The history of thrombotic thrombocytopenic purpura research: a narrative review

Abstract

Background and objective: Thrombotic thrombocytopenic purpura (TTP) is a rare but debilitating thrombotic microangiopathy that results from severe deficiency of the enzyme ADAMTS13. The disorder was first described in the early 20^th century, but the pathophysiology of the disease has only been elucidated in the past three decades. In this narrative review, we will summarize the milestone moments in the history of TTP research and discovery.

Methods: We searched literature using PubMed from 1924 to 2023 using the following free text searches: "thrombotic thrombocytopenic purpura", "Moschcowitz disease", and "thrombotic microangiopathy". We found 6,917 peer-reviewed articles and sorted through these for relevant literature pertinent to the review. A total of 46 articles were included for review and the remainder were excluded.

Key content and findings: The history of TTP research was reviewed, with a sampling of major events in the evolution of the understanding of the pathophysiology and treatment of the disease discussed here. There remains much to be learned about the nature of the disease in order to develop more specific and less harmful treatments.

Conclusions: An overview of the major discoveries that have led to our current understanding of TTP reveals the results of collaboration of multiple groups of physicians and scientists through the past century, with additional breakthroughs likely to occur in the future because of that same collaborative spirit.

Keywords: Thrombotic thrombocytopenic purpura (TTP); therapeutic plasma exchange; thrombotic microangiopathy; von Willebrand factor (VWF).

Figure 1

Timeline of selected major events in the history of TTP research. TTP, thrombotic thrombocytopenic purpura; VWF, von Willebrand factor; IgG, immunoglobulin G; FDA, Food and Drug Administration; EMA, European Medicines Agency; ISTH, International Society on Thrombosis and Haemostasis.