Allogeneic Hematopoietic Stem Cell Transplantation for Myelodysplastic Syndrome
On March 6, the Centers for Medicare & Medicaid Services (CMS) released a final National Coverage Determination (NCD) for Allogeneic Hematopoietic Stem Cell Transplantation (HSCT) for Myelodysplastic Syndromes (MDS), which was effective upon its release. The decision memo finalized three important changes to the NCD. First, CMS will now cover the use of umbilical cord blood stem cell. Prior to this decision, only bone marrow and peripheral blood stem cell products were covered under the NCD. Second, the decision memo finalizes the use of additional risk designations and scoring systems. Finally, coverage with evidence will no longer be a requirement for HSCT.
Final National Coverage Determination
The NCD will amend the Medicare National Coverage Determination Manual at section 110.23 – Stem Cell Transplantation under Nationally Covered Indications for Allogeneic Hematopoietic Stem Cell Transplantation (HSCT) and will read as follows (note that italicized text is the new text):
a) Effective for services performed on or after August 1, 1978, for the treatment of leukemia, leukemia in remission, or aplastic anemia when it is reasonable and necessary,
b) Effective for services performed on or after June 3, 1985, for the treatment of severe combined immunodeficiency disease (SCID) and for the treatment of Wiskott-Aldrich syndrome, and
c) Effective for services performed on or after xx/xx/xx, allogeneic hematopoietic stem cell transplant using bone marrow, peripheral blood or umbilical cord blood stem cell products for Medicare patients with myelodysplastic syndromes who have prognostic risk scores of:
• ≥ 1.5 (Intermediate-2 or high) using the International Prognostic Scoring System (IPSS), or
• ≥ 4.5 (high or very high) using the International Prognostic Scoring System - Revised (IPSS-R), or
• ≥ 0.5 (high or very high) using the Molecular International Prognostic Scoring System (IPSS-M).
The NCD also indicates that local Medicare Administrative Contractors (MACs) will determine coverage for all other indications for stem cell transplantation that are not otherwise specified as covered or non-covered under the NCD.
Background:
In June of 2023, CMS created a national coverage analysis for HSCT for MDS. At that time, CMS opened a 30-day comment period to allow stakeholders to submit comments on the clinical studies and scientific information relevant to the analysis. The national coverage analysis was initiated after ASH, ASTCT, NMDP, CIBMTR, and BMT-CTN sent a request for reconsideration in October, 2021 asking for “full coverage of allogeneic HSCT for individuals with myelodysplastic syndromes and the removal of the coverage with evidence development (CED) requirement currently tied to coverage for HSCT for individuals with MDS”.
ASH, along with ASTCT, NMDP, CIBMTR, and BMT-CTN, sent a comment letter in response. This comment letter provided evidence in support of removing the CED requirement, provided a detailed summary of clinical guidelines, and requested that cord blood be included as a covered donor source.
After reviewing comments and conducting further analyses, the agency released its proposed decision memo on December 7, 2023. The decision memo proposed the following: “effective for services performed on or after xx/xx/xx, allogeneic HSCT using only bone marrow or peripheral blood stem cell products for Medicare patients with myelodysplastic syndromes (MDS) designated as high-risk or very high-risk with a score of ≥ 4.5 points according to criteria specified by the International Prognostic Scoring System-Revised (IPSS-R)”, and included a statement that “coverage of all other indications for stem cell transplantation not otherwise noted above as covered or non-covered will be made by local Medicare Administrative Contractors.”
ASH, in collaboration with ASTCT, NMDP, CIBMTR, and BMT-CTN, submitted comments on the proposed decision memo in January, 2024 specifically asking for coverage of hematopoietic stem cell sources, which would therefore include cord blood, urging CMS to not specify the risk level needed for coverage, and recommending that the coverage determination allow the use “current validated scoring system, as recognized by authoritative clinical bodies such as the World Health Organization or National Comprehensive Cancer Network (NCCN).”
Final Decision Summary
The final decision on the NCD for HSCT for MDS was released on March 6, 2024.Table 1 outlines the comments submitted by ASH and its partners, and CMS response to those comments.
Table 1: ASH Comments and CMS Response
| Comment | CMS Response |
| Supported removal of the coverage with evidence development requirement. | Finalized the removal of the coverage with evidence development requirement. |
| Urged inclusion of cord blood as a covered stem cell donor source. | Finalized the coverage of cord blood as a stem cell donor source. |
| Supported restriction of use of stem cells taken from placenta and amniotic fluid. | CMS agreed and will not include placenta and amniotic fluid as sources of covered stem cells for transplant. |
| Recommended that CMS does not require the use of IPSS-R for risk stratification, as this model is out of date or soon will be and is being replaced by IPSS-M which is the clinical standard. | CMS agreed, and believes it is appropriate to use the newest model and will allow coverage when IPSS-M is used for risk stratification. |
| Urged CMS to not require adherence to a specific risk scoring system and allow use of scoring systems recognized by international bodies. |
CMS did not agree and has instead created policy that uses three versions for three levels of risk classification: • ≥ 1.5 (Intermediate-2 or high) using the International Prognostic Scoring System (IPSS), or • ≥ 4.5 (high or very high) using the International Prognostic Scoring System - Revised (IPSS-R), or • ≥ 0.5 (high or very high) using the Molecular International Prognostic Scoring System (IPSS-M) |
| Recommended that patients with intermediate risk MDS be included in the indication for allogenic HSCT. | Intermediate risk will be a covered indication. |
| Encouraged CMS to cover allogenic HSCT for treatment of secondary MDS. | CMS did not include secondary MDS in the NCD. Coverage for HSCT for secondary MDS will be at the discretion of the MACs. |
| Requested that CMS correct the decision memo which misstated that the BMT CTN 1102 study was funded by industry. | CMS made the correction, and the final decision memo now reads correctly that the study in question was funded by NIH grants. |
Effective Date:
The NCD was effective upon its release on March 6, 2024. The agency will draft a forthcoming transmittal to accompany the NCD that will outline other information necessary for the implementation of the revised NCD. ASH staff will closely monitor the release of the information and inform members of the Society as soon as possible.