1. ¹Hematology and Stem Cell Transplant Programs, Department of Medicine, Boston University Medical Center, Boston, MA, USA
2. ²Amyloid Treatment and Research Program, Department of Medicine, Boston University Medical Center, Boston, MA, USA

Correspondence: Dr V Sanchorawala, Boston Medical Center, Section of Hematology/Oncology, 88 East Newton Street, Boston, MA 02118, USA. E-mail: Vaishali.Sanchorawala@bmc.org

³Current address: The Hematology Service, Memorial Sloan Kettering Cancer Center, New York, NY, USA

Received 7 May 2003; Accepted 8 September 2003; Published online 15 December 2003.

Abstract

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.