Hemophilia A in the third millennium.

Area of Science:

• Hematology
• Genetics
• Pharmacology

Background:

• Hemophilia A is an X-linked hereditary bleeding disorder resulting from coagulation factor VIII (FVIII) deficiency.
• Clinical severity ranges from mild to severe based on FVIII levels, impacting bleeding patterns.
• Severe hemophilia A involves spontaneous or excessive bleeding, particularly in joints and muscles.

Purpose of the Study:

• To summarize current knowledge on the clinical features of hemophilia A.
• To review contemporary management strategies for hemophilia A.
• To highlight challenges and advancements in hemophilia A care.

Main Methods:

• Narrative review of existing literature on hemophilia A.
• Synthesis of information on diagnosis, treatment, and complications.
• Analysis of treatment outcomes and patient quality of life.

Main Results:

• Management relies on FVIII products (plasma-derived and recombinant) and desmopressin for mild cases.
• Prophylaxis with continuous FVIII substitution is recommended for severe hemophilia A to prevent bleeding and joint damage.
• Improved care has increased life expectancy to near-normal levels in high-income countries, but inhibitor development is a major challenge.

Conclusions:

• Hemophilia A management has advanced significantly, improving patient outcomes and life expectancy.
• The development of FVIII inhibitors is a critical complication, necessitating ongoing research and management strategies.
• Comprehensive care, including prophylaxis and management of inhibitors, is essential for optimizing the quality of life for hemophilia A patients.