Donor stem cell transplantation may improve survival in elderly patients with MDS, research shows

A new clinical trial provides the most compelling evidence to date that a donor stem cell transplant can improve the survival rates of elderly patients at higher risk for myelodysplastic syndrome (MDS), researchers at the Dana-Farber Cancer Institute report on the virtual 62nd Annual Meeting of the American Society of Hematology (ASH).

Despite being the only current cure for MDS and widely used for younger patients, transplantation is generally not offered to older patients because it has not been proven to be beneficial for that population. The new study, conducted by the Blood and Marrow Transplant Clinical Trials Network, is likely to change that, according to study leaders. The study involved 384 patients at 34 medical centers in the US. The study found that transplantation of hematopoietic stem cells from compatible donors nearly doubled the survival rate of patients aged 50-75.

Transplantation has historically been underused in this patient group. In any case, based on our findings, all patients should be referred to a transplant center so that those who qualify and have a suitable donor can undergo a transplant and have a better survival. It is important to refer these patients early so that the transplant center can work on finding an optimal donor from the start. “

Corey Cutler, MD, MPH, FRCPC, Study Senior Author, Dana-Farber Cancer Institute

MDS includes a group of conditions in which blood-forming cells in the bone marrow become abnormal, resulting in the production of defective blood cells. In about one in three patients, MDS can develop into acute myeloid leukemia, a rapidly growing cancer of bone marrow cells.

Allogeneic hematopoietic stem cell transplantation replaces a patient’s abnormal blood-forming stem cells with healthy stem cells from a compatible donor. Because the procedure has not been proven to be helpful for elderly patients, it is not covered by Medicare for people over the age of 65 unless performed as part of an approved trial. Medicare approved the design of the trial and is expected to consider the findings when determining future payment policies.

Participants in the new trial were referred to transplant centers, who were looking for suitable stem cell donors. The 260 patients matched with a donor within 90 days were ordered to undergo a stem cell transplant; the remaining 124 patients received standard supportive care.

About three years after study entry, 47.9% of those scheduled for transplant were alive, compared with 26.6% of those who had not found a donor after 90 days. Survival without recurrence of leukemia was also higher in those who had a transplant (35.8%) than in those who did not (20.6%). The researchers saw no significant differences between subgroups and no differences in quality of life between the two study arms.

Cutler will present the findings on this study at the “What’s on the Horizon: Practice-Changing Clinical Trials” press conference on Friday, December 4 at 12:30 pm EST. Further details will be presented at session 732, Abstract 75, on Saturday, December 5 at 10:30 am EST.


Dana-Farber Cancer Institute

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