Stem cell rescue

A prospective randomized study of 100 patients with immunoglobulin light-chain amyloidosis compared melphalan plus high-dose dexamethasone with high-dose melphalan plus autologous stem cell rescue.[25] After a median follow-up of 3 years, median overall survival (OS) favored the nontransplant arm (56.9 months vs. 22.2 months; P = .04).[25][Level of evidence A1] The 24% transplant-related mortality in this series and others reflects the difficulties involved with high-dose chemotherapy in older patients with organ dysfunction.[25-30] Between 2007 and 2012, the International Blood and Marrow Transplant Research Program identified 800 patients with amyloidosis who underwent autologous stem cell transplant (SCT); the 5-year OS rate was 77% and the transplant-related mortality rate was 5%, suggesting better selection of patients for transplant.[31][Level of evidence C1] Similarly, in a retrospective review of 672 consecutive patients with amyloidosis who underwent autologous SCT over 20 years, the treatment-related mortality rate declined to 2.4% between 2010 and 2016, compared with rates of 8.6% between 2003 and 2009 and 14.5% between 1996 and 2002.[32][Level of evidence C2] A randomized trial confirming the benefit of autologous SCT is not anticipated.[3,33]

An anecdotal series described full-intensity and reduced-intensity allogeneic SCT.[34]