Tampa General Hospital First in Florida, One of the First in the Nation to Administer New Gene Therapy for Patients with Hemophilia B

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Jan 19, 2024, 13:38 ET

The academic health system is also the third hospital in the nation to treat a patient with Hemgenix, an FDA-approved gene therapy infusion for hemophilia.

TAMPA, Fla., Jan. 19, 2024 /PRNewswire/ -- Tampa General Hospital (TGH) announced that the TGH Cancer Institute recently completed Florida's first successful infusion of Hemgenix, a gene therapy option for patients with severe hemophilia B — a rare bleeding disorder characterized by failure of the blood to clot properly. Under the direction of Dr. Nathan Visweshwar, an associate professor in the Division of Hematology and Medical Oncology in the USF Health Morsani College of Medicine, and a hematology and oncology specialist for Tampa General, the academic health system became the first in the state and the third hospital in the United States to administer this therapy with positive results.

"The infusion of this therapy is a hallmark of the TGH Cancer Institute's dedication to propelling innovation to improve outcomes and quality of life in a meaningful way for our hematology/oncology patients," said Dr. Eduardo M. Sotomayor, vice president and executive director of the TGH Cancer Institute. "We are proud that in offering this cutting-edge genetic therapy in our newly built, state-of-the-art infusion centers, we are uniquely positioned to address unmet needs for patients living with hemophilia B in our community and beyond."

Hemgenix (etranacogene dezaparvovec) uses a gene therapy approach called gene transfer that is administered through intravenous (IV) infusion. This approach introduces a working, or functional, gene into liver cells to "instruct them" to produce factor IX protein (deficient in patients with hemophilia), which helps form blood clots to prevent prolonged or excessive bleeding.

"This is an exciting opportunity to pioneer this treatment option, which will allow us to continue expanding access to a wide range of therapies for patients with hemophilia in our community and throughout the state," Visweshwar said.

Because the genes that cause hemophilia A and B are located on the sex-determining X chromosome, the disorder disproportionately affects males. According to the U.S. Centers for Disease Control and Prevention (CDC), as many as 33,000 males in the United States are living with hemophilia. Hemophilia B is a rarer form of the disorder, only prevalent in 3.7 cases per 100,000 U.S. males. The CDC reports that further studies are needed to assess the population of females living with hemophilia, both A and B.

"Our team's successful administration of this gene therapy is the perfect case study for the distinct benefits an academic and research health system can deliver to patients," said Dr. Abraham Schwarzberg, executive vice president, chief of Oncology, president of the Tampa General Provider Network (TGPN) and co-vice president of clinical and translational research. "The introduction of a new therapeutic option for this rare disease is a testament to our shared commitment to apply the latest research and clinical best practices to safely and effectively treat even the most complex conditions."

This milestone is the latest achievement for the TGH Cancer Institute. U.S. News & World Report's Best Hospitals list for 2023-2024 ranks the TGH Cancer Institute in the top 10% of hospitals in the nation for cancer care. Additionally, Newsweek named the Institute one of America's Best Cancer Hospitals and one of the top 10 cancer facilities in Florida, and the academic health system was also recognized on a 2023 list of 100 Hospitals and Health Systems with Great Oncology Programs by Becker's Hospital Review.

ABOUT TAMPA GENERAL HOSPITAL
Tampa General Hospital, a 1,040-bed, not-for-profit, academic health system, is one of the largest hospitals in America and delivers world-class care as the region's only center for Level l trauma and comprehensive burn care. Tampa General Hospital is the highest-ranked hospital in the market in U.S. News and World Report's 2023-24 Best Hospitals, with six specialties ranking among the top 50 best hospital programs in the United States. Tampa General Hospital has been designated as a model of excellence by the 2022 Fortune/Merative 100 Top Hospitals list. The academic health system's commitment to growing and developing its team members is recognized by two prestigious Forbes magazine rankings – in the top 100 nationally in the 2023 America's Best Employers for Women and top 25 in Florida in the 2023 America's Best Employers by State. Tampa General is the safety net hospital for the region, caring for everyone regardless of their ability to pay, and in fiscal year 2021, provided a net community benefit worth more than $224.5 million in the form of health care for underinsured patients, community education, and financial support to community health organizations in Tampa Bay. It is one of the nation's busiest adult solid organ transplant centers and is the primary teaching hospital for the USF Health Morsani College of Medicine. With six medical helicopters, Tampa General Hospital transports critically injured or ill patients from 23 surrounding counties to receive the advanced care they need. Tampa General houses a nationally accredited comprehensive stroke center, and its 32-bed Neuroscience, Intensive Care Unit is the largest on the West Coast of Florida. It also is home to the Jennifer Leigh Muma 82-bed neonatal intensive care unit, and a nationally accredited rehabilitation center. Tampa General Hospital's footprint includes TGH North which is comprised of three hospitals and several outpatient locations in Citrus and Hernando counties, 17 Tampa General Medical Group Primary Care offices, TGH Family Care Center Kennedy, TGH Outpatient Center, TGH Virtual Health, and 21 TGH Imaging outpatient radiology centers throughout Hillsborough, Pasco, Pinellas and Palm Beach counties. Tampa Bay area residents also receive world-class care from the TGH Urgent Care powered by Fast Track network of clinics. To see a medical care professional live anytime, anywhere on a smartphone, tablet or computer, visit Virtual Health | Tampa General Hospital (tgh.org). As one of the largest hospitals in the country, Tampa General Hospital is the first in Florida to partner with GE Healthcare and open a clinical command center that provides real-time situational awareness to improve and better coordinate patient care at a lower cost. For more information, go to www.tgh.org.