Published online 19 May 2009
Haematologica, Vol 94, Issue 7, 1029-1032 doi:10.3324/haematol.2008.001925
Copyright © 2009 by Ferrata Storti Foundation
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Amyloidosis

Hepatic response after high-dose melphalan and stem cell transplantation in patients with AL amyloidosis associated liver disease

Saulius Girnius1, David C. Seldin2,3, Martha Skinner3, Kathleen T. Finn2,3, Karen Quillen2,4, Gheorghe Doros3,5, Vaishali Sanchorawala2,3

1 Department of Medicine
2 Stem Cell Transplantation Program of the Section of Hematology-Oncology and
3 Amyloid Treatment and Research Program, Department of Medicine;
4 Department of Pathology and Laboratory Medicine, Boston University School of Medicine and
5 Department of Biostatistics, Boston University School of Public Health, Boston, MA, USA

Correspondence: Vaishali Sanchorawala, MD, Section of Hematology/Oncology, FGH 1007, 820 Harrison Avenue, Boston, MA 02118, USA. E-mail:vaishali.sanchorawala{at}bmc.org

High-dose melphalan chemotherapy and autologous peripheral blood stem cell transplantation has been shown to result in durable hematologic response and prolonged overall survival in systemic AL amyloidosis. In this retrospective study, we evaluated clinical and hematologic responses in 69 patients with predominant liver involvement who were treated with high-dose intravenous melphalan and autologous stem cell transplantation from 1998 to 2006. Nine patients (13%) died from treatment-related mortality, similar to patients without hepatic involvement. The overall survival was 81% at one year and 61% at five years, by Kaplan-Meier estimates. A hematologic complete response was achieved by 53% (31/58) of patients at one year. A hepatic response occurred in 57% (33/58) at one year after high-dose intravenous melphalan and autologous stem cell transplantation and 63% (19/30) at two years after high-dose intravenous melphalan and autologous stem cell transplantation. In conclusion, hepatic disease improves in almost 2/3 patients treated with high-dose intravenous melphalan and autologous stem cell transplantation who have a complete or partial hematologic response to treatment.

Key words: AL amyloidosis, stem cell transplantation, liver disease.