What is the role of autologous stem-cell transplantation (ASCT) in the treatment of amyloidosis?

Updated: Nov 12, 2018
  • Author: Robert O Holmes, Jr, DO; Chief Editor: Herbert S Diamond, MD  more...
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Answer

Autologous stem-cell transplantation (ASCT) has been used in patients with AL amyloidosis. Initially, high rates of early mortality raised concern about the safety of ASCT in this setting, but a study of Mayo Clinic patients found that treatment-related mortality declined from 14.5% in 1996-2002 to 2.4% in 2010-2016 (P < 0.001). Median overall survival for those periods increased from 75 months to not reached. Hematologic response rates rose from 69% to 84%. The authors conclude that ASCT is likely to remain an important first-line treatment, even when novel agents become available.


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