Medicare to cover stem cell transplantation for sickle cell disease

The Centers for Medicare & Medicaid Services recently ruled that they will pay most medical costs for clinical trials that use stem cell transplantation to treat sickle cell disease, multiple myeloma and myelofibrosis for people covered by Medicare. The expanded coverage will begin this summer.

The decision puts a new potential cure for sickle cell disease within reach of more adult patients at the University of Illinois Hospital & Health Sciences System through an ongoing clinical trial.

The new technique, pioneered by clinicians at the National Institutes of Health in Bethesda, Maryland, and offered only at the NIH and UI Health, eliminates the need for chemotherapy to prepare a patient to receive healthy stem cells from a donor.

Children have been cured of sickle cell disease using bone marrow stem cell transplants for many years. Their young bodies are able to handle the chemotherapy. In adults, such cures have not been possible, because the preparative chemotherapy is too dangerous.

In the new procedure, patients receive immunosuppressive drugs just before the transplant, along with a very low dose of total body irradiation – a treatment much safer than chemotherapy. Donor cells from a tissue-matched healthy sibling are then transfused into the patient. Stem cells from the donor soon produce new blood cells in the patient and eventually eliminate symptoms without the need for regular blood transfusions. In many cases, sickle cells can no longer be detected. Patients must continue to take immunosuppressant drugs for at least a year after the transplant.

“Like any other transplant procedure, the patient undergoes an extensive screening process to determine if they are a good candidate,” said Dr. Damiano Rondelli, the Michael Reese Endowed Professor in Hematology and director of the blood and marrow transplant program at UI Health. Patients must have a tissue-matched donor, usually a sibling, to provide the stem cells that will be used in the transplant. Determining a patient’s ability to pay for the transplant is also a necessary part of the screening.

“Prior to Medicare’s decision, adult patients insured by Medicare weren’t able to get the chemo-free transplant because of its cost – about $200,000 for the transplant and medications,” said Rondelli. Medicare coverage, he said, “means that many more that would likely have died relatively young from the disease now have hope for a full life.”

Last September, Rondelli and colleagues published a paper in the journal Biology of Blood & Marrow Transplantation validating the NIH’s sickle cell procedure in 12 patients treated and cured at UI Health.

Patients eligible for the new coverage must be covered under Medicare. People 65 and older and those getting disability benefits from Social Security or certain disability benefits from the Railroad Retirement Board for 24 months are eligible for Medicare.