Allogeneic Hematopoietic Stem Cell Transplantation for Myelodysplastic Syndromes

Published 09/03/2024

On March 6, 2024, the Centers for Medicare & Medicaid Services (CMS) issued a final decision under National Coverage Determination (NCD) 110.23, expanding Medicare coverage for allogeneic hematopoietic stem cell transplant using bone marrow, peripheral blood, or umbilical cord blood stem cell products for Medicare patients with MDS who meet specific criteria. 

Hematopoietic stem cell transplantation (HSCT) 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 the process stem cells are harvested from either the patient (autologous) or a 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 (decreased number of red blood cells, white blood cells and platelets) due to bone marrow failure and (2) the potential development of acute myeloid leukemia (AML). The bone marrow does not produce enough healthy, functioning blood cells. For treatment purposes, patients with MDS are often stratified into risk groups based on the potential development of AML, which varies widely across MDS subtypes.

Medicare patients with MDS must have the following 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)

For these patients, the evidence demonstrates that the treatment is reasonable and necessary under Section 1862(a)(1)(A) of the Social Security Act (the Act).

In addition, coverage of all other indications for stem cell transplantation not otherwise specified will be made by local Medicare Administrative Contractors (MACs) under Section 1862(a)(1)(A) of the Act. 

Refer to Publication (Pub) 100-03, NCD Manual, Chapter 1, Section 110.23 (PDF), for information regarding this NCD and Pub. 100-04, Claims Processing Manual (CPM), Chapter 3, Section 90.3.1 (PDF) for further billing instructions.

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