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Blood, 15 November 2007, Vol. 110, No. 10, pp. 3561-3563.
Prepublished online as a Blood First Edition Paper on August 2, 2007; DOI 10.1182/blood-2007-07-099481.
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CLINICAL TRIALS AND OBSERVATIONS
Brief Report
Long-term outcome of patients with AL amyloidosis treated with high-dose melphalan and stem-cell transplantation
Vaishali Sanchorawala1,2,
Martha Skinner2,
Karen Quillen1,3,
Kathleen T. Finn1,2,
Gheorghe Doros4, and
David C. Seldin1,2
1 Stem Cell Transplantation Program of the Section of Hematology-Oncology,
2 Amyloid Treatment and Research Program, Department of Medicine,
3 Department of Pathology and Laboratory Medicine, Boston University School of Medicine; and
4 Department of Biostatistics, Boston University School of Public Health, MA
Long-term survival and outcome were determined for 80 patients with immunoglobulin light chain (AL) amyloidosis treated with high-dose melphalan and stem cell transplantation (HDM/SCT) more than 10 years ago. Seventeen (21%) patients died within the first year of 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 with 50 months for those not achieving a CR (P < .001). In conclusion, HDM/SCT leads to durable remissions and prolonged survival, particularly for those patients who achieve a hematologic CR.

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