Single Center Experience of Autologous Stem Cell Transplantation in Patients with Systemic Light Chain Amyloidosis in Korea

The outcomes of ASCT for AL amyloidosis in a clinical setting were reviewed. Total 100 patients in amyloidosis cohort in a single institute were analyzed. In hematologic response, objective response rate was 79.0%, and the hematologic complete remission was essential for long term-survival. This study is one of the largest studies that reviewed the outcomes of ASCT in AL amyloidosis in Asia. Background: Systemic light chains is the most common systemic amyloidosis. In patients with AL amyloidosis, the prognosis is influenced by the extent of organ damage, especially cardiac involvement. Autologous stem cell transplan-tation (ASCT) is a highly effective treatment for AL amyloidosis for selective patient Methods: One hundred patients treated with ASCT for AL amyloidosis were reviewed in the Samsung Medical Center amyloidosis cohort. The cardiac, renal, and hematologic response was analyzed, and survival results compared based on organ involvement and hemato-logic response. Results: The most common involved organ was kidney (n = 62) followed by heart (n = 50). The organ response rate was 44.0% and 37.1% in the patients with cardiac and renal involvement, respectively. In hematologic response, overall response rate (ORR) was 79.0%, including 48.0% complete response (CR). Median overall survival (OS) in patients with and without hematologic CR were not reached and 64.2 months (95% CI, 19.5 to 109.0), respec-tively (P < .001). The survival rate was not significantly different between patients with or without cardiac or renal involvement. Treatment-related mortality (TRM) in 30 days and 100 days was 2.0% and 3.0%, respectively. Conclu-sions: ASCT is an effective treatment option for eligible patients with AL amyloidosis. Achieving hematologic CR is essential for long-term survival.