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

• Published in: Haematologica (Roma) Vol. 94; no. 7; pp. 1029 - 1032
• Main Authors: Girnius, Saulius, Seldin, David C, Skinner, Martha, Finn, Kathleen T, Quillen, Karen, Doros, Gheorghe, Sanchorawala, Vaishali
• Format: Journal Article
• Language: English
• Published: Pavia Ferrata Storti Foundation 01-07-2009
• Subjects: Adult; Aged; Amyloidosis - drug therapy; Amyloidosis - therapy; Biological and medical sciences; Brief Reports; Female; Hematologic and hematopoietic diseases; Humans; Liver - pathology; Liver Diseases - complications; Liver Diseases - drug therapy; Male; Medical sciences; Melphalan - therapeutic use; Middle Aged; Myeloablative Agonists - therapeutic use; Retrospective Studies; Stem Cell Transplantation - methods; Treatment Outcome; Antineoplastic agent; Human; AL amyloidosis; Hematology; liver disease; Digestive system; Stem cell; Liver; stem cell transplantation; Hematopoietic cell; Hepatic disease; Dose activity relation; Alkylating agent; Nitrogen mustard; Melphalan; Digestive diseases; Graft; High dose
• ISSN: 0390-6078; 1592-8721
• DOI: 10.3324/haematol.2008.001925

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.