Success criteria

ITI outcome defined on a cumulative basis, according to the achievement of three stringent efficacy criteria:

• Criterion I: Inhibitor titer < 0.6 BU
• Criterion II: FVIII in vivo recovery ≥ 80%
• Criterion III: FVIII half-life ≥ 7 hours
    - Complete success (all 3 criteria above met)
    - Partial success (2 of 3 criteria above met)
    - Partial response (1 of 3 criteria above met)
    - Partial failure (inhibitor still present, but titre has decreased to < 5 BU)
    - Failure (persistent high titre inhibitor)