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National Coverage Analysis (NCA) Tracking Sheet for Allogeneic Hematopoietic Stem Cell Transplantation (HSCT) for Myelodysplastic Syndrome (CAG-00415N)

Myelodysplastic Syndrome (MDS) refers to a heterogeneous group of acquired blood disorders characterized by dysplastic growth of hematopoietic progenitors and a hypercellular bone marrow with peripheral cytopenia. These syndromes are varied with regard to clinical characteristics, cytologic and pathologic features, and cytogenetics. Most patients present with signs or symptoms of anemia accompanied by infection or bleeding, although some are asymptomatic. MDS becomes more common as people age; the overall MDS incidence is 3.3.per 100,000, but the incidence in patients over age 70 is between 15 and 50 per 100,000. The median age at presentation is 76 years. Medicare aged patients 65 and older represent 80 percent of the total population receiving an MDS diagnosis. One potential therapy for MDS is allogeneic HSCT from either a related or unrelated donor.

Stem cell transplantation is a process in which stem cells are harvested from either a patient's (autologous) or donor's (allogeneic) bone marrow or peripheral blood for intravenous infusion. Allogeneic stem cell transplantation is a procedure in which a portion of a healthy donor's stem cell or bone marrow is obtained and prepared for intravenous infusion. It may also be used to restore function in recipients having an inherited or acquired deficiency or defect. Hematopoietic stem cells are multipotent stem cells that give rise to all the blood cell types including myeloid and lymphoid lineages.  A hematopoietic stem cell is a cell isolated from blood or bone marrow that can renew itself, differentiate to a variety of specialized cells,  can mobilize out of the bone marrow into circulating blood, and can undergo programmed cell death , called apoptosis—a process by which cells that are unneeded or detrimental self destruct.

For purposes of this NCA, this treatment will be called allogeneic HSCT. This NCA will evaluate the available evidence to determine whether a national coverage determination is warranted.

The NCD (110.8.1) for Stem Cell Transplantation provides covered and non-covered clinical indications. Anything not in either of these two categories may be covered only at local contractor discretion. Allogeneic Hematopoietic Stem Cell Transplantation for Myelodysplastic Syndrome is neither a covered or non-covered indication under the NCD; it may be covered only under local contractor discretion.

Inpatient Hospital Services
Physicians' Services
Requestor Name(s)The National Marrow Donor Program, American Society for Blood and Marrow Transplantation, American Cancer Society , American Cancer Society Cancer Action Network, AABB, American Society of Hematology, American Society of Clinical Oncology, Aplastic Anemia and MDS International Foundation, Blood and Marrow Transplant Information Network, National Bone Marrow Transplant Link, The Bone Marrow Foundation, The Leukemia and Lymphoma Society
Requestor Letter(s)View Letter
Formal Request Accepted and Review Initiated11/10/2009
Expected NCA Completion Date08/04/2010
Public Comment Period11/10/2009 - 12/10/2009
Proposed Decision Memo Released05/06/2010
Proposed Decision Memo Public Comment Period: 05/06/2010 - 06/05/2010 
Decision Memo Released08/04/2010
Comments for this NCA:View Public Comments
Lead Analyst(s)
Lead Medical Officer(s)
  • Lori Paserchia, MD

November 10, 2009

CMS initiates this national coverage analysis for the use of allogeneic stem cell transplantation for patients with myelodysplastic syndrome. The public has 30 days to submit comments on this topic. CMS considers all public comments, and is particularly interested in any additional recent clinical studies and other scientific information related to the outcomes of this treatment when used on Medicare patients. If the evidence is determined to be inadequate for coverage, we are especially interested in what types of studies are needed.

May 6, 2010

CMS posts the proposed decision memorandum and invites public comment.

CMS is requesting public comment on this proposed decision memorandum. CMS considers all public comments and we are particularly interested in comments that include evidence we did not review or comments that assess how we evaluated the evidence included.

In general, CMS does not need individually identifiable health information to inform its proposed or final decisions. Any public comment received with individually identifiable health information will not be posted to the CMS website at this time. Examples of individual personal health information can be found at the bottom of the comment submission form.

Instructions on submitting public comments can be found at You can also submit a public comment by clicking on the highlighted word comment in the title bar at the top of this page. We strongly urge that all public comments be submitted through this website.

August 4, 2010

CMS posts the final decision memo.