High-dose melphalan versus melphalan plus dexamethasone in patients with AL amyloidosis
Anna G. Smirnova1, Alexey V. Smirnov2, Axel R. Zander3, Boris V. Afanasyev1
1Chair of Hematology, Transfusiology, and Transplantology, Raisa Gorbacheva Memorial Institute of Children Hematology and Transplantation; 2Chair of Internal Medicine, Pavlov State Medical University, St. Petersburg, Russia; 3Department of Stem Cell Transplantation, University Medical Center Hamburg-Eppendorf, Hamburg, Germany
Anna G. Smirnova, Memorial R. M. Gorbacheva Institute of Children Hematology and Transplantation, St. Petersburg Pavlov State Medical University, 6/8, Tolstoy str., St. Petersburg, 199044, Russia
AL amyloidosis is a clonal plasma cell dyscrasia related to multiple myeloma, in which monoclonal light chains transform to amyloid deposit in various tissues and lead to progressive organ dysfunction. The prognosis is poor and median survival is about 1 year in untreated patients. This study assessed whether high-dose melphalan followed by autologous hematopoietic stem-cell transplantation (AHSCT) improves the outcome of these patients.
Patients and methods
Between June 2002 and February 2009, 36 patients with AL amyloidosis were included: 20 males, 16 females. The median age was 57 y.o. The number of involved organs ranged from 1 to 5, 17 (47%) patients had more than 2 organs involved and 26 (72%) had cardiac involvement. All patients younger than 70 y.o. with a performance status of 0–2 by ECOG were aimed to AHSCT. Patients ineligible for ASCT received melphalan and dexamethasone.
Seventeen patients received AHSCT (conditioning regimen was melphalan 100–200 mg/m2), 11 chemotherapy (melphalan+dexamethasone), and 8 died before treatment.
After a median follow-up of 36 months, the 3-year overall survival (OS) was 70% and 30% in the AHSCT and chemotherapy groups, respectively (p=0.00) (Fig. 1). Progression-free survival (PFS) of 1.5 was also better in the AHSCT group: 40% and 10%, respectively (p=0.007). The main factors that decreased OS were cardiac involvement and a performance status of more than 2 by ECOG at the time of diagnosis. Age, number of involved organs, and melphalan dose did not have an impact on OS and PFS in our study.
AL amyloidosis is usually associated with poor health status, rapid disease progression, and involvement of multiple organs. Survival depends on:
• cardiac involvement
• the general health of the patient
• the intensity of the treatment
AL amyloidosis, treatment, melphalan, hematopoietic stem-cell transplantation