Thrombotic thrombocytopenic purpura: early diagnosis and effective treatment in 2025

Abstract

In 2024, the centenary of thrombotic thrombocytopenic purpura (TTP) marks a significant milestone in the understanding and management of this rare and life-threatening condition. To commemorate this occasion, the European Society of Intensive Care Medicine launched an educational initiative aimed at consolidating and disseminating key knowledge on TTP, spanning its history, pathophysiology, diagnosis, and treatment. This paper summarizes the advances that have transformed TTP from a nearly universally fatal disease to one with a survival rate exceeding 95%. The journey from Eli Moschcowitz's first description of TTP in 1924 to today's sophisticated therapeutic options highlights the crucial contributions of clinicians, basic scientists, and industry collaborators. Central to TTP's pathophysiology is the deficiency of the plasma protease ADAMTS13, caused either by autoantibodies as in immune TTP (iTTP) or by genetic mutations of ADAMTS13 in congenital TTP (cTTP). The discovery of ADAMTS13 and its deficiency has led to breakthroughs in diagnosis and differential diagnoses, and the development of targeted treatments, including therapeutic plasma exchange, caplacizumab (an anti-VWF nanobody), and recombinant ADAMTS13. This review provides a comprehensive summary of TTP, with a focus on diagnostic challenges, therapeutic advancements, and future perspectives. It also serves as a tribute to the collaborative efforts that have shaped our understanding of this disease over the past century. By raising awareness and sharing best practices, this educational initiative aims to further improve outcomes for patients with TTP and foster continued progress in the field.

Keywords: ADAMTS13; Acute kidney injury; Hematology; Plasmapheresis; Stroke; Thrombocytopenia; Thrombosis.