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What is TTP? A rare, life-threatening medical emergency

Early diagnosis and treatment of Thrombotic thrombocytopenic purpura (TTP) can help prevent the high risk of early deaths.

TTP is an extremely rare and potentially fatal thrombotic microangiopathy manifested by the presence of microvascular occlusions and subsequent thrombocytopenia, hemolytic anemia, and organ ischemia. It has a reported annual incidence of 4 to 5 cases per million in the US. TTP primarily affects young, healthy adults, with a median age of 40 years.1,2

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Purpura:
spots of variable size on the skin or in the mucous membranes, due to small hemorrhages; they can be dark red and as small as a pinhead (petechiae) or purplish and 2 to 3 cm in diameter (ecchymosis).

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Thrombocytopenic:
a lower-than-normal platelet count (below 30x10^9/L) due to the consumption of platelets during microthrombi formation, which causes occlusion of microvasculature.

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Thrombotic:
characterized by the formation of microthrombi (blood clots) within a blood vessel, which clog the vessel.

Types of TTP

Acquired TTP (aTTP)3,4

  • aTTP, also known as immune-mediated thrombotic thrombocytopenic purpura (iTTP)
  • The most common form of TTP; approximately 95% of TTP cases are aTTP
  • Caused by autoantibody inhibition of ADAMTS13 activity

Hereditary TTP4,5

  • Also known as congenital TTP, inherited TTP, familial TTP, or Upshaw–Schulman syndrome
  • Very rare form of TTP; mainly detected in children
  • Caused by mutations in the ADAMTS13 gene

aTTP can be divided according to whether there is a confirmed triggering cause of not. However, all causes require immediate treatment.6

Primary aTTP6

  • Primary aTTP refers to acquired autoimmune TTP for which there is no obvious underlying/precipitating cause/disease
  • Primary aTTP accounts for the majority of cases of TTP

Secondary aTTP6

  • Secondary aTTP refers to acquired autoimmune TTP for which a defined underlying disorder or trigger can be identified, including connective tissue disease (eg, systemic lupus erythematosus), HIV infection, cytomegalovirus infection, and/or a specific precipitating factor (eg, pregnancy or drugs)
  • Treatment of the underlying disorder and/or removal of the underlying precipitant may be required, as well as standard TTP therapy

Without treatment, the fatal outcome of TTP is rapid, with a mortality rate of 90%7-9

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Signs and symptoms of aTTP include7-10

skin

Skin

Petechiae, purpura, bruising

Neurological

Neurological

Headache, confusion, seizures, coma

cardiac

Cardiac

EKG abnormalities

gastrointestinal

Gastrointestinal

Abdominal pain, diarrhea

kidney

Kidney

Proteinuria/hematuria

These symptoms, along with the rarity of TTP, may cause confusion with other thrombotic microangiopathies (TMA), making the diagnosis of TTP challenging.1,11,12

Références

  1. Terrell DR, et al. The incidence of thrombotic thrombocytopenic purpura-hemolytic uremic syndrome: all patients, idiopathic patients, and patients with severe ADAMTS-13 deficiency. J Thromb Haemost. 2005 Jul;3(7):1432-6

  2. Miller DP, et al. Incidence of thrombotic thrombocytopenic purpura/hemolytic uremic syndrome. Epidemiology. 2004 Mar;15(2):208-15

  3. Scully M, et al. Caplacizumab Treatment for Acquired Thrombotic Thrombocytopenic Purpura. N Engl J Med. 2019 Jan 24;380(4):335-346

  4. Zheng XL, et al. ISTH guidelines for the diagnosis of thrombotic thrombocytopenic purpura. J Thromb Haemost. 2020 Oct;18(10):2486-2495

  5. National Institutes of Health. Congenital thrombotic thrombocytopenic purpura. Updated July 12, 2018. Accessed March 16, 2022. https://rarediseases.info.nih.gov/diseases/9430/congenital-thrombotic-thrombocytopenic-purpura

  6. Scully M, et al. Consensus on the standardization of terminology in thrombotic thrombocytopenic purpura and related thrombotic microangiopathies. J Thromb Haemost. 2017;15(2):312–22.

  7. Scully M, et al. Guidelines on the diagnosis and management of thrombotic thrombocytopenic purpura and other thrombotic microangiopathies. Br J Haematol. 2012 Aug;158(3):323-35

  8. Sayani FA, Abrams CS. How I treat refractory thrombotic thrombocytopenic purpura. Blood. 2015;125(25):3860–7.

  9. Kremer Hovinga JA, et al. Survival and relapse in patients with thrombotic thrombocytopenic purpura. Blood. 2010; 115(8):1500–11.

  10. Azoulay E, et al. Expert statement on the ICU management of patients with thrombotic thrombocytopenic purpura. Intensive Care Med. 2019;45(11):1518-1539.

  11. Gallan AJ, Chang A. A new paradigm for renal thrombotic microangiopathy. Semin Diagn Pathol. 2020;37(3):121-126.

  12. Tsai H-M. Pathophysiology of thrombotic thrombocytopenic purpura. Int J Hematol. 2010;91(1):1-19.

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