Once-weekly Altuviiio offers bleed protection for children with severe hemophilia A
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Once-weekly prophylaxis with a high-sustained factor VIII replacement therapy exhibited effective bleed protection for children with severe hemophilia A, according to the agent’s manufacturer.
Antihemophilic factor (recombinant), Fc-VWF-XTEN fusion protein (Altuviiio, Sanofi) is approved in the United States for routine prophylaxis and on-demand treatment to control bleeding episodes for adults and children with hemophilia A. It also is approved for perioperative management.
Clearance of administered factor concentrates in blood is greater among children than adults. Consequently, children often require two to four injections per week with standard or extended half-life factor VIII products, according to a Sanofi press release.
The nonrandomized, phase 3 XTEND-Kids study included 74 previously treated children aged younger than 12 years with severe hemophilia A. Children received once-weekly prophylaxis with Altuviiio dosed at 50 IU/kg for 52 weeks
Occurrence of inhibitor development served as the primary endpoint.
Findings presented at International Society on Thrombosis and Haemostasis Congress showed the study met its primary endpoint, with no factor VIII detected (0%; 95% CI, 0-4.9).
The study also met key secondary endpoints, including annualized bleeding rate (median, 0; interquartile range, 0-1.02; mean, 0.89; interquartile range, 0.56-1.42) and maintenance of factor VIII activity above prespecified levels.
Nearly two-thirds (64%) of patients had zero bleeding episodes, 82% had zero joint bleeds and 88% had zero spontaneous bleeds.
The agent exhibited a safety profile consistent with that observed in a prior trial. Researchers reported no serious allergic reactions, anaphylaxis, or embolic or thrombotic events, and no patients discontinued treatment due to adverse events.
Treatment-emergent adverse events that occurred among at least 10% of study participants included COVID-19 positivity, upper respiratory tract infection and pyrexia.
“The results from XTEND-Kids mark an important breakthrough as we strive for optimized bleed protection as the standard of care,” Lynn Malec, MD, associate professor of medicine and pediatrics at Medical College of Wisconsin, medical director of Comprehensive Center for Bleeding Disorders and associate investigator at The Versiti Blood Research Institute, said in the release.
“Achieving high-sustained factor activity with once weekly dosing means a freedom from the tradeoffs between treatment burden and efficacy we often see in treating severe hemophilia A,” Malec added. “The reliable and consistent bleed protection ALTUVIIIO provides offers confidence for children living with hemophilia and their families to manage hemophilia with less worry.”
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