Test predicts response to early treatment for GVHD related to stem cell transplants in leukemia patients

Jan. 2, 2015

A new test may reveal which patients will respond to treatment for graft versus host disease (GVHD), an often life-threatening complication of stem cell transplants (SCT) used to treat leukemia and other blood disorders, according to a study led by researchers at the Icahn School of Medicine at Mount Sinai and published recently in the journal Lancet Haematology.

Patients with fatal blood cancers such as leukemia often require allogenic stem cell SCT to survive. Donor stem cells are transplanted to a recipient, but not without the risk of developing GVHD, a major cause of death after SCT. The disease occurs when the transplanted donor cells (the graft) attack the patient (the host). Symptom severity, however, does not accurately define how patients will respond to treatment, and patients are often treated uniformly with high-dose steroids. Although SCT cures cancer in 50 percent of the patients, 25 percent die from relapsed cancer and there remaining go into remission but later succumb to effects of GVHD.

“High-dose steroids is the only proven treatment for GVHD,” says study co-author James L. M. Ferrara, MD, DSc. “Those with low-risk GVHD are often over-treated and face significant side effects from treatment. Patients with high risk GVHD are undertreated and the GVHD progresses, often with fatal consequences. Our goal is to provide the right treatment for each patient. We hope to identify those patients at higher risk and design an aggressive intervention while tailoring a less-aggressive approach for those with low risk.”

Dr. Ferrara, along with a multi-center team of researchers, developed and tested a scoring system using almost 500 patient blood samples with newly diagnosed GVHD in varying grades from two different centers. They used three validated biomarkers, TNFR1, ST2 and Reg3a, to create an algorithm that calculated the probability of non-relapse mortality (usually caused by GVHD) that provided three distinct risk scores to predict the patient’s response to GVHD treatment. The algorithm was validated in a set of 300 additional patients from twenty different SCT centers throughout the United States.

Learn more from the Icahn School of Medicine press release