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A groundbreaking study reveals promising alternative for thrombosis in children

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Learn About Congenital Heart Disease

In a novel clinical study led by distinguished pediatric cardiologist at Riley Children’s Health, Ronald M. Payne, MD, insights into the prevention of thrombosis in children with congenital or acquired heart disease reveal significant new potentials.

The study, known as SAXOPHONE (Safety of ApiXaban On Pediatric Heart Disease On the pre-ventioN of Embolism), explores the safety and efficacy of apixaban as another alternative to current standard treatments, offering hope in preventing blood clots among pediatric patients.

Children grappling with heart disease, whether congenital or acquired, often face thrombosis, where these blood clots contribute substantially to morbidity and mortality. Forming within the heart, blood vessels or coronary arteries, traditional medications to treat the condition involve vitamin K antagonists or low-molecular-weight heparin. While effective, these treatments necessitate constant monitoring and dietary restrictions and may affect bone health.

As a result, the SAXOPHONE study, in collaboration with national specialists and published through the Journal of the American College of Cardiology, explores using apixaban, a medication typically used in adults, as a viable alternative to standard therapies. Spanning a diverse pool of 192 children with congenital or acquired heart disease needing ongoing clot prevention, the trial aimed to assess the safety, dosing and effectiveness of apixaban. By the end, the study findings were ultimately striking–the alternative discovered safe levels when compared to the existing treatments, with a lower frequency of bleeding events overall.

“What sets apixaban apart is its convenience and potential benefits over current treatments,” Dr. Payne said. “With the study’s implications, this could signify an incredibly positive turning point for pediatric cardiology.”

Notably, the study also reported no clot-related incidents or fatalities, indicating promising patient outcomes. Unlike the standard treatments, apixaban requires no frequent monitoring and has no interaction with foods like the vitamin K antagonists do, serving as a more practical and efficient way to treating patients with thrombotic complications.

“It’s clear apixaban is proven safe, and the future of children with congenital heart disease needing thromboprophylaxis is heading in the right direction,” Dr. Payne added.

The SAXOPHONE study marks a significant leap forward in the field of pediatric cardiology, and apixaban is an ongoing transformative shift to preventing thrombosis in children with a heart disease. With this recent avenue, a new standard of care for cardiac patients is on its way.

About pediatric cardiology at Riley Children’s

The leading Riley Heart Center treats and manages the full continuum of cardiac care. With specialists in every clinical area, the cardiology program is active in research to address the unmet needs of patients. Additional highlights include:

  • National leader in patient outcomes with mortality rates below the national benchmark, according to the Society of Thoracic Surgeons (STS)
  • Only formalized comprehensive adult congenital heart disease program in Indiana, as accredited by the Adult Congenital Heart Association

Learn more about pediatric cardiology.

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Learn About Congenital Heart Disease
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Designated as Best Children's Hospitals by U.S. News & World Report, Ranked in 10 Specialties in 2023-24

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