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 transplantation (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 hematologic 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), respectively (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.
Conclusions: ASCT is an effective treatment option for eligible patients with AL amyloidosis. Achieving hematologic CR is essential for long-term survival.
Keywords: Hematologic response; Organ involvement; Overall survival; Systemic amyloidosis; Treatment-related mortality.
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