The Fontan procedure is performed in children with congenital heart disease who have a single functioning ventricle to redirect blood flow to the lungs to be reoxygenated. Children who undergo the Fontan procedure often face significant morbidity and mortality stemming from thrombotic events, especially during the critical 3- to 12-month period following the procedure. While it is common for physicians to prescribe aspirin, there are limited data regarding aspirin resistance or the optimal dose for thromboprophylaxis in children.
'For years, health care providers have had limited options to help reduce potentially fatal thrombotic events that often occur in young children following the Fontan procedure,' saidBrian
The UNIVERSE study was conducted to determine the comparative efficacy and safety of XARELTO versus aspirin and to provide clear evidence-based dosing recommendations in this pediatric population.
The study was conducted in two parts: Part A evaluated pharmacokinetic (PK) and pharmacodynamic (PD) properties of XARELTO, and Part B examined the safety and efficacy of XARELTO compared to aspirin when used for thromboprophylaxis for 12 months. The primary safety outcome was major bleeding events defined by the
Key findings:
A comparable and low prevalence of bleeding events was observed withXARELTO compared to aspirin. There was one major non-fatal bleeding event (nosebleed) with XARELTO, and compared to aspirin, a slightly lower prevalence of non-major clinically relevant bleeding (6 vs. 9 percent of participants) and trivial bleeding (33 vs. 35 percent of participants). The prevalence and pattern of adverse events were comparable between the two groups.
There were fewer thrombotic events withXARELTO, though the study was not powered for efficacy outcomes (post-hoc log-rank test p=0.095). In the XARELTO group, one patient had a pulmonary embolism (2 percent event rate). In the XARELTO Part A group, there was one venous thrombotic event for an overall rate of 3 percent in the XARELTO groups combined. In the aspirin group, one participant had ischemic stroke and two had venous thrombosis (9 percent overall event rate).
'The promise of the EXPLORER research program for XARELTO was to uncover new insights into how the medical community can help improve the care of patients who are faced with thrombotic risk, from young to old,' said
UNIVERSE study design
A randomized, multicenter, open-label, active controlled, two-part, Phase 3 study, UNIVERSE examined the use of a novel, oral liquid suspension XARELTO formulation in children 2-8 years old with single ventricle physiology who had the Fontan procedure within four months before enrollment. From
During Part A of the study, PK and PD blood samples were collected on days 1 and 4 to determine if the patient could continue receiving the study drug for the 12-month study period. During Part B, patients were randomized in a 2:1 ratio to receive either body weight-adjusted XARELTO, administered orally twice daily (dose regimen that matched the exposure range in adults treated with rivaroxaban 10 mg once daily), or aspirin, given once daily (approximately 5 mg/kg). The primary safety outcome was major bleeding events, and the primary efficacy outcome was any thrombotic event, including venous or arterial clots, or any thromboembolic event, including stroke or a pulmonary embolism. Patients had additional PK and PD samples collected at months 3 and 12. PK/PD results will be published at a later date.
About the Janssen Pharmaceutical Companies of
At Janssen, we're creating a future where disease is a thing of the past. We're the Pharmaceutical Companies of
Learn more at www.janssen.com. Follow us at www.twitter.com/JanssenUS and https://twitter.com/JanssenGlobal.
Cautions Concerning Forward-Looking Statements
This press release contains 'forward-looking statements' as defined in the Private Securities Litigation Reform Act of 1995 regarding rivaroxaban. The reader is cautioned not to rely on these forward-looking statements. These statements are based on current expectations of future events. If underlying assumptions prove inaccurate or known or unknown risks or uncertainties materialize, actual results could vary materially from the expectations and projections of
[1] The UNIVERSE study was not powered to demonstrate efficacy results.
[i]
Contact:
Media
Mobile: (973) 479-9845
E: JSilvent@its.jnj.com
Mobile: (917) 547-8078
E: LPasqua7@its.jnj.com
(C) 2021 Electronic News Publishing, source