Week of 5–11 October 2026¶
What is the estimated annualized bleeding rate (ABR) for patients with severe hemophilia A with on-demand therapy vs prophylaxis?¶
Answer: In the landmark Valentino trial, patients were treated on demand for 6 months and then randomized to standard or PK-tailored prophylaxis, both targeting an FVIII trough ≥1%. The median ABR was 43.9 on demand compared with 1.0 on standard and 2.0 on PK-tailored prophylaxis (1.1 for any prophylaxis).