What question did this study set out to answer?

This research aims to compile and analyze the clinical experience of congenital TTP cases over 25 years.

March 14, 2026Open Access

Emerging from the shadows of congenital TTP

Puntos clave

This research aims to compile and analyze the clinical experience of congenital TTP cases over 25 years.
Analyzed data from the French thrombotic microangiopathy registry
Documented demographics and treatment experiences of 88 congenital TTP cases
Reviewed clinical outcomes for patients transitioning to recombinant ADAMTS13.
Documented 88 cases of congenital TTP over 25 years
39 patients switched to recombinant ADAMTS13 with reported outcomes
Highlighted FDA and EMA approvals of recombinant ADAMTS13 in 2023 and 2024.

Resumen

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.

Me gusta

Guardar

Ver artículo completo