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. 2014 Oct;46(8):2877-81.
doi: 10.1016/j.transproceed.2014.09.053.

High-dose melphalan and autologous hematopoietic stem cell transplantation in primary amyloidosis: single-center results

G Charlinski  1 , M Ziarkiewicz  1 , P Boguradzki  1 , E Wiater  1 , T Torosian  1 , J Dwilewicz-Trojaczek  1 , W Wiktor-Jedrzejczak  2
Affiliations

High-dose melphalan and autologous hematopoietic stem cell transplantation in primary amyloidosis: single-center results

G Charlinski et al. Transplant Proc. 2014 Oct.

Abstract

Background: Systemic immunoglobulin light-chain amyloidosis (AL) is a plasma cell dyscrasia resulting in multisystem organ failure and death. Autologous hematopoietic stem-cell transplantation (ASCT) has been widely used to treat patients with AL. However, treatment-related mortality remains high and reported series are subject to selection bias.

Methods: To define the role of patient selection in stem cell transplantation, we evaluated 24 consecutive AL patients transplanted at our center.

Results: Complete hematologic response was achieved in all 20 patients surviving >100 days posttransplantation. The 1-year overall survival (OS) rate after ASCT was 78.5%. The 5- and 10-year progression-free and OS rates were 57% and 47%, respectively. Treatment-related deaths owing to cardiovascular problems occurred in 16% of cases.

Conclusion: ASCT for AL amyloidosis can be safely performed in experienced transplantation centers, and increased risk is associated mainly with cardiovascular system involvement.

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