Professional Statements and Societal Positions Guidelines
National Comprehensive Cancer Network (NCCN)-2019
Current National Comprehensive Cancer Networkclinical guidelines for myelodysplastic syndromes (v.1.2020)make the following general recommendation about allo-HCT:
“For individuals who are transplant candidates, an HLA [human leukocyte antigen]-matched sibling, or HLA-matched unrelated donor can be considered. Results with HLA-matched unrelated donors have improved to levels comparable to those obtained with HLA-matched siblings. With the increasing use of cord blood or HLA-haploidentical related donors, HCT has become a viable option for many individuals. High-dose conditioning is typically used for younger individuals, whereas RIC [reduced-intensity conditioning] for HCT is generally the strategy in older individuals.”
American Society for Blood and Marrow Transplantation-2015
The American Society for Blood and Marrow Transplantation (2015) categorized the Indications for HCT into five areas as follows:
- Standard of care (S), where indication for HCT is well defined and supported by evidence:
- This category includes indications that are well defined and are generally supported by evidence in the form of high-quality clinical trials and/or observational studies (e.g., through CIBMTR or EBMT).
- Standard of care (C), clinical evidence available, where large clinical trials and observational studies are not available but HCT has been shown to be effective therapy:
- This category includes indications for which large clinical trials and observational studies are not available. However, HCT has been shown to be an effective therapy with acceptable risk of morbidity and mortality in sufficiently large single- or multi-center cohort studies. HCT can be considered as a treatment option for individual individuals after careful evaluation of risks and benefits. As more evidence becomes available, some indications may be reclassified as “Standard of Care”.
- Standard of care (R), rare indication, for rare diseases where HCT has demonstrated effectiveness but large clinical trials and observational studies are not feasible:
- Indications included in this category are rare diseases for which clinical trials and observational studies with sufficient number of individuals are not currently feasible because of their very low incidence. However, single- or multi-center or registry studies in relatively small cohorts of individuals have shown HCT to be effective treatment with acceptable risks of morbidity and mortality. For individuals with diseases in this category, HCT can be considered as a treatment option for individual individuals after careful evaluation of risks and benefits.
- Developmental (D), for diseases where pre-clinical and/or early phase clinical studies show HCT to be a promising treatment option:
- Developmental indications include diseases where pre-clinical and/or early phase clinical studies show HCT to be a promising treatment option. HCT is best pursued for these indications as part of a clinical trial. As more evidence becomes available, some indications may be reclassified as “Standard of Care, Clinical Evidence Available” or “Standard of Care”.
- Not generally recommended (N), where available evidence does not support the routine use of HCT:
- Transplantation is not currently recommended for these indications where evidence and clinical practice do not support the routine use of HCT. The effectiveness of non-transplant therapies for an earlier phase of a disease does not justify the risks of HCT. Alternatively, a meaningful benefit is not expected from the procedure in individuals with an advanced phase of a disease. However, this recommendation does not preclude investigation of HCT as a potential treatment and transplantation may be pursued for these indications within the context of a clinical trial.
The ASBMT will periodically review these guidelines and will update them as new evidence becomes available.
