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Disease Profile
Thrombotic thrombocytopenic purpura, acquired
Prevalence estimates on Rare Medical Network websites are calculated based on data available from numerous sources, including US and European government statistics, the NIH, Orphanet, and published epidemiologic studies. Rare disease population data is recognized to be highly variable, and based on a wide variety of source data and methodologies, so the prevalence data on this site should be assumed to be estimated and cannot be considered to be absolutely correct.
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Age of onset
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ICD-10
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Inheritance
Autosomal dominant A pathogenic variant in only one gene copy in each cell is sufficient to cause an autosomal dominant disease.
Autosomal recessive Pathogenic variants in both copies of each gene of the chromosome are needed to cause an autosomal recessive disease and observe the mutant phenotype.
X-linked
dominant X-linked dominant inheritance, sometimes referred to as X-linked dominance, is a mode of genetic inheritance by which a dominant gene is carried on the X chromosome.
dominant X-linked dominant inheritance, sometimes referred to as X-linked dominance, is a mode of genetic inheritance by which a dominant gene is carried on the X chromosome.
X-linked
recessive Pathogenic variants in both copies of a gene on the X chromosome cause an X-linked recessive disorder.
recessive Pathogenic variants in both copies of a gene on the X chromosome cause an X-linked recessive disorder.
Mitochondrial or multigenic Mitochondrial genetic disorders can be caused by changes (mutations) in either the mitochondrial DNA or nuclear DNA that lead to dysfunction of the mitochondria and inadequate production of energy.
Multigenic or multifactor Inheritance involving many factors, of which at least one is genetic but none is of overwhelming importance, as in the causation of a disease by multiple genetic and environmental factors.
Not applicable
Other names (AKA)
Purpura, thrombotic thrombocytopenic; TTP; Moschowitz syndrome;
Categories
Blood Diseases
Summary
Thrombotic thrombocytopenic purpura (TTP), acquired is a
Symptoms
- Purplish bruises on the skin or mucous membranes (such as in the mouth). These bruises, called purpura, are caused by bleeding under the skin
- Pinpoint-sized red or purple dots on the skin. These dots, called petechiae, often are found in groups and may look like a rash.Bleeding under the skin causes petechiae.
- Paleness or
jaundice (a yellowish color of the skin or whites of the eyes) - Fatigue (feeling very tired and weak)
- Fever
- A fast heart rate or shortness of breath.
- Nausea or vomiting
- Headache, speech changes, confusion, coma, stroke, or
seizure - A low amount of urine, or
protein or blood in the urine
Acquired TTP usually presents as severe low
Diagnosis
Treatment
In about 10% of cases, acquired TTP does not respond well to these treatments. Other medications including certain
FDA-Approved Treatments
The medication(s) listed below have been approved by the Food and Drug Administration (FDA) as orphan products for treatment of this condition. Learn more orphan products.
- Caplacizumab(Brand name: Cablivi) Manufactured by Ablynx NV
FDA-approved indication: February 2019, caplacizumab (Cablivi) was approved for the treatment of adult patients with acquired thrombotic thrombocytopenic purpura (aTTP), in combination with plasma exchange and immunosuppressive therapy.
National Library of Medicine Drug Information Portal
Organizations
Support and advocacy groups can help you connect with other patients and families, and they can provide valuable services. Many develop patient-centered information and are the driving force behind research for better treatments and possible cures. They can direct you to research, resources, and services. Many organizations also have experts who serve as medical advisors or provide lists of doctors/clinics. Visit the group’s website or contact them to learn about the services they offer. Inclusion on this list is not an endorsement by GARD.
Organizations Supporting this Disease
-
Answering T.T.P. (Thrombotic Thrombocytopenic Purpura) Foundation
22 Prince George Drive
Toronto, ON, M9A 1Y1
Toll-free: 1-888-506-5458
Telephone: 416 792 4656
E-mail: [email protected]
Website: https://www.answeringttp.org/ -
Platelet Disorder Support Association
8751 Brecksville Road
Suite 150
Cleveland, OH 44141
Toll-free: 87-PLATELET (1-877-528-3538)
Telephone: 440-746-9003
Fax: 844-270-1277
E-mail: [email protected]
Website: https://www.pdsa.org/
Learn more
These resources provide more information about this condition or associated symptoms. The in-depth resources contain medical and scientific language that may be hard to understand. You may want to review these resources with a medical professional.
Where to Start
- MedlinePlus was designed by the National Library of Medicine to help you research your health questions, and it provides more information about this topic.
- Genetics Home Reference (GHR) contains information on Thrombotic thrombocytopenic purpura, acquired. This website is maintained by the National Library of Medicine.
- The National Heart, Lung, and Blood Institute (NHLBI) has information on this topic. NHLBI is part of the National Institutes of Health and supports research, training, and education for the prevention and treatment of heart, lung, and blood diseases.
- The National Organization for Rare Disorders (NORD) has a report for patients and families about this condition. NORD is a patient advocacy organization for individuals with rare diseases and the organizations that serve them.
In-Depth Information
- Medscape Reference provides information on this topic. You may need to register to view the medical textbook, but registration is free.
- The Monarch Initiative brings together data about this condition from humans and other species to help physicians and biomedical researchers. Monarch’s tools are designed to make it easier to compare the signs and symptoms (phenotypes) of different diseases and discover common features. This initiative is a collaboration between several academic institutions across the world and is funded by the National Institutes of Health. Visit the website to explore the biology of this condition.
- PubMed is a searchable database of medical literature and lists journal articles that discuss Thrombotic thrombocytopenic purpura, acquired. Click on the link to view a sample search on this topic.
Selected Full-Text Journal Articles
- Gandhi K, Aronow WS, Desai H, Amin H, Sharma M, Lai HM, Singh P. Cardiovascular manifestations in patients with thrombotic thrombocytopenic purpura: a single-center experience. Clin Cardiol. 2010 Apr;33(4):213-6.
- Viswanathan S, Rovin BH, Shidham GB, Raman SV, Weinberg M, Patricia A, George JN, Wu HM, Cataland SR. Long-term, sub-clinical cardiac and renal complications in patients with multiple relapses of thrombotic thrombocytopenic purpura. Br J Haematol. 2010 May;149(4):623-5. Epub 2010 Feb 9.
References
- George JN and Cuker A. Acquired TTP: Clinical manifestations and diagnosis. UpToDate. November 25 2015; https://www.uptodate.com/contents/acquired-ttp-clinical-manifestations-and-diagnosis.
- Explore Thrombotic Thrombocytopenic Purpura. National Heart, Lung, and Blood Institute. February 1 2016; https://www.nhlbi.nih.gov/health/health-topics/topics/ttp/.
- Wun T. Thrombotic Thrombocytopenic Purpura. Medscape Reference. June 20 2016; https://emedicine.medscape.com/article/206598-overview.
- Tersteeg C, Verhenne S, Roose E, Schelpe AS, Deckmyn H, De Meyer SF, and Vanhoorelbeke K. ADAMTS13 and anti-ADAMTS13 autoantibodies in thrombotic thrombocytopenic purpura current perspectives and new treatment strategies. Expert Rev Hematol. 2016; 9(2):209-21. https://www.ncbi.nlm.nih.gov/pubmed/26581428.
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