Experience of prophylaxis treatment in children with severe haemophilia

Area of Science:

• Paediatric Haematology
• Bleeding Disorders Management
• Prophylactic Therapy

Background:

• Severe haemophilia management traditionally focused on episodic treatment.
• Prophylaxis aims to prevent bleeding episodes and long-term joint damage.
• Early evaluation of prophylactic strategies is crucial for optimizing outcomes.

Purpose of the Study:

• To evaluate the practice and outcomes of prophylactic treatment in boys with severe haemophilia.
• To assess the effectiveness of prophylaxis in preventing bleeds and inhibitor development.
• To analyze the impact of prophylaxis on quality of life and patient management.

Main Methods:

• Retrospective analysis of 41 boys with severe haemophilia under 17 years.
• Data collection on age at prophylaxis initiation, follow-up duration, and joint bleeds.
• Assessment of inhibitor development, venous access methods, and clinical joint scores.

Main Results:

• Prophylaxis initiated at a median age of 3.7 years in 93% of patients.
• 76% started prophylaxis after initial bleeds, yet 70% achieved a clinical joint score of 0.
• No new inhibitors developed on prophylaxis; pre-existing low-titre inhibitors became undetectable.

Conclusions:

• Prophylactic treatment is effective in severe haemophilia, preventing bleeds and inhibitors.
• It enables increased physical activity and improves quality of life for patients and families.
• Home-based peripheral venous access reduces hospital visits, despite initial training investment.