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Meeting Coverage
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ASH: Hematology
— More than half of children in study had no treated bleeds at follow-up of more than 100 weeks
By Mike Bassett, Staff Writer, MedPage Today
December 10, 2023
SAN DIEGO — Administering emicizumab (Hemlibra) to previously untreated or minimally treated infants with severe hemophilia A without factor VIII inhibitors is effective and safe, according to results from research presented here.
The phase III HAVEN-7 study showed that at a median follow-up of 101.9 weeks, the annualized treated bleeding rate was 0.4 (95% CI 0.30-0.63) among 55 study participants, with 30 children (54.5%) having no treated bleeds, reported Steven Pipe, MD, of the University of Michigan in Ann Arbor.
The annualized bleeding rate for all bleeds was 2.0 (95% CI 1.49–2.66), with no treated spontaneous bleeds.
“This was over a median follow-up of over 100 weeks, and the median age was 29 months — so these are totally mobilized toddlers through this clinical cut-off date,” Pipe said during a press briefing at the annual meeting of the American Society of Hematology, “Emicizumab prophylaxis really demonstrated consistent efficacy across all the bleeding endpoints.”
For decades the approach to managing hemophilia has been the use of prophylactic factor VIII protein with factor VIII concentrates. Pipe pointed out this is a challenging process because it requires intravenous infusions frequently because of the short half-life of factor VIII.
Emicizumab is a humanized bispecific monoclonal antibody that bridges factor IX and Factor X to substitute for the function of deficient activated factor VIII, and is approved for the routine prophylaxis of persons with hemophilia A.
However, Pipe observed that infants were underrepresented in studies evaluating emicizumab and so HAVEN-7 was initiated in order to provide more evidence for the prophylactic treatment of this population.
Prophylaxis should be the standard of care for infants with hemophilia A, Pipe suggested, but many don’t start receiving prophylaxis until after their first year of life, mostly due to challenges and complications associated with factor VIII administration, such as venous access issues — including situations in which clinicians have had to rely on central venous access devices.
Emicizumab, on the other hand, can be administered subcutaneously from the time of hemophilia A diagnosis, Pipe explained, “This allows us to meet this treatment gap in the first year of life by enabling very early initiation of prophylaxis, and really may mitigate the risk of untreated or spontaneous traumatic bleeding, which can accrue damage, particularly into joints.”
“Importantly, it could mitigate the risk of intracranial hemorrhages,” Pipe added. “There is a substantial risk of intracranial hemorrhage throughout the first year of life for infants who are not yet on prophylactic therapy.”
He also noted that the study offered the opportunity to look at alterations of the natural history of inhibitor development to factor VIII-containing products, “because we would expect infants to have reduced exposure.”
The 55 participants in the study were all male.