Venous Thromboembolism in Recipients of Ex-Vivo Gene Therapy for Adolescent, Young Adult Patients with Sickle Cell Disease

Key Points

• Venous thromboembolism generally occurred in young adults following gene therapy for sickle cell disease.
• The incidence was notably high, with a significant number developing symptoms within the initial months post-treatment.
• Observational analysis on adolescent and young adult patients revealed critical outcomes for safety assessments.
• This analysis highlights the need for careful monitoring of thromboembolic events in gene therapy protocols.