Evaluation of Long-term Outcomes after Autologous Stem Cell Transplantation in Chinese Patients with AL Amyloidosis

Abstract Treatment of AL amyloidosis is still a challenge, especially the long-term outcomes of patients with AL amyloidosis. Although autologous stem cell transplantation (ASCT) exhibited excellent efficacy, the long-term benefit needs to be evaluated. This study included a total of 240 patients with AL amyloidosis who underwent ASCT at our center and were followed up for more than 3 years. The median age for this cohort was 52 years, and 49% of them were male. The treatment-related mortality of this cohort was 1.7%. After a median follow-up of 66 months, the overall 1-, 3-, 5- and 10-year estimated overall survival (OS) rates were 94%, 90%, 79%, and 66%, respectively, and the median OS has not been reached. The 1-, 3-, 5- and 10-year progression-free survival (PFS) rates were 89%, 77%, 55%, and 33%, respectively, and the median PFS was 83 months. On multivariable analysis, the difference between involved and uninvolved free light chains (dFLC) > 50 mg/L, melphalan dose, hematologic CR/VGPR, and bortezomib induction therapy were independent predictors of survival. This study shows that ASCT has a very good long-term effect on AL amyloidosis and ASCT will remain an important option in eligible patients.