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 organ severe failure. TTP is a life threatening disease and urgent and adequate treatment is essential.
The disease can be either congenital (inherited), or acquired (autoimmune). Only a small percentage of individuals (~ 5 %) present with inherited TTP while the majority of cases (~95%) are of the autoimmune nature (acquired TTP).
What are the symptoms?
Easy mnemonic: FASTRN.
- F– Fever;
- A– Anemia (Microangiopathic hemolytic anemia; low levels of red blood cells due to their premature destruction)
- S– Schistocytes (ruptured red blood cells) observed in blood smear
- T– Thrombocytopenia (severe; low levels of blood platelets)
- R– Renal failure
- N– 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 (acquired TTP) are not entirely understood. It has been shown that in most patients with acquired TTP 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 to keep the immune system under control.
Relapses occur but it’s hard to predict if or when they will occur.
What are the needs?
There is a need for:
- Developing novel diagnositic methods that are fast and easy to use;
- Optimizing existing treatment regimens to each patient’s ‘profile’ (introduction of personalized medicine);
- Developing new and effective treatment options.
Learn More on TTP
Visit these links to know more about TTP.
You can also see the educational videos below.
Educational videos about TTP
This video belongs to the American Society of Hematology.
This clip was excerpted from the documentary film “Blood Detectives.” To learn more about the film, visit http://www.hematology.org/Patients/Blood-Detectives.aspx
These videos are shown for educational purposes only.
Funded by the Horizon 2020
Framework Programme of the