What is the primary treatment for Hemophilia (coagulation disorder)?

Primary Treatment for Hemophilia

The primary treatment for hemophilia is factor replacement therapy with either plasma-derived or recombinant factor concentrates to replace the deficient coagulation factor (factor VIII for hemophilia A or factor IX for hemophilia B). 1, 2

Treatment Approaches Based on Hemophilia Type

For Hemophilia A (Factor VIII Deficiency):

• Factor VIII concentrates are the cornerstone of treatment and can be administered as prophylactic or on-demand therapy 2
• Prophylactic therapy (regular scheduled infusions) is preferred over on-demand therapy to prevent spontaneous bleeding episodes and joint damage 1, 3
• Even low-dose prophylaxis (10 IU/kg 2-3 times per week) is effective in preventing joint bleeds and damage compared to episodic treatment 1
• Emicizumab, a non-replacement therapy, is now approved for prophylactic treatment of patients with severe hemophilia A with and without inhibitors 2, 1

For Hemophilia B (Factor IX Deficiency):

• Factor IX concentrates are the primary treatment, administered either prophylactically or on-demand 1
• Prophylactic therapy significantly reduces bleeding episodes compared to on-demand treatment 4

Treatment for Patients with Inhibitors

Inhibitors are neutralizing antibodies that develop against replacement factors, making standard therapy ineffective:

• Bypassing agents are the first-line treatment for patients with inhibitors experiencing bleeding 1, 2
  • Recombinant activated factor VII (rFVIIa) - administered at 90 μg/kg every 2-3 hours until hemostasis 5
  • Activated prothrombin complex concentrates (aPCCs) 1
• Emicizumab is an effective alternative for prophylaxis in hemophilia A patients with inhibitors 1
• Immune tolerance induction therapy should be considered to eradicate inhibitors 6

Prophylaxis vs. On-Demand Treatment

• Prophylactic treatment significantly reduces annual bleeding rates compared to on-demand therapy (4.9 vs. 28.0 bleeds per year in hemophilia A) 4
• Primary prophylaxis (started after first joint bleed or before age two) is recognized as first-line treatment in children with severe hemophilia 3
• Secondary prophylaxis aims to prevent progression of arthropathy and improve quality of life 3

Dosing Considerations

• Dosing should be based on the severity of hemophilia, with higher and more frequent dosing for severe hemophilia 2
• Target plasma factor levels should be tailored to the patient's bleeding phenotype and activity level 7
• For severe bleeding episodes in hemophilia A without inhibitors, factor VIII levels should be raised to 80-100% of normal 2

Common Pitfalls and Considerations

• Inhibitor development is more common in severe hemophilia A (20-35%) than in severe hemophilia B (4-9%) 1
• Combination therapy with rFVIIa and aPCC should be avoided except in life-threatening bleeds due to increased thrombotic risk 2
• Regular monitoring is essential to detect inhibitor development early 2
• Despite advances in treatment, approximately two-thirds of persons with hemophilia worldwide, living in developing countries, lack access to safe factor concentrates 3

Emerging Therapies

• Non-replacement therapies like emicizumab provide alternative options for prophylaxis in hemophilia A 1
• Extended half-life factor products are available that require less frequent dosing 4