The 2012 recommendations have been ENDORSED. This means that the recommendations are still current and relevant for decision making. Please see Section 3: Document Assessment and Review for a summary of updated evidence published between 2010 and 2019, and for details on how this Recommendation Report was ENDORSED.

# CLINICAL QUESTION
What is the role of stem cell transplantation (SCT) in the treatment of primary systemic (AL, amyloid light-chain) amyloidosis?

# TARGET POPULATION
All adult patients with primary (AL) amyloidosis who are being considered for treatment that includes either bone marrow or SCT.

# RECOMMENDATIONS AND KEY EVIDENCE
High-dose chemotherapy (CT) and autologous SCT is an option for selected patients with primary systemic amyloidosis, preferably within an investigative setting.
A single meta-analysis (1) met the inclusion criteria for this review, and that meta-analysis found no significant difference between autologous stem cell transplantation (ASCT) and CT for AL patients in survival outcomes. An RCT included in that meta-analysis (Jaccard et al, 2007) found treatment with ASCT to be associated with a significant increase in treatment-related mortality (TRM).
This meta-analysis has some limitations that must be considered when making evidencebased recommendations. The quality of the included evidence was low and consisted of a small RCT and non-RCTs with likely patient selection bias. The single included RCT needed 340 patients to detect a 15% survival difference at =0.05, but only 100 were accrued. Secondly, AL patients typically also have significant co-morbidities precluding them from study enrolment.
In consideration of the lack of curative treatment options and the limitations of the evidence reviewed, the Expert Panel believes that offering ASCT may be a reasonable option for some patients, depending on performance status, co-morbidities, patient preferences, and ultimate treatment goals.
There is no evidence supporting the use of allogeneic SCT for patients with AL.

# QUALIFYING STATEMENT
The patient selection process and the ultimate decision to perform an SCT should take into account not only disease-related characteristics, but also comorbidities and patient preferences.

# Added to the 2019 Endorsement:
Careful patient selection based on degree of light chain amyloidosis involvement and organ function is an emerging concept in amyloidosis that should be considered to reduce transplant-related mortality.
Transplantation in amyloidosis is an evolving area. New emerging areas include consideration of transplantation in first relapse and the impact of novel proteasome inhibitors on outcomes. New evidence is expected within a time frame of 2 to 3 years.

# FUTURE RESEARCH
Newer agents are being investigated in the treatment of AL amyloidosis. At this time it is not known how they may impact the need for SCT.

# IMPLICATIONS FOR POLICY
The number of transplants provincially for systemic AL amyloidosis remains very low, and is unlikely to change in the foreseeable future. Copyright This report is copyrighted by Cancer Care Ontario; the report and the illustrations herein may not be reproduced without the express written permission of Cancer Care Ontario. Cancer Care Ontario reserves the right at any time, and at its sole discretion, to change or revoke this authorization.

# RELATED PROGRAM IN EVIDENCE-BASED CARE REPORTS