Actionable Insights Newsletter

Translating research to power whole health

Issue 9   |    August 2024

Apixaban emerges as efficient alternative to traditional anticoagulants in pediatric heart disease

Apixaban appears to be a safe and effective alternative to other treatments for preventing blood clots in children with congenital or acquired heart disease. It requires less monitoring, no dietary modifications, and eliminates the need for injections, providing a more convenient option for pediatric anticoagulation.

Enterprise Analytics Core domain(s): cardiovascular

Summary

Background
Thromboembolism (TE) is a condition where a blood clot forms in the body, and the risk of this is higher in children with congenital or acquired heart disease. The American Heart Association and the American College of Chest Physicians suggest the use of anticoagulation or 'blood thinning' medicine, like vitamin K antagonists (VKA) or low-molecular-weight heparin (LMWH), to prevent TE in children. However, these drugs pose challenges such as frequent blood draws and dietary interactions for VKAs, while LMWH also requires frequent blood draws, injections, and has potential side effects like decreased bone mineral density and heparin-induced thrombocytopenia. Apixaban, another anticoagulation drug known as a direct oral anticoagulant (DOAC), could potentially offer an alternative solution for children, but it has not been extensively studied in a pediatric setting.

Objective
The Safety of ApiXaban On Pediatric Heart disease On the prevention of Embolism (SAXOPHONE) study assessed the safety and efficacy of apixaban for the prevention of TE in children with congenital or acquired heart disease requiring chronic anticoagulation.

Methods
SAXOPHONE was a prospective, open-label, randomized study using VKA or LMWH as a comparator to assess the safety and efficacy of apixaban in children ages 28 days to 18 years. Children received the study drug or VKA/LMWH for 12 months or until anticoagulation was no longer needed, whichever was shorter. From January 2017 to October 2021, 192 children in 33 centers in 12 different countries were enrolled in the trial. 123 children on the apixaban arm and 61 on the VKA/LMWH completed the study. Information about how apixaban works and how it stays in the body was collected, as well as information about how well apixaban worked compared to VKA/LMWH to inform healthcare providers about apixaban dosing and management in children. Quality of life measurements (PedsQL and KIDCLOT questionnaires) and bone density images were collected from children on both apixaban and VKA/LMWH.

Results
Researchers found no TE-related deaths or TE events in either arm during the study. Bleeding events were similar between the two treatment arms, with the study participants on apixaban having a greater number of minor bleeds, mostly nosebleeds. Drug exposure across a wide range of weights and ages in study participants was comparable to target exposures in adults that have been shown to be safe and effective. These results show the efficacy and safety of using apixaban in children. QoL and bone density analyses were limited by small sample size, though children in the apixaban arm demonstrated lower anxiety.

Key takeaways