High-dose intravenous melphalan and autologous stem cell transplantation as initial therapy or following two cycles of oral chemotherapy for the treatment of AL amyloidosis: results of a prospective randomized trial.

Autor: Sanchorawala, V., Wright, D. G., Seldin, D. C., Falk, R. H., Finn, K. T., Dember, L. M., Berk, J. L., Quillen, K., Anderson, J. J., Comenzo, R. L., Skinner, M.
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Zdroj: Bone Marrow Transplantation; Feb2004, Vol. 33 Issue 4, p381-388, 8p, 4 Charts, 4 Graphs
Abstrakt: Summary:A prospective randomized trial was conducted to study the timing of high-dose intravenous melphalan and autologous stem cell transplantation (HDM/SCT) in AL amyloidosis. In all, 100 newly diagnosed patients were randomized to receive HDM/SCT, either as initial therapy (Arm-1) or following two cycles of oral melphalan and prednisone (Arm-2). The objectives of the trial were to compare survival and hematologic and clinical responses. With a median follow-up of 45 months (range 24-70), the overall survival was not significantly different between the two treatment arms (P=0.39). The hematologic response and organ system improvements after treatment did not differ between the two groups. Fewer patients received HDM/SCT in Arm-2 because of disease progression during the oral chemotherapy phase of the study, rendering them ineligible for subsequent high-dose therapy. This affected patients with cardiac involvement particularly, and led to a trend for an early survival disadvantage in Arm-2. Hence, newly diagnosed patients with AL amyloidosis eligible for HDM/SCT did not benefit from initial treatment with oral melphalan and prednisone, and there was a survival disadvantage for patients with cardiac involvement if HDM/SCT was delayed by initial oral chemotherapy.Bone Marrow Transplantation (2004) 33, 381-388. doi:10.1038/sj.bmt.1704346 Published online 15 December 2003 [ABSTRACT FROM AUTHOR]
Databáze: Complementary Index