TTP-Thrombotic Thrombocytopenic Purpura. What is this disease?

What is TTP?

Thrombotic thrombocytopenic purpura (TTP) is a rare but life-threatening thrombotic microangiopathic disorder.
In this disease the blood coagulation system is more active leading to increased formation of a special kind of blood clots mainly consisting of platelets. These clots block the normal blood flow through the organs of the human body, leading to severe organ failure. TTP is a life-threatening disease and urgent and adequate treatment is essential.
The disease can be either congenital (inherited), or acquired of which the immune-mediated form of TTP (iTTP) is most prevalent. Only a small percentage of individuals (~5%) present with inherited TTP while the majority of cases (~95%) are of the autoimmune nature (iTTP).

What are the signs and symptoms?

Symptoms include:

  • Anemia (Microangiopathic hemolytic anemia; low levels of red blood cells due to their premature destruction)
  • Thrombocytopenia (severe; low levels of blood platelets)
  • Schistocytes (ruptured red blood cells) observed in blood smear
  • Renal failure
  • Neurologic symptoms

A low activity of ADAMTS13 – a protein in the blood – has been correlated with TTP. Patients with hereditary TTP have mutations in their ADAMTS13 gene. The mechanisms by which patients acquire a deficiency in this protein’s activity (iTTP) are not entirely understood. It has been shown that in most patients with iTTP the immune system reacts against this protein, as patients produce autoantibodies. Why this happens is currently unknown.

Are there any treatments?

Available treatments, although they reduce mortality significantly, are very expensive, invasive and have a complex regimen. Plasma infusion is used in cases of congenital TTP. Plasma exchange therapy is the treatment of choice in patients with acquired TTP, often in combination with immune system modulators (corticosteroids, Rituximab) to keep the immune system under control.
In 2018 and 2019, Caplacizumab, was approved by the EMA and the FDA for use in treatment of iTTP. It is the latest major breakthrough for patient treatment. However, other treatments are currently under research, including recombinant ADAMTS13. Relapses occur but it’s hard to predict if or when they will occur.

What are the needs?

There is a need for:

  • Developing new and effective treatment options, especially to treat the immunologic component of iTTP
  • Developing novel diagnostic methods that are fast and easy to use

Learn More on TTP

Visit these links to know more about TTP.

You can also see the educational video below.