A recent clinical trial conducted by researchers at the UNMC Fred & Pamela Buffett Cancer Center tested a possible treatment option for patients with a severe, treatment-resistant form of chronic graft-versus-host disease (GVHD).
Graft-versus-host disease, or GVHD, is a late-onset complication that can follow a blood or bone marrow transplant. The disease, also known as “sclerotic GVHD,” leads to skin hardening and joint problems, significantly impacting patients’ quality of life.
The study, led by Vijaya Bhatt, MBBS, professor of internal medicine and section leader of the Malignant Hematology Program at UNMC, was published in the Journal of Clinical Oncology, an American Society of Clinical Oncology journal.
Data from the multicenter phase II clinical trial shows that nearly half of the patients with sclerotic GVHD experienced significant improvements with ruxolitinib, a drug that blocks signals to cells that cause GVHD.
Chronic GVHD occurs when transplanted donor cells attack a patient’s healthy tissues following a blood or bone marrow transplant, which are common treatments for blood cancers. This immune response can cause widespread inflammation and damage, particularly affecting the skin, joints and other organs.
“While steroids are the standard treatment, many patients do not respond well, highlighting the urgent need for alternative therapeutic options,” Dr. Bhatt said. “The sclerotic form of GVHD is particularly challenging to treat.”
Ruxolitinib blocks specific proteins called Janus kinases, which are involved in immune system overactivity and inflammation. By reducing harmful immune responses, the drug helps control GVHD symptoms.
Key findings from the trial, which included 47 patients who had not responded to steroids and at least one other treatment, include:
- 49% of patients showed improved skin or joint symptoms.
- 38% of patients reported a better quality of life.
- 24% of patients on steroids were able to reduce their doses by more than half.
The study found that these benefits often persisted beyond six months, with some patients continuing to see improvements at 12 months. Notably, patients who had received fewer prior treatments before starting ruxolitinib responded best, suggesting that earlier drug use may enhance its effectiveness.
“Ruxolitinib was generally well-tolerated by study participants,” Dr. Bhatt said. “The 12-month survival rate was 97.8%, and none of the patients developed cancer relapse during the study period. These findings suggest that the drug improves symptoms and supports long-term disease management.”
As of 2021, ruxolitinib is FDA-approved for treating patients with chronic GVHD who have failed one or two lines of systemic therapy. The drug is therefore available for use in the clinic, and this study reinforces its role as a viable therapeutic option for sclerotic GVHD.
“This study additionally highlights the importance of researching different types of chronic GVHD separately to find the most effective treatments for each patient’s specific condition,” he said. “The results of this study offer hope for patients with limited treatment options to control their GVHD and regain quality of life.”
The research took place at five medical centers across the United States: UNMC, Fred Hutchinson Cancer Research Center, Moffitt Cancer Center, Ohio State University and the Cleveland Clinic.
Said Joann Sweasy, PhD, director of the Fred & Pamela Buffett Cancer Center, “UNMC’s leadership in this collaborative study underscores how Nebraska’s investment in medical research yields both scientific breakthroughs and economic growth, while positioning the Fred & Pamela Buffett Cancer as a hub for innovative cancer research.”
Very impactful study. Congratulations, Vijaya!
Congratulations!!