Submitted July 13, 2007
Accepted July 31, 2007
Long-term outcome of patients with AL amyloidosis treated with high-dose melphalan and stem cell transplantation
Vaishali Sanchorawala*, Martha Skinner, Karen Quillen, Kathleen T Finn, Gheorghe Doros, and David C Seldin
Department of Medicine, Stem Cell Transplantation Prog of the Sect of Hematology-Oncology, Boston Univeristy School of Med., Boston, MA, United States
Department of Medicine, Amyloid Treatment and Research Program, Boston Univeristy School of Med., Boston, MA, United States
Department of Pathology and Laboratory Medicine, Boston Univeristy School of Med., Boston, MA, United States
Department of Biostatistics, Boston Univeristy School of Public Health, Boston, MA, United States
* Corresponding author; email: vaishali.sanchorawala{at}bmc.org.
Long-term survival and outcome were determined for 80 patients with AL amyloidosis treated with high-dose melphalan and stem cell transplantation (HDM/SCT) more than 10 years ago. Seventeen patients (21%) died within the first year after treatment, of treatment related complications (14%) or progressive disease (8%). Of the 63 surviving evaluable patients at one year, 32 (51%) achieved a complete hematologic response (CR). For all 80 patients, the median survival was 57 months (4.75 yrs). The median survival exceeds 10 years for patients achieving a CR after HDM/SCT, compared to 50 months for those not achieving a CR (p<0.001). In conclusion, HDM/SCT leads to durable remissions and prolonged survival, particularly for those patients who achieve a hematologic CR.