Printer friendly PDF versions in English and professionally translated (older version) French, Italian, Portuguese, Punjabi, Russian and Simplified Chinese can be found HERE. Please note that German and Spanish (older) versions are also available HERE, but those translations have been generously completed by volunteers and the accuracy of the content has not been validated. The content is intended for informational purposes only, and is not meant to substitute consultation from a recognized health professional.
What is TTP?
Thrombotic Thrombocytopenic Purpura is a rare blood disorder that is considered a true medical emergency. TTP is diagnosed at a rate of 3-4in 1 million people per year. Potentially fatal complications can result from internal blood clotting, with damage to critical organs such as the brain and heart.
Due to a deficiency in the ADAMTS13 enzyme, blood becomes “sticky” and forms clots in blood vessels throughout the body. These clots are made up of platelets, one of the elements in blood. Vital blood flow to the body’s organs is restricted, placing the organs at risk for damage due to a lack of oxygen and nutrients from the blood. Moreover, since platelets are being used to form numerous unnecessary blood clots, their availability to perform their normal function, which is to seal injury sites to prevent excess bleeding, is compromised.
Types of TTP
Hereditary TTP (hTTP)
1% of TTP cases are due to an inherited deficiency or abnormality of the ADAMTS 13 enzyme.
Immune-mediated TTP (iTTP)
99% of TTP cases have no defined cause. In all cases, there is a decreased level of the ADAMTS 13 enzyme as a result of antibodies attacking the enzyme.
Symptoms
- fatigue
- fever
- bleeding (from nose, gums)
- diarrhea
- chest pain
- abdominal pain
- neurologic symptoms (confusion, headaches, visual changes)
- thrombocytopenia (bruising, purpura, petechiae)
Diagnosis
A medical history and a physical exam, in combination with a complete blood count (CBC), lactate dehydrogenase level (LDH) and blood smear are used to determine a diagnosis of TTP. More recently an ADAMTS 13 enzyme level test may be used to help confirm the diagnosis. Importantly, diagnosis and immediate treatment should not await the results of an ADAMTS 13 assay.
Treatment
Hereditary TTP (hTTP)
Monthly prophylactic plasma is sometimes administered to patients to replenish and maintain adequate levels of functioning ADAMTS 13, the enzyme which the patient is unable to produce themselves.
Immune-mediated TTP (iTTP)
Most patients receive steroids, e.g. prednisone, to slow the immune system and therefore the progression of this autoimmune disorder. The side effects of steroids can be challenging.
In all cases of iTTP, plasma exchange is the basic treatment of choice. Plasma exchange involves the use of automated machinery which permits the removal of the patient’s plasma and replacement with donor plasma during a 3 to 4 hour treatment. Plasma exchange both removes antibodies and replenishes normal plasma proteins. To treat iTTP, a series of daily or every other day plasma exchanges is used. Rituximab is increasingly used to achieve and maintain remission.
Other medications and/or removal of the spleen are used when patients fail to achieve remission from first line therapy.
Prognosis
Without treatment, 95% of patients succumb to the disease; however, with treatment 80 – 90% of iTTP patients achieve remission. Of these, about 30% will relapse. Early detection of such a flare of the disease is critical to minimize the risk of death or irreversible injury to vital organs.
Pregnancy may be a trigger for women with both hTTP and iTTP. Women considering pregnancy should discuss their individual case with their TTP specialist. Research is showing an ADAMTS 13 assay to be helpful in evaluating the risk of relapse during pregnancy, and in suggesting a risk mitigating prophylaxis treatment.
A growing percentage of patients are recognized with anxiety, depression and neurocognitive deficits after recovering from an episode of TTP.
Research
Links to current iTTP and hTTP research and journal articles can be found on our website here.
Answering TTP Foundation
A TTP diagnosis is scary and complex. Many patients have never heard of this acronym before, nor do they have any idea of its ramifications. Moreover, patients are told over and over that we just don’t know:
- why it happens
- what may trigger a relapse from remission
- why some patients relapse and others do not
- what the long term prognosis is
- how to ease treatment
- how to cure TTP
The purpose of Answering TTP Foundation is to help find answers to TTP by funding TTP research, connecting patients and supporters, and providing education and support.
Support
TTP affects a small and geographically dispersed population. Join the international Answering TTP community today for supportive newsletters, and to learn about peer, virtual, and face-to-face support initiatives. Each person touched by TTP brings further insight and strength to the community.
Printer Friendly Versions
Printer friendly version PDF versions in English and professionally translated French and Spanish are available in addition to previously translated older versions in Italian, Portuguese, Punjabi, Russian and Simplified Chinese can be found HERE.
Note: German translation (older version) are also available HERE, but they have been generously completed by volunteers and the accuracy of the content has not been validated.
The content is intended for informational purposes only, and is not meant to substitute consultation from a recognized health professional.
Special thanks to Lisa Thielecke and Jaime Sanchez for volunteering their time to translate the content.
Please contact us if you are interested in translating content into additional languages.