.