[Hemophilia : a disease on the move]

Area of Science:

• Hematology
• Pharmacology
• Gene Therapy

Background:

• Hemophilia treatment historically relies on factor replacement therapy.
• Current treatments improve life expectancy but face challenges like inhibitor development and frequent injections.
• Quality of life remains a key consideration in hemophilia management.

Purpose of the Study:

• To review the evolution of hemophilia treatment over the past century.
• To highlight recent therapeutic advancements and their implications.
• To discuss the challenges and future directions in hemophilia care.

Main Methods:

• Review of historical and current hemophilia treatment modalities.
• Analysis of novel therapeutic agents, including long-acting factors and bispecific monoclonal antibodies.
• Exploration of emerging treatments such as gene therapy.
• Consideration of laboratory monitoring challenges.

Main Results:

• Factor replacement therapy has evolved, improving life expectancy and quality of life.
• New therapies, including long-acting concentrates and a subcutaneous bispecific monoclonal antibody, reduce injection frequency and manage inhibitors.
• Gene therapy presents a promising future treatment option.
• Advances necessitate adaptation of clinical laboratory monitoring techniques.

Conclusions:

• Hemophilia treatment has seen remarkable progress, enhancing patient outcomes and quality of life.
• Innovative therapies offer more convenient and effective management, including for patients with inhibitors.
• Future directions in hemophilia care, including gene therapy, hold significant promise despite ongoing challenges and costs.