Today, Lynn Malec, MD, MSc, Medical Director at Versiti Comprehensive Center for Bleeding Disorders (CCBD); Associate Investigator at Versiti Blood Research Institute (VBRI); Senior Director at Versiti Medical Sciences Institute; and Associate Professor of Medicine and Pediatrics, Division of Hematology/Oncology, Medical College of Wisconsin was published in the prestigious New England Journal of Medicine as part of a clinical trial that studied the efficacy of a new treatment for children with severe hemophilia A.

Groundbreaking clinical trial

The international pediatric clinical trial of efanesoctocog alfa, XTEND-Kids, included children under age 12 with severe hemophilia A who previously received treatment with another FVIII infusion therapy. Researchers sought to determine whether efanesoctocog alfa was safe for bleed prevention in children under 12, if these patients developed antibodies to efanesoctocog alfa, and if the antibodies impacted the drug’s efficacy.

Versiti Comprehensive Center for Bleeding Disorders (CCBD), a federally designated hemophilia treatment center (HTC), had the highest number of patients enrolled in the study. Over the course of 52 weeks, 73 of the 74 clinical trial participants received once-weekly injections of efanesoctocog alfa, while one patient received two or three injections per week as part of an intensified consolidation treatment.

At the conclusion of the year-long trial, researchers found no evidence of neutralizing antibodies to FVIII, inhibitors or anti-drug antibodies, indicating that taking efanesoctocog alfa weekly is a highly effective treatment for protecting children with hemophilia A against bleeding. Furthermore, they found that FVIII levels remained in the normal to near-normal range for up to three days after dosing and noted high sustained FVIII activity throughout weekly dosing intervals.

To learn more, find the full article at https://versiti.org/versiti-blood-research-institute/articles/2024/versiti-participates-in-successful-clinical-trial-for-new-treatment-for-children-with-hemophilia-a