Below we have listed news about Congenital TTP, articles are listed by most recent to oldest. If you have found an article on the web please email us the link so we can post it to this page for others to find.
The Hereditary TPP Registry
The Hereditary TTP Registry is an observational study for patients suffering from hereditary TTP as well as for their family members. The goal is to learn as much as possible about the course of the disease, potential triggers of acute episodes, treatment requirements, etc.
We would like to give as many patients and physicians as possible the opportunity to participate in this study.
We are asking the patient or the legal representatives to sign the informed consent form. As soon as we get a copy/scan/fax of the documents we are allowed to enroll the patients and their family member and analyze the samples. We do cover shipment and ADAMTS13 testing including the molecular analysis. If the family members (parents, siblings…) are interested in joining the project, they are very welcome to get tested. After we confirmed the hereditary TTP the collection of information can start. We’d generate a login to our data base for the physicians and would then ask physicians and patients to fill out the questionnaire. Additionally every study site will receive a small reimbursement for every completed participant’s (patient and family members) baseline questionnaire. Every year after the inclusion we’d ask for a small follow up on their patient beside information about had TTP-Episodes.
The patient data are all anonymized by a 9-digit pseudonym.
Forecasting the future for patients with hereditary TTP
PDF of Article Here
NEW RESOURCE from our friends in Canada
Royal Liverpool leading the way to fight TTP
Click to see Video
Doctors at the Royal Liverpool University Hospital are leading the fight against a life-threatening disorder.
They are now a regional centre for TTP - a condition that causes clots to form in blood vessels throughout the body, damaging vital organs such as the brain,
heart and kidneys and without urgent treatment, the risk of dying is up to 90%.
The only treatment which can reduce the risk is plasma exchange - the replacement of a patient's circulating
blood with fresh plasma. But TTP is so rare that when people suffer from symptoms, including hallucinations,
bizarre behaviour, and jaundiced skin. Mel Barham has more:
Successful Treatment of Congenital TTP using Plasma-derived Factor VIII
Click here to download the PDF
A Registry for Patients with Hereditary Thrombotic
Click here to download the Word Doc.
Archemix’ ARC1779 Granted Orphan Designation for the Treatment of TTP, a Life-Threatening Blood Disorder
Archemix Corp., a biotechnology company focused on discovering, developing and commercializing aptamer therapeutics for rare hematological diseases, today announced that ARC1779 has received orphan drug designation from the U.S. Food and Drug Administration. ARC1779, a novel aptamer therapeutic, is a first-in-class anti-platelet agent for the treatment of a rare, life-threatening blood disorder known as thrombotic thrombocytopenic purpura, or TTP. There is no drug approved for the treatment of TTP and acute episodes of TTP are associated with a significant mortality rate, estimated to be as high as 20 percent. more
More Links Related to Archemix' ARC1779:
Baxter Adds Recombinant Protein to Specialty Therapeutics Portfolio
Baxter International Inc. (NYSE: BAX) today announced it has signed an agreement with Kaketsuken, the Chemo-Sero-Therapeutic Research Institute, based in Kumamoto Japan, for the worldwide rights to develop, manufacture and market the recombinant protein ADAMTS13. The development of recombinant ADAMTS13, a bio-engineered version of a naturally occurring enzyme that plays a critical role in blood coagulation...more
What Is Thrombotic Thrombocytopenic Purpura?
Thrombotic thrombocytopenic purpura (TTP) is a rare blood condition. It causes blood clots to form in small blood vessels throughout the body. These blood clots can cause serious problems if they block blood vessels and limit blood flow to the brain, kidneys, or heart...more
TTP and ADAMTS13: When Is Testing Appropriate?
The last 10 years witnessed the publication of many studies on the pathophysiology of thrombotic thrombocytopenic purpura (TTP), a life-threatening disease characterized by microangiopathic hemolytic anemia, thrombocytopenia and multiorgan failure. The most important finding was the identification of a novel metalloprotease, named ADAMTS13 (a disintegrin and metalloproteinase with thrombospondin type 1 motives), that is involved in the regulation of the size of von Willebrand factor (VWF), a major modulator of platelet adhesion and aggregation in the microcirculation...more
Congenital TTP treatment shows promise
UK researchers say that effective prophylaxis and treatment for children with congenital thrombocytopenic purpura (TTP) is possible using a US-sourced plasma-derived factor VIII concentrate called BPL 8Y.The team, led by Ri Liesner, from Great Ormond Street Hospital for Children in London, makes this recommendation based on their study in which they recorded the outcome of BPL 8Y treatment for seven children with TTP...more
Recent Advances in Thrombotic Thrombocytopenic Purpura
Thrombotic thrombocytopenic purpura (TTP) is characterized by microangiopathic hemolytic anemia and thrombocytopenia, accompanied by microvascular thrombosis that causes variable degrees of tissue ischemia and infarction. Intravascular coagulation is not a prominent feature of the disorder. Plasma exchange can induce remissions in approximately 80% of patients with idiopathic TTP, but patients have a much worse prognosis when thrombotic microangiopathy is associated with cancer, certain drugs, infections, or tissue transplantation. Recently, acquired autoimmune deficiency of a plasma metalloprotease named ADAMTS13 was shown to cause many cases of idiopathic TTP. This review describes our current understanding of how to use this knowledge clinically...more