Milwaukee, May 8, 2024 – The Centers for Medicare & Medicaid Services recently issued a decision to expand Medicare coverage for allogeneic hematopoietic stem cell transplants (HCT) for eligible Medicare patients with myelodysplastic syndromes (MDS), based on decades-long research led by investigators at the Medical College of Wisconsin (MCW) Cancer Center, in collaboration with investigators in the Blood and Marrow Transplant Clinical Trials Network (BMT CTN) and advocacy experts at the American Society of Hematology (ASH), American Society for Transplantation and Cellular Therapy (ASTCT) and the National Marrow Donor Program (NMDP).

MDS is a group of cancers that affect the body’s ability to produce healthy blood cells. Allogeneic HCT is the only curative therapy for patients with MDS.

“Until now, patients 65 and older with Medicare did not receive coverage for HCT, largely due to limited clinical research demonstrating that transplantations benefited patients in older age brackets (specifically Medicare beneficiaries) in the same way they benefitted younger patients,” said Douglas Rizzo, MD, MS, Senior Scientific Director of the Center for International Blood and Marrow Transplant Research (CIBMTR), a research affiliation of MCW and NMDP, and Cancer Service Line Director at Froedtert & the Medical College of Wisconsin. This challenge was further exacerbated since older adults were, consequently, overlooked as candidates for transplant because they did not have Medicare coverage for the procedure.

“We used our expertise in transplantation and the CIBMTR and BMT CTN research platforms to develop observational and interventional trials that would serve as a mechanism for gathering evidence to inform Medicare payment policy. For the first time in history, this made it possible for Medicare beneficiaries with MDS to enroll for treatment at transplant centers across the country and get coverage to support their care,” said Dr. Rizzo. “In the first three years after Medicare agreed to provide coverage conditional on participating in approved studies, the number of transplants more than quadrupled, and we started to see lifesaving outcomes that proved our belief that older patients would benefit from transplantation, just the same as younger patients.”

Since then, studies on MDS led by MCW, and in collaboration with investigators from other institutions participating in the CIBMTR and BMT CTN, have provided access to transplants for nearly 6,000 Americans with MDS over the age of 65. The substantial increase in patients treated with allogeneic HCT provided data on the safety and efficacy of these transplants and created access for a critical and disadvantaged group of patients. Furthermore, results presented at ASH meetings and published in the Journal of Clinical Oncology demonstrated patients over 65 who received the transplant had a significant survival advantage over those without a donor; and saw improved quality of life, as published in the American Journal of Hematology.

“We are proud to be part of this important work to bring greater HCT access to people on Medicare with MDS,” said Mohandas Narla, DSc, ASH President, and distinguished scientist at New York Blood Health Enterprises. “This would not have been possible without the dedicated research and collaboration from Dr. Rizzo, CIBMTR and our other partners. We will continue to work together to expand access and improve care and outcomes for people on Medicare.”

As a result of advocacy efforts that included MCW, NMDP, ASH and ASTCT, a final decision was recently made by Medicare in the form of a National Coverage Analysis Decision Memo to expand Medicare coverage for the affected population deeming the treatment “reasonable and necessary under section 1862(a)(1)(A) of the Social Security Act.”