Mount Sinai Researchers Find New, Safer Treatment for Serious Side Effect of Bone Marrow Transplant

Newswise — New York, NY (December 14, 2022) — Researchers at The Tisch Cancer Institute at Mount Sinai have identified a treatment that is effective and safer than the standard of care for a serious, and sometimes fatal, side effect of bone marrow transplant in cancer patients. Results from a phase 2 clinical trial were presented at the annual meeting of the American Society of Hematology (ASH) in December.

The clinical trial in adolescents and adults found that a drug that inhibits the immune system in patients who have graft-versus-host disease (GvHD) is safer than steroids, the current standard treatment. GvHD is a side effect seen in patients who have received a bone marrow transplant from a donor to treat blood cancers. The study used a blood test developed at Mount Sinai to identify patients with GvHD most likely to benefit from the new treatment.

“Steroids are known to cause numerous complications in bone marrow transplant recipients who require treatment for GvHD such as serious infections, bone and muscle damage, poor sleep, and poor quality of life,” said Aaron Etra, MD, Assistant Professor of Medicine (Hematology and Medical Oncology) at The Tisch Cancer Institute, who presented the study at the ASH meeting.

“A steroid-free treatment for GvHD would be an important advance in improving transplant outcomes, but it’s not likely to be the best choice for all patients. Being able to use GvHD biomarkers to personalize treatment intensity was key to the success of this trial,” added John Levine, MD, Professor of Medicine (Hematology and Medical Oncology) at The Tisch Cancer Institute, and co-senior author for this research. Alexandra Capellini, a Mount Sinai medical student, was co-first author.

GvHD occurs when the cells from a donor attack the healthy organs of the recipient. It results in the release of certain tissue proteins into the bloodstream; these proteins can be used as biomarkers to quantify the severity of the tissue damage. Patients with low levels of these biomarkers tend to respond well to treatment in general, but before now there was no way to identify them. A research laboratory at Mount Sinai was able to quantify the GvHD biomarkers in blood samples obtained from patients within 30 hours, which made it feasible to study this treatment in patients who need to start treatment quickly.

Results from the trial were compared to a matched control group of patients who were treated with steroids. This research found that a short course of itacitinib, a JAK1 inhibitor that can tamp down the immune system, produced very high response rates that occurred faster than treatment with steroids. Responses to itacitinib were as durable as steroids and long-term outcomes were equally good.  

Both itacitinib and steroids were effective in 86 percent of patients. The one-year survival rate for itacitinib was 88 percent compared to 80 percent for steroids. Patients treated with itacitinib also had significantly fewer severe infections, likely a result of dramatically less exposure to systemic steroids.

“This was the first time anyone was able to use real-time GvHD biomarkers to identify low-risk patients whose treatment could be de-escalated,” said James L Ferrara, MD, Professor of Medicine (Hematology and Medical Oncology) at The Tisch Cancer Institute, and co-senior author for this research. “Mount Sinai developed this biomarker approach, which allowed us to present this new and meaningful finding for patients and their quality of life.”

Incyte funded this trial. Trial sites included MD Anderson Cancer Center, City of Hope, Ohio State University, Mass General, Mayo Clinic, Vanderbilt University, Emory University, Fred Hutchinson Cancer Research Center, University of Pennsylvania, Columbia University, University of Hamburg, University of Regensburg, and University of Erlangen.

About the Mount Sinai Health System

Mount Sinai Health System is one of the largest academic medical systems in the New York metro area, with more than 43,000 employees working across eight hospitals, over 400 outpatient practices, nearly 300 labs, a school of nursing, and a leading school of medicine and graduate education. Mount Sinai advances health for all people, everywhere, by taking on the most complex health care challenges of our time — discovering and applying new scientific learning and knowledge; developing safer, more effective treatments; educating the next generation of medical leaders and innovators; and supporting local communities by delivering high-quality care to all who need it.

Through the integration of its hospitals, labs, and schools, Mount Sinai offers comprehensive health care solutions from birth through geriatrics, leveraging innovative approaches such as artificial intelligence and informatics while keeping patients’ medical and emotional needs at the center of all treatment. The Health System includes approximately 7,300 primary and specialty care physicians; 13 joint-venture outpatient surgery centers throughout the five boroughs of New York City, Westchester, Long Island, and Florida; and more than 30 affiliated community health centers. We are consistently ranked by U.S. News & World Report's Best Hospitals, receiving high "Honor Roll" status, and are highly ranked: No. 1 in Geriatrics and top 20 in Cardiology/Heart Surgery, Diabetes/Endocrinology, Gastroenterology/GI Surgery, Neurology/Neurosurgery, Orthopedics, Pulmonology/Lung Surgery, Rehabilitation, and Urology. New York Eye and Ear Infirmary of Mount Sinai is ranked No. 12 in Ophthalmology. U.S. News & World Report’s “Best Children’s Hospitals” ranks Mount Sinai Kravis Children's Hospital among the country’s best in several pediatric specialties. The Icahn School of Medicine at Mount Sinai is one of three medical schools that have earned distinction by multiple indicators: It is consistently ranked in the top 20 by U.S. News & World Report's "Best Medical Schools," aligned with a U.S. News & World Report "Honor Roll" Hospital, and top 20 in the nation for National Institutes of Health funding and top 5 in the nation for numerous basic and clinical research areas. Newsweek’s “The World’s Best Smart Hospitals” ranks The Mount Sinai Hospital as No. 1 in New York and in the top five globally, and Mount Sinai Morningside in the top 20 globally.

For more information, visit https://www.mountsinai.org or find Mount Sinai on Facebook, Twitter and YouTube.