The protein elafin may be a good indicator of graft-versus-host-disease (GVHD) in patients who underwent an allogeneic stem cell transplant. These findings were published recently in the journal Science Translational Medicine.

Stem cell transplantation is the preferred treatment plan for multiple myeloma patients under the age of 65. When patients cannot produce enough of their own healthy stem cells for an autologous transplant, they undergo an allogeneic transplant, in which they receive stem cells from a donor.

A common complication of allogeneic stem cell transplantation, graft-versus-host disease, occurs when the body’s immune system flares in response to the donated stem cells. It often begins as a skin rash and is the leading cause of treatment-related death among patients who receive allogeneic stem cell transplants.

Currently, doctors can distinguish skin GVHD from other common skin rashes only by performing a skin biopsy, a labor-intensive and time-consuming procedure. Because skin GVHD can be life-threatening, doctors begin high-dose treatment in all patients developing skin rashes while waiting for the results of the procedure.

This one-size-fits-all protocol is problematic because patients whose results come back negative for skin GVHD are unnecessarily exposed to high-dose treatment which is associated with potentially fatal side effects.

In this study, researchers aimed to identify biomarkers for skin GVHD that would help in diagnosing the disease so doctors can develop treatment plans accordingly. A biomarker is a protein in the blood or tissue that can be used as an indicator for a disease.

“It [a biomarker] will help determine which rashes are caused by GVHD and need treatment with steroids,” explained Dr. James Ferrara, the study’s lead investigator, in an email to the Beacon, “as opposed to rashes from other causes which require a different treatment.”

To find biomarkers, researchers compared the blood samples of ten patients with skin GVHD and ten patients without it. They looked for proteins that had at least doubled in quantity in the blood samples of patients with GVHD and found fourteen such proteins, including elafin.

Because elafin is one of the easier proteins to screen for, researchers selected it as their top candidate for a biomarker. They found that on average, patients with skin GVHD had three times as much elafin in their blood as patients without GVHD.

Researchers also found that compared to other biomarkers for skin GVHD, elafin proved to be the most accurate for the diagnosis of skin GVHD.

Researchers also determined that in patients diagnosed with skin GVHD, those who had a high elafin concentration were three times more likely to die from the disease than those with a low elafin concentration. This indicates that elafin may be a prognostic indicator for a patient’s overall survival of GVHD.

They recommended that physicians use elafin concentration testing to develop individualized therapy for patients with skin GVHD. “In the long run, we hope using elafin levels will lead to more appropriate treatment, and therefore, reduce mortality,” said Ferrara.

For more information, please see the journal Science Translational Medicine (abstract).