Neurological symptoms as the sole presentation of relapsed thrombotic thrombocytopenic purpura without microangiopathic haemolytic anaemia

Htun, Kay T; Davis, Amanda

doi:10.1160/th14-04-0359

Cited by 4 publications

(2 citation statements)

References 6 publications

Supporting

Mentioning

Contrasting

Order By: Relevance

“…26 However, occasional patients with ADAMTS13 ,10% of normal (,10 IU/dL) present with life-threatening microvascular thrombosis, including stroke or seizures, long before they develop microangiopathic hemolytic anemia or thrombocytopenia. [27][28][29][30][31] This variability is consistent with the pathophysiology of TTP. Thrombocytopenia is a consequence of platelet sequestration in the microvasculature, which need not occur in a high-shear location.…”

Section: Asynchronous Development Of Ttp Signs and Symptomssupporting

confidence: 80%

Pathophysiology of thrombotic thrombocytopenic purpura

Sadler

2017

Blood

199

218

View full text Add to dashboard Cite

The discovery of a disintegrin-like and metalloproteinase with thrombospondin type 1 motif, member 13 (ADAMTS13) revolutionized our approach to thrombotic thrombocytopenic purpura (TTP). Inherited or acquired ADAMTS13 deficiency allows the unrestrained growth of microthrombi that are composed of von Willebrand factor and platelets, which account for the thrombocytopenia, hemolytic anemia, schistocytes, and tissue injury that characterize TTP. Most patients with acquired TTP respond to a combination of plasma exchange and rituximab, but some die or acquire irreversible neurological deficits before they can respond, and relapses can occur unpredictably. However, knowledge of the pathophysiology of TTP has inspired new ways to prevent early deaths by targeting autoantibody production, replenishing ADAMTS13, and blocking microvascular thrombosis despite persistent ADAMTS13 deficiency. In addition, monitoring ADAMTS13 has the potential to identify patients who are at risk of relapse in time for preventive therapy.

show abstract

Section: Asynchronous Development Of Ttp Signs and Symptomssupporting

confidence: 80%

Pathophysiology of thrombotic thrombocytopenic purpura

Sadler

2017

Blood

199

218

View full text Add to dashboard Cite

show abstract

“…The total volume of platelets in circulation of a normal adult is typically only 10–20 ml, making it easy to develop pronounced thrombocytopenia through VWF–platelet aggregates 76 . More rarely however, the development of TTP signs and symptoms can be asynchronous, as the sequestration of platelets may occur in sites with lower shear stress without causing haemolytic anaemia, and/or the thrombus may be localized to a critical site, causing a life‐threatening thromboembolic event before overt heamolytic anaemia or thrombocytopenia 77 . Lesions tend to occur in the heart, pancreas, kidney and the brain, without extensive necrosis, suggesting transient occlusion of the blood vessels in these sites 78 …”

Section: Ttp As We Know It Today – Where Are We?mentioning

confidence: 99%

TTP: From empiricism for an enigmatic disease to targeted molecular therapies

et al. 2022

View full text Add to dashboard Cite

The 100th anniversary of the first description of Thrombotic Thrombocytopenic Purpura (TTP) as a disease by Dr. Eli Moschcowitz approaches. For many decades, TTP remained mostly a mysterious fatal condition, where diagnosis was often postmortem. Initially a pentad of symptoms was identified, a pattern that later revealed to be fallible. Sporadic observations led to empiric interventions that allowed for the first impactful breakthrough in TTP treatment, almost 70 years after its first description: the introduction of plasma exchange and infusions as treatments. The main body of knowledge within the field was gathered in the latest three decades: patient registries were set and proved crucial for advancements; the general mechanisms of disease have been described; the diagnosis was refined; new treatments and biomarkers with improvements on prognosis and management were introduced.Further changes and improvements are expected in the upcoming decades. In this review, we provide a brief historic overview of TTP, as an illustrative example of the success of translational medicine enabling to rapidly shift from a management largely based on empiricism to targeted therapies and personalized medicine, for the benefit of patients. Current management options and present and future perspectives in this still evolving field are summarized.

show abstract