New approach makes mismatched stem cell transplants safer for blood cancer patients

Advances in blood stem cell transplants now make it possible for people with blood cancers to get safe and effective "mismatched" transplants that will potentially cure their disease, new UVA Cancer Center research reveals. The advances will allow far more people to receive the lifesaving treatment.

Patients who could not find a perfect match traditionally have not received transplants because of the potential for graft-versus-host disease. This occurs when the immune system recognizes the transplanted cells as foreign and attacks them. This can be serious and, in some cases, even deadly.

The new study, however, found that a treatment approach using the drug cyclophosphamide can prevent most graft-versus-host disease. The researchers followed 145 patients who received the cyclophosphamide treatment and found that eight out of 10, or about 80%, were alive after a year. This is similar to the outcomes seen in studies of patients who receive fully matched transplants.

This study is important because all patients, regardless of background, now have a stem cell or bone marrow transplant donor, a significant advance for our field, our patients and our community. At UVA Health, in the past year, all eligible patients for stem cell transplant were able to find a suitably matched donor." 

Karen Ballen, MD, UVA Health's Chief of Hematology/Oncology and Medical Director of Stem Cell Transplant

About the blood cancer study

UVA's Stem Cell Transplant Program was one of the main sites for the trial. Participants had leukemia, lymphoma or myelodysplastic syndrome and had been unable to find fully matched donors. They received a partially matched peripheral blood stem cell transplant and were given cyclophosphamide to prevent graft-versus-host disease.

Among the patients who received cyclophosphamide, only 10% developed moderate to severe or chronic graft-versus-host disease. This is about the same percentage as among patients who receive fully matched donations.

In a new scientific paper outlining their findings, the researchers describe the outcomes as "excellent" and call the addition of cyclophosphamide an "important advance." 

Findings published

The researchers have published their results in The Journal of Clinical Oncology. A full list of the researchers and their disclosures is included in the paper, which is free to read.

The study was supported by the National Institutes of Health's National Cancer Institute, grant U24CA076518; the National Heart, Lung and Blood Institute, grants U24CA076518 and UG1HL174426; the National Institute of Allergy and Infectious Diseases, grants U24CA076518 and U01AI184132; the Health Resources and Services Administration, grant 75R60222C00011; and the Office of Naval Research, grants N00014-24-1-2057 and N00014-25-1-2146.