Although stem cell transplantation is the only current therapy with the potential to cure myelodysplastic syndromes (MDS), it is rarely used as an initial treatment for older patients because it hasn’t been proven superior to other therapies.

New research by Dana-Farber Cancer Institute investigators stands to overturn that practice. In a clinical trial involving 384 older patients with advanced MDS, researchers found that those with a suitable matched donor for a reduced-intensity transplant had a substantially higher survival rate — and a lower chance of developing leukemia — than those who received drug therapy or supportive care. The quality of life of patients in the transplant group was no different from that of other trial participants.

The findings, published in the Journal of Clinical Oncology, may prompt a revision of Medicare and Medicaid reimbursement policies for transplant in this group of patients. The two federal health insurance plans traditionally haven’t covered the cost of transplant for older patients with MDS because the procedure hadn’t been shown to be better than the alternatives. With the publication of the new study, the Centers for Medicare and Medicaid Services (CMS) have been asked to launch a formal review of that policy.

“Our findings, coupled with those of other recent studies, suggest that treatment centers should not be limiting stem cell transplantation for MDS to younger patients,” says Corey Cutler, MD, MPH, senior author of the study and medical director of the Adult Stem Cell Transplantation Program at Dana-Farber. “We’ve shown that for fit older adults with high-risk MDS, transplant offers a superior outcome to other forms of treatment.”

Corey Cutler, MD

MDS is a collection of diseases that lead to low blood counts and have a high propensity to transform into acute leukemia. Patients have abnormal cells within the bone marrow and low blood-cell counts, which can lead to infections or bleeding. It usually appears in one’s late 60s or early 70s.

The main treatments for MDS are a transplant of hematopoietic (blood-making) stem cells, which can cure some patients, or drugs known as hypomethylating agents, which can make cancer cells behave more like normal cells but do not cure the disease. Traditionally, transplant has been thought to be too risky for older patients with MDS, particularly those burdened by other diseases as well, Cutler notes.

The new clinical trial, conducted by the Blood and Marrow Transplant Clinical Trials Network, was designed with officials at CMS to answer the question of whether stem cell transplantation is a better strategy than non-transplant treatments.

The 384 trial participants, who ranged in age from 50 to 75, had MDS with an intermediate or high risk of advancing to leukemia. The 260 patients with a closely matched stem cell donor were assigned to one arm of the trial; the 124 patients without a donor were assigned to the other. Patients in the first arm were expected to undergo a reduced-intensity transplant within six months at one of the 34 centers participating in the study. (Reduced-intensity transplants involve lower doses of chemotherapy and radiation therapy than standard transplants.)

Patients in the other arm received whatever therapy — drug regimens or supportive care — was deemed appropriate by the treatment center. (Some of the patients in this arm eventually did receive a transplant after less closely matched donors were identified for them. And not all the patients in the donor arm wound up receiving a transplant.)

Researchers compared how patients in each arm were doing three years after enrolling in the study.

“We found that almost 50% of those in the donor arm were alive, compared to about 26% in the non-donor arm,” Cutler states. “When we compared those in the donor arm who actually received a transplant to those who hadn’t, the difference was even greater.”

Researchers also found a similar-sized difference between the two arms in survival without the development of leukemia.

And contrary to the common perception that patients who undergo a transplant have a worse quality of life than those who don’t, the investigators found that the overall quality of life of those in the donor arm was just as good as that of those in the other arm.

The findings are in line with those of other recent studies, Cutler notes. One such trial, led by Dana-Farber’s Gregory Abel, MD, MPH, involved patients with a variety of conditions, including low-risk MDS. In that group, researchers found that transplant was just as beneficial as non-transplant therapy.

“Based on the results of our study and others, we’re hopeful that CMS will reverse its current policy of coverage and payment for older transplant patients with MDS,” Cutler remarks. A decision from the agency is expected within the next 12 months.

Watch Related Video >>

Key Word: Stem Cell Transplantation