Program in Evidence-based Care
Full Text Guideline
Evidence Supporting the Recommendations
The recommendations are supported by a systematic review with meta-analysis.
Implementation of the Guideline
An implementation strategy was not provided.
Quick Reference Guides/Physician Guides
Benefits/Harms of Implementing the Guideline Recommendations
Appropriate recommendation regarding stem cell transplantation (SCT) in patients with primary systemic amyloidosis
- A randomized controlled trial (RCT) found treatment with autologous stem cell transplantation (ASCT) to be associated with a significant increase in treatment-related mortality.
- The ASCT treatment-related morbidity included infection, neutropenia, mucositis, adverse gastrointestinal effects, central nervous system effects (including seizures), acute renal failure, and bacterial sepsis syndrome.
Rating Scheme for the Strength of the Recommendations
- The patient selection process and the ultimate decision to perform a stem cell transplantation (SCT) should take into account not only disease-related characteristics, but also comorbidities and patient preferences.
- Care has been taken in the preparation of the information contained in this report. Nonetheless, any person seeking to apply or consult the report is expected to use independent medical judgment in the context of individual clinical circumstances or seek out the supervision of a qualified clinician. Cancer Care Ontario makes no representation or guarantees of any kind whatsoever regarding the report content or use or application and disclaims any responsibility for its application or use in any way.
Searches of Electronic Databases
Literature Search Strategy
The MEDLINE (OVID) database (2006 through October [week two] 2010) was systematically searched for evidence on October 21, 2010 using the strategy that appears in Appendix A of the original guideline document. A total of 23 hits were obtained, and after excluding irrelevant papers according to a title and abstract review, three were ordered for full-text review. Of these three, only one met the inclusion criteria and was retained.
Study Selection Criteria
Articles were selected if they were the following:
- Systematic reviews (SRs) with or without meta-analysis or clinical practice guidelines (CPGs) if the evidence was obtained with an SR
- Fully published randomized controlled trials (RCTs) on patients with amyloidosis who received stem cell transplantation (SCT) that reported on survival and/or quality of life (QoL)
- Fully published non-randomized studies on patients with amyloidosis who received SCT and had an appropriate contemporaneous control group that reported on survival or QoL
- Reports published in English only
Only one report, a systematic review with meta-analysis, was included.
Expert Consensus (Committee)
Review of Published Meta-Analyses
Systematic Review with Evidence Tables
Synthesizing the Evidence
While no pooling was planned, it would be considered if data allow.
Assessment of Study Quality
The quality of the included evidence was assessed as follows. For systematic reviews that would be used as the sole evidence base for the recommendations, the Assessment of Multiple Systematic Reviews (AMSTAR) tool would be used to assess quality. For clinical practice guidelines (CPGs), the Appraisal of Guidelines for Research and Evaluation (AGREE) II Instrument would be used but only if adaptation of the recommendations was being considered. Any meta-analysis would be assessed for quality using criteria similar to that used for randomized controlled trials (RCTs), where appropriate. RCTs would be assessed for quality by examining the following seven criteria: the method of randomization, reporting of blinding, the power and sample size calculation, length of follow-up, reporting details of the statistical analysis, reporting on withdrawals to treatment and other losses to follow-up, and reporting on the sources of funding for the research. Comparative, but non-randomized, evidence would be assessed according to a full reporting of the patient selection criteria, the interventions each patient received, and of all relevant outcomes.
A formal cost analysis was not performed and published cost analyses were not reviewed.
Identifying Information and Availability
Kouroukis CT, Rumble RB. Stem cell transplantation in primary systemic amyloidosis. Toronto (ON): Cancer Care Ontario (CCO); 2012 Mar 29. Various p. (Recommendation report; no. SCT-2). [7 references]
Not applicable: The guideline was not adapted from another source.
The Program in Evidence-based Care (PEBC) is a Province of Ontario initiative sponsored by Cancer Care Ontario and the Ontario Ministry of Health and Long-Term Care.
The Program in Evidence-based Care (PEBC) is a provincial initiative of Cancer Care Ontario supported by the Ontario Ministry of Health and Long-Term Care through Cancer Care Ontario. All work produced by the PEBC is editorially independent from its funding source.
Hematology Disease Site Group
For a current list of past and present members, please see the Cancer Care Ontario Web site.
The authors of this recommendation report disclosed potential conflicts of interest relating to the topic of this special advice report and declared there were none.
This is the current release of the guideline.
The RECOMMENDATION REPORT, initially the full original Guideline, over time will expand to contain new information emerging from their reviewing and updating activities.
Please visit the Cancer Care Ontario Web site for details on any new evidence that has emerged and implications to the guidelines.
Electronic copies: Available in Portable Document Format (PDF) from the Cancer Care Ontario Web site.
The following are available:
- Stem cell transplantation in primary systemic amyloidosis. Summary. Toronto (ON): Cancer Care Ontario; 2012 Mar 29. 4 p. Electronic copies: Available in Portable Document Format (PDF) from the Cancer Care Ontario (CCO) Web site.
- Program in evidence-based care handbook. Toronto (ON): Cancer Care Ontario (CCO); 2012. 14 p. Available in Portable Document Format (PDF) from the CCO Web site.
This summary was completed by ECRI Institute on September 6, 2013.
This NGC summary is based on the original guideline, which is subject to the guideline developer's copyright restrictions. Please refer to the Copyright and Disclaimer Statements posted at the Program in Evidence-based Care section of the Cancer Care Ontario Web site.
Primary systemic (amyloid light-chain [AL]) amyloidosis
Assessment of Therapeutic Effectiveness
- To evaluate the role of stem cell transplantation (SCT) in the treatment of primary systemic (amyloid light-chain [AL]) amyloidosis
- To review the most current evidence comparing conventional chemotherapy (CT) with high-dose chemotherapy (HDCT) with autologous stem cell transplantation (ASCT)
- To make a series of clinical recommendations to inform clinicians, patients, and other stakeholders of the treatment options available
All adult patients with primary (amyloid light-chain [AL]) amyloidosis who are being considered for treatment that includes either bone marrow or stem cell transplantation (SCT)
- High-dose chemotherapy (HDCT)
- Autologous stem cell transplantation (ASCT)
- Allogeneic stem cell transplantation (SCT) (not recommended for patients with primary systemic amyloidosis)
- Overall survival
- Complete and partial hematological response
- Treatment-related morbidity and mortality
- High-dose chemotherapy (HDCT) and autologous stem cell transplantation (ASCT) is an option for selected patients with primary systemic amyloidosis, preferably within an investigative setting.
- Allogeneic stem cell transplantation (SCT) is not recommended for patients with primary systemic amyloidosis.
Institute of Medicine (IOM) National Healthcare Quality Report Categories
Living with Illness
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