Management

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The management of the patient with haemophilia is based various principles:

• correct the bleeding tendency
• treat pain
• prevent deformity
• assist normal intellectual and emotional development in children with haemophilia
• use of prophylaxis
  • primary goal of factor IX prophylaxis is to enhance the quality of life by reducing hemarthrosis episodes, preventing the progression into hemophilic arthropathy, and minimizing episodes of intracerebral and muscular bleeds (1)
  • choice of product should be individualized based on clinical factors and activity levels (2)
  • marstacimab is recommended, within its marketing authorisation, as an option for preventing bleeding episodes caused by severe (factor IX [9] activity less than 1%) haemophilia B (congenital factor 9 deficiency) in people 12 years and over who (3):
    • weigh at least 35 kg and
    • do not have factor 9 inhibitors (anti-factor antibodies)

Reference:

1. Alshaikhli A, Killeen RB, Rokkam VR. Hemophilia B. [Updated 2023 Oct 29]. In: StatPearls [Internet]. Treasure Island (FL): StatPearls Publishing; 2024 Jan-. Available from: https://www.ncbi.nlm.nih.gov/books/NBK560792/
2. Konkle BA, Nakaya Fletcher S. Hemophilia B. 2000 Oct 2 [Updated 2024 Jun 6]. In: Adam MP, Feldman J, Mirzaa GM, et al., editors. GeneReviews® [Internet]. Seattle (WA): University of Washington, Seattle; 1993-2024. Available from: https://www.ncbi.nlm.nih.gov/books/NBK1495/
3. NICE (June 2025). Marstacimab for treating severe haemophilia A or B in people 12 years and over without anti-factor antibodies