A substantial portion of the submitted materials did not meet the criteria for consideration described above. Analysis of these studies identifies important biases that were not addressed. Small sample sizes and discriminatory enrollment of patients introduces selection bias, which can lead to skewed results. Furthermore, none of these studies compare HDCT and AuSCT to either a control group or other treatment modalities. It is difficult to determine the effectiveness of a treatment unless it is compared to current standards of care.
Safety is another concern that was identified.
Moreover, there is little indication that AuSCT has become the standard of care in treating AL amyloidosis. The utilization of this procedure in the United States appears to be highly localized to specialized medical centers. It is apparent by the evidence that much of the research conducted on this topic is relatively preliminary, perhaps no more than five years old. Leading researchers in the field differ on the safety and efficacy of the procedure. We note that none of the three cited studies involved any patients over 63.