Emerging from the shadows of congenital TTP
Abstract
In this issue of Blood Advances, Joly et al, 1 from the French thrombotic microangiopathy registry, report on their congenital thrombotic thrombocytopenic purpura (cTTP) experience.They present data spanning over 25 years, including the demographics, treatment, and experience of complications in 88 cTTP cases.Recombinant ADAMTS13 (rADAMTS13) (Adzynma) received US Food and Drug Administration approval in 2023 and European Medicines Agency approval in 2024.This study includes the details of 39 patients with cTTP, who switched to the recombinant protein.
Fields of Study & Subjects
Complement system in diseases
Platelet Disorders and Treatments
Blood Coagulation and Thrombosis Mechanisms
Publication details
- Journal / source
- Blood Advances
- Volume
- 10
- Issue
- 5
- Pages
- 1828–1830
- Publication date
- 10 March 2026
- Language
- EN