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Prophylaxis with Factor VIII reduced inhibitor risks in hemophilia A
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Prophylactic use of Factor VIII appeared to decrease inhibitor risk in a cohort of children with severe hemophilia A, study results showed.
Researchers conducted the investigation to evaluate links between treatment intensity and inhibitor incidence in a cohort of consecutive, previously untreated patients. Treatment intensity ranged from high-dosed intensive Factor VIII treatment to prophylactic therapy.
Eligible participants had Factor VIII activity <0.01 IU/mL and were born between 2000 and 2010. Investigators followed the cohort during their first 75 Factor VIII exposure days.
Researchers observed a link between intensive Factor VIII treatment for hemorrhages or surgery at the start of treatment and increased inhibitor risk (adjusted HR=2.0; 95% CI, 1.3-3.0). High-dosed Factor VIII treatment yielded a higher inhibitor risk than the low-dosed regimen (HR=2.3; CI 1.0-4.8).
Researchers also observed a link between prophylaxis and decreased overall inhibitor incidence, but only after 20 days of exposure to Factor VIII. In the prophylaxis group, patients with low-risk F8 genotypes carried an HR of 0.61 (95% CI, 0.19-2.0), whereas those with high-risk F8 genotypes carried an HR of 0.85 (95% CI, 0.51-1.4).
“Our findings suggest that, in previously untreated patients with severe hemophilia A, high-dosed intensive [Factor] VIII treatment increases the inhibitor risk and prophylactic [Factor] VIII treatment decreases the inhibitor risk, especially in patients with low-risk F8 mutations,” the researchers wrote.
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