video: Jeffrey Glassberg, MD, tests the effects of inhaled steroids to treat patients with sickle cell disease who didn't have asthma. view more
Credit: Mount Sinai Health System
(New York - August 13, 2018) The Departments of Emergency Medicine and Hematology at the Icahn School of Medicine at Mount Sinai have been awarded a $4 million grant from the National Institutes of Health toward further study of inhaled corticosteroids to treat sickle cell disease (SCD) in individuals who do not have asthma. The IMPROVE 2 study is a one-year randomized, placebo-controlled trial involving 80 SCD patients.
According to the U.S. Centers for Disease Control and Prevention, SCD affects 100,000 Americans and is more prevalent in certain ethnic groups, such as African Americans and Hispanic Americans. The Healthcare Cost and Utilization Project, a database consortium sponsored by the federal government, reports that per-patient health care costs in the U.S. are higher for SCD than any other disease, with total expenditures exceeding $2.4 billion. The disease causes the production of abnormal hemoglobin, a protein inside red blood cells that attaches to oxygen in the lungs and carries it to the rest of the body. Healthy red blood cells are flexible, but in SCD, abnormal hemoglobin forms long rods and distorts the red cells into a sickle shape. The repeated damage to red cells causes the blood to become inflamed and sticky and to break down faster, causing pain attacks, infections, stroke, and early death. The only organ in the body that can reverse sickling is the lung, but it is highly inflamed in SCD, which is why researchers thought to use inhaled steroids as a treatment.
Over the last decade, researchers at Mount Sinai have demonstrated that pulmonary inflammation is present in mice and humans with SCD. Furthermore, those with symptoms of more inflammation in the lungs are four times more likely to die prematurely. In Mount Sinai's first study of inhaled steroids for SCD, a triple-blind randomized trial of 52 patients called IMPROVE 1, inhaled steroids that are FDA-approved for other conditions were shown to reduce systemic inflammation, hemolysis, and daily pain.
The research team will include experts in hematology, pulmonology, and immunology and will be led by Jeffrey Glassberg, MD, Associate Professor of Emergency Medicine and of Medicine (Hematology, and Medical Oncology) at the Icahn School of Medicine at Mount Sinai.
"In sickle cell disease, the interaction between the lung and red blood cells represents a potentially high-value therapeutic target," said Dr. Glassberg. "Hydroxyurea is the primary FDA-approved drug for treatment, but it is underutilized due to side effects, stigma, and fertility concerns. Other promising drugs are coming, but their costs, efficacy, and scalability are hard to predict at this point. Inhaled corticosteroids offer a creative new approach with the potential to dramatically improve patient outcomes."
Dr. Glassberg says patients with SCD will often end up in the emergency department because they experience excruciating pain. He says alternative treatments will improve patient care, reduce ED visits, and lower healthcare expenditures.
Dr. Glassberg is director of the Mount Sinai Comprehensive Program for Sickle Cell Disease, a specialized clinic that offers clinical and psychosocial services to patients in New York City, which has the largest population of SCD patients in the country. The team includes physicians, nurses, and social workers who specialize in the treatment of sickle cell disease.
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The Mount Sinai Health System is New York City's largest integrated delivery system encompassing seven hospital campuses, a leading medical school, and a vast network of ambulatory practices throughout the greater New York region. Mount Sinai's vision is to produce the safest care, the highest quality, the highest satisfaction, the best access and the best value of any health system in the nation. The System includes approximately 6,600 primary and specialty care physicians; 10 joint-venture ambulatory surgery centers; more than 140 ambulatory practices throughout the five boroughs of New York City, Westchester, Long Island, and Florida; and 31 affiliated community health centers. The Icahn School of Medicine is one of three medical schools that have earned distinction by multiple indicators: ranked in the top 20 by U.S. News & World Report's "Best Medical Schools", aligned with a U.S. News & World Report's "Honor Roll" Hospital, No. 13 in the nation for National Institutes of Health funding, and among the top 10 most innovative research institutions as ranked by the journal Nature in its Nature Innovation Index. This reflects a special level of excellence in education, clinical practice, and research. The Mount Sinai Hospital is ranked No. 18 on U.S. News & World Report's "Honor Roll" of top U.S. hospitals; it is one of the nation's top 20 hospitals in Cardiology/Heart Surgery, Diabetes/Endocrinology, Gastroenterology/GI Surgery, Geriatrics, Nephrology, and Neurology/Neurosurgery, and in the top 50 in four other specialties in the 2017-2018 "Best Hospitals" issue. Mount Sinai's Kravis Children's Hospital also is ranked nationally in five out of ten pediatric specialties by U.S. News & World Report. The New York Eye and Ear Infirmary of Mount Sinai is ranked 12th nationally for Ophthalmology and 50th for Ear, Nose, and Throat, while Mount Sinai Beth Israel, Mount Sinai St. Luke's and Mount Sinai West are ranked regionally.
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