Allogeneic Hematopoietic Stem Cell Transplant for MDS

The NCD for Allogeneic Hematopoietic Stem Cell Transplantation (allo HSCT) for the treatment of Myelodysplastic Syndromes (MDS), released by CMS in August 2010, concluded that, absent convincing evidence that allo HSCT improves patient health outcomes, additional research from clinical trials would be appropriate under the Coverage with Evidence Development aspect of CMS’ coverage authority. The NCD specifies that allo HSCT for the treatment of MDS is covered only when provided to Medicare beneficiaries enrolled in an approved clinical study

Allo HSCT is a procedure in which a portion of a healthy donor's stem cell or bone marrow is obtained and prepared for intravenous infusion. In accordance with the Stem Cell Therapeutic and Research Act of 2005 (US Public Law 109-129) a standard dataset is collected for all allogeneic transplant patients in the United States by the Center for International Blood and Marrow Transplant Research. The elements in this dataset, comprised of two mandatory forms plus one additional form, encompass the information we require for a study under CED.

A prospective clinical study seeking Medicare payment for treating a beneficiary with allogeneic HSCT for MDS pursuant to CED must meet one or more aspects of the following questions:

• Prospectively, compared to Medicare beneficiaries with MDS who do not receive HSCT, do Medicare beneficiaries with MDS who receive HSCT have improved outcomes as indicated by:
  • Relapse-free mortality,
  • progression free survival,
  • relapse, and
  • overall survival?
• Prospectively, in Medicare beneficiaries with MDS who receive HSCT, how do International Prognostic Scoring System (IPSS) score, patient age, cytopenias and comorbidities predict the following outcomes:
  • Relapse-free mortality,
  • progression free survival,
  • relapse, and
  • overall survival?
• Prospectively, in Medicare beneficiaries with MDS who receive HSCT, what treatment facility characteristics predict meaningful clinical improvement in the following outcomes:
  • Relapse-free mortality,
  • progression free survival,
  • relapse, and
  • overall survival?

Potentially, the results of trials may provide sufficient evidence of clinical utility that a future NCD on this topic will provide coverage for such testing for all Medicare beneficiaries.

Decision Memo

Approved Studies

Study Title: A Multi-Center Biologic Assignment Trial Comparing Allogeneic Hematopoietic Cell Transplant to Hypomethylating Therapy or Best Supportive Care in Patients w/Intermediate-2 & High Risk Myelodysplastic Syndrome (BMT CTN #1102)
Sponsor: Medical College of Wisconsin
ClinicalTrials.gov Number: NCT02016781
CMS Approval Date: 12/11/2013

Study Title: Assessment of Allogeneic Hematopoietic Stem Cell Transplantation in Medicare Beneficiaries with Myelodysplastic Syndrome and Related Disorders" - A Sub-study Protocol For A Research Database For Hematopoietic Stem Cell Transplantation, Other Cellular Therapies and Marrow Toxic Injuries
Sponsor: Center for International Blood and Marrow Transplant Research
ClincialTrials.gov Number: NCT01166009
CMS Approval Date: 12/13/2010