The Evolution of Hemophilia Therapeutics: An Illustrated Review

• Published in: Research and practice in thrombosis and haemostasis Vol. 8; no. 3; p. 102308
• Main Authors: Español, María G., Mistretta, Jessica N., Tarantino, Michael D., Roberts, Jonathan C.
• Format: Journal Article
• Language: English
• Published: United States Elsevier Inc 01-03-2024; Elsevier
• Subjects: bleeding disorder; gene therapy; Hemophilia; hemophilia treatment; Illustrated Review; treatment; treatment; hemophilia treatment; gene therapy; illustrated review; Hemophilia; bleeding disorder
• ISSN: 2475-0379; 2475-0379
• DOI: 10.1016/j.rpth.2023.102308

Hemophilia is a rare genetic bleeding disorder historically associated with high morbidity and mortality. Some individuals with hemophilia suffer associated chronic joint disease, chronic pain, and other physical and mental health challenges. In the last 50 years, a better understanding of the pathophysiology of the disease has resulted in extraordinary therapeutic advances leading to enhanced quality of life and increased life expectancy.
We present an illustrated review of the evolution of hemophilia treatment from the development of non-factor therapies to gene therapy.
•Hemophilia is a bleeding disorder associated with high morbidity and mortality.•Advances in hemophilia treatment have led to safer treatment options and a potential cure.•Access to treatment and novel agents remains limited in low to middle-income countries.