Treatment options for previously untreated patients (PUPs) with
hemophilia A or B who will require prophylaxis. Studies of extended
half-life products conducted in PUPs and other pediatric patients
are included in Supplemental Table 1.
Product availability will vary between countries. Licensing differs
between the European Union and the United States. FVIII, factor
VIII; FIX, factor IX; rFVIIIFc, recombinant factor VIII Fc fusion
protein; rFIXFc, recombinant factor IX Fc fusion protein; rIX-FP,
recombinant factor IX albumin fusion protein.
arFVIIIFc,36,37 rFIXFc,38,39 and rIX-FP40,41 are approved for all age groups in the European
Union and the United States; in the United States, rurioctocog alfa pegol,
42
turoctocog alfa pegol,
43
and nonacog beta pegol
44
are also approved for children.
bIn the European Union, use of rurioctocog alfa pegol,
45
turoctocog alfa pegol,
46
damoctocog alfa pegol,
47
and nonacog beta pegol
48
is licensed only in patients ⩾12 years of age; in the United
States, damoctocog alfa pegol
49
is licensed only in patients ⩾12 years of age; in both the
European Union and the United States, damoctocog alfa
pegol47,49 is not licensed for PUPs.