A study by researchers at the Icahn School of Medicine at Mount Sinai could 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.
Patients with fatal blood cancers like leukemia often require allogenic SCT for survival. Donor stem cells are transplanted to a recipient, but not without the risk of developing GVHD, a major cause of death after SCT. GVHD occurs when the transplanted donor cells (graft) attack the patient (host). However, the severity of symptoms severity does not accurately define how patients will respond to treatment and patients are often treated alike with high-dose steroids.
Researcher James L. M. Ferrara said, "High dose steroids are the only proven treatment for GVHD. 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 and his team of researchers developed and tested this new scoring system with three distinct risk scores to predict the patient's response to GVHD treatment. The algorithm worked perfectly, with the cumulative incidence of non-relapse mortality significantly increasing as the GVHD score increased, and so the response rate to primary GVHD treatment decreased.
Dr. Ferrara said, "This new scoring system will help identify patient who may not respond to standard treatments, and may require an experimental and more aggressive approach. And it will also help guide treatment for patients with lower-risk GVHD who may be over-treated. This will allow us to personalize treatment at the onset of the disease. Future algorithms will prove increasingly useful to develop precision medicine for all SCT patients."
The study is published in the journal 'Lancet Haematology'.