Real-world use of emicizumab in Chinese children with hemophilia A: Retrospective data from a comprehensive care center.

ABSTRACT:

Importance

Emicizumab (EMI) is efficacious and safe for hemophilia A (HA) prophylaxis. However, its high cost poses a challenge in China.

Objective

To explore the possibility of using reduced-dosage EMI in Chinese HA children.

Methods

We conducted a retrospective study for HA children in our Comprehensive Care Center. Data were collected pre- and post-EMI treatment to evaluate bleeding rates. Laboratory analyses included factor VIII (FVIII)-like activity and EMI concentration measurements.

Results

Thirty-four HA children receiving EMI prophylaxis for a median (range) 24.5 (2.5-47.9) months by June 2023. Of these, 25 (73.5%) were under 3 years of age, 26 (76.5%) had severe hemophilia and 12 (35.3%) were minimally treated or previously untreated patients. Thirty-

SUBMITTER: Mao Q 

PROVIDER: S-EPMC11664537 | biostudies-literature | 2024 Dec

REPOSITORIES: biostudies-literature