Safety watch: tracking rare blood disorder treatment in real patients

This study aims to monitor the long-term safety of Adzynma, a replacement enzyme therapy for people with congenital thrombotic thrombocytopenic purpura (cTTP), a rare inherited blood clotting disorder. Researchers will review medical records of about 50 patients who have already received Adzynma in normal clinical practice to check for allergic reactions, antibody development, and pregnancy outcomes. The goal is to better understand the real-world safety profile of this treatment over time.