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Autologous stem cell transplantation (ASCT) is used for disease stabilization and improvement of performance status in patients with immunoglobulin light chain amyloidosis. This therapeutic strategy has a relatively high treatment-related mortality. Therefore the limited treatment efficiency (cure is not possible) versus the risk of severe toxicity must be considered in each individual patient. The treatment-related mortality (TRM) has been up to 24% (Jaccard et al., 2007) and even 70% in certain studies (Moreau et al., 1998), but mostly lower mortality is reported (Vesole et al., 2006). These differences are probably due to patient selection criteria because TRM seems, among the other factors, to be associated mainly with cardiac amyloidosis. However, two main questions need to be answered with regard to efficiency versus toxicity of ASCT in amyloidosis. Firstly, one would appraise ASCT to be an effective treatment also in patients with cardiac amyloidosis, and ASCT could be considered also for these patients if the risk of severe toxicity could be reduced. Secondly, it can be difficult to select patients without cardiac amyloidosis for ASCT due to some uncertainity in assessment of cardiac involvement. Determination of cardiac involvement is routinely performed by echocardiography despite the fact that the sensitivity of echocardiography is limited. Endomyocardial biopsy provides more reliable diagnosis but is associated with 1-2% complications (Cooper et al., 2007; Yilmaz et al., 2010). Thus, increased safety for patients with cardiac involvement may also reduce the risk of severe toxicity for patients without detectable cardiac involvement. Dimethyl sulfoxide (DMSO) is used for cryopreservation of the stem cell autografts and the amount of DMSO infused together with the transplant may probably contribute to the early TRM in patients with amyloidosis. Several reports have described fatal outcome of autograft infusion, especially in patients with cardiac amyloidosis (Saba et al., 1999; Zenhausern et al., 2000). By reducing the amount of infused DMSO, it may be possible to reduce the risk of ASCT-associated mortality and thereby make this treatment available for additional patients. In this chapter we will review various strategies that can be used to reduce the amount of DMSO infused together with autologous stem cell grafts.

Autologous Stem Cell Transplantation in the Treatment of Amyloidosis – Can Manipulation of the Autograft Reduce Treatment-Related Toxicity?