Stem cell transplantation is a process that includes mobilization, harvesting, and transplant of stem cells and the administration of high dose chemotherapy and/or radiotherapy prior to the actual transplant. During hematopoietic stem cell transplantation (HSCT), stem cells are harvested from a related or unrelated donor (allogeneic) and subsequently administered by intravenous infusion to the patient.
Myelodysplastic Syndromes (MDS) are a heterogeneous group of hematologic disorders characterized by (1) cytopenia due to bone marrow failure and (2) the potential development of acute myeloid leukemia (AML). In MDS, groups of clonal stem cell disorders are observed, characterized by low blood cell counts, abnormal blood cell development, genetic markers, hypercellular bone marrow, cytopenias, mutations and dysplastic cells.
Currently, CMS has a National Coverage Determination (NCD 110.23) covering allogeneic HSCT for the treatment of leukemia, leukemia in remission, or aplastic anemia; for the treatment of severe combined immunodeficiency disease (SCID); for the treatment of Wiskott-Aldrich syndrome; and for the treatment of MDS only for beneficiaries participating in a Medicare-approved, prospective clinical study through Coverage with Evidence Development (CED).
CMS received a complete, formal request to reconsider the NCD, specifically coverage of allogeneic HSCT for beneficiaires with MDS. This NCA will align with the scope of the request which is for coverage of allogeneic HSCT for beneficiaries with MDS absent a CED requirement. CMS is not reconsidering any other section of the NCD.
CMS is soliciting public comment relevant to the request. We are particularly interested in comments that include scientific evidence and that address the breadth of the request. We are also interested in aspects of health disparities and health equity that should be considered in the review.
