https://www.ncbi.nlm.nih.gov/pmc/articles/PMC8091490/
Twelve Population, Intervention, Comparison, and Outcome treatment questions (PICO) (Appendix S1C) relating to the treatment of acute immune-mediated TTP ( iTTP) and hereditary/congenital TTP (cTTP) (the first event, the relapse, and during remission without and with pregnancy) were fully appraised and recommendations were provided.

https://ashpublications.org/blood/article/133/15/1644/273301/Characterization-and-treatment-of-congenital
Congenital thrombotic thrombocytopenic purpura (cTTP), also known as Upshaw-Schulman syndrome, is an ultra-rare thrombomicroangiopathy due to an inherited deficiency of the von Willebrand factor (VWF)-cleaving metalloprotease, a disintegrin and metalloproteinase with a thrombospondin type 1 motif, member 13 (ADAMTS13), resulting in the abnormal presence of ultra-large VWF multimers and the

https://onlinelibrary.wiley.com/doi/full/10.1111/jth.15010
The panel discussed 12 treatment questions related to immune-mediated TTP (iTTP) and hereditary or congenital TTP (cTTP). The panel used the Grading of Recommendations Assessment, Development, and Evaluation approach, including evidence-to-decision frameworks, to appraise evidence and formulate recommendations.

https://ashpublications.org/blood/article/137/25/3469/476205/Congenital-TTP-next-stop-acuity-and-therapy
Hereditary TTP or cTTP was initially described by Schulman and colleagues in 1960. 2 They presented a case of chronic thrombocytopenia in which plasma therapy resulted in an increase in the platelet count. In 1978, Upshaw 3 concluded that his patient with recurrent acute episodes of severe thrombocytopenia had a congenital deficiency in a factor present in plasma that had an important role in

https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10219024/
Congenital thrombotic thrombocytopenic purpura (cTTP) is an extremely rare disease characterized by the severe deficiency of a disintegrin and metalloproteinase with thrombospondin type 1 motifs 13 (ADAMTS13), caused by ADAMTS13 mutations. While ADAMTS13 supplementation by fresh frozen plasma (FFP) infusion immediately corrects platelet consumption and resolves thrombotic symptoms in acute

https://my.clevelandclinic.org/health/diseases/22380-thrombotic-thrombocytopenic-purpura
Thrombotic thrombocytopenic purpura (TTP) is a rare blood disorder in which thrombi (blood clots) form in small blood vessels throughout your body. These blood clots can restrict the flow of oxygen-rich blood to your organs, causing a number of life-limiting complications. Treatments include plasma therapy, medication and surgery.

https://www.ncbi.nlm.nih.gov/pmc/articles/PMC7867179/
Thrombotic thrombocytopenic purpura (TTP) is a rare thrombotic microangiopathy characterized by microangiopathic hemolytic anemia, severe thrombocytopenia, and ischemic end organ injury due to microvascular platelet-rich thrombi. ... Shortly thereafter, case reports detailing the successful treatment of congenital TTP (cTTP) patients with

https://link.springer.com/article/10.1007/s12185-023-03657-0
Thrombotic thrombocytopenic purpura (TTP) can rapidly become a life-threatening condition, and the importance of its appropriate diagnosis and treatment cannot be overstated. Until recently, TTP has mainly been diagnosed by clinical findings such as thrombocytopenia and hemolytic anemia. In addition to these clinical findings, however, reduced activity of a disintegrin-like and metalloprotease

https://pubmed.ncbi.nlm.nih.gov/32914526/
Methods: In June 2018, the ISTH formed a multidisciplinary guideline panel to issue recommendations about treatment of TTP. The panel discussed 12 treatment questions related to immune-mediated TTP (iTTP) and hereditary or congenital TTP (cTTP). The panel used the Grading of Recommendations Assessment, Development, and Evaluation approach

https://ashpublications.org/ashclinicalnews/news/5315/New-Rules-of-the-Road-in-TTP-Management
The TTP Times Are Changing. For a disease that hasn't had a major breakthrough since the identification of ADAMTS13 in 2001 or arguably the use of rituximab in the mid-2000s, the past 2 years have been tumultuous in the TTP world. 1,2 The publication of the HERCULES study results in January 2019 put a spotlight on caplacizumab as a potentially game-changing treatment. 3 Just 1 month later, it

https://emedicine.medscape.com/article/206598-treatment
A recombinant ADMTS13 (Adzynma) was approved by the FDA in 2023 for prophylactic or on-demand enzyme replacement therapy in adults and children with congenital thrombotic thrombocytopenic purpura (cTTP). Approval was based on data from a randomized, open-label crossover phase 3 trial and continuation study in which none of the 38 patients experienced acute TTP events during a mean of 13.2

https://www.nejm.org/doi/full/10.1056/NEJMoa1806311
The median time to normalization of the platelet count was shorter with caplacizumab than with placebo (2.69 days [95% confidence interval {CI}, 1.89 to 2.83] vs. 2.88 days [95% CI, 2.68 to 3.56

https://onlinelibrary.wiley.com/doi/epdf/10.1111/jth.15010
Abstract. Background: Despite advances in treatment options for thrombotic thrombocyto-penic purpura (TTP), there are still limited high quality data to inform clinicians re-garding its appropriate treatment. Methods: In June 2018, the ISTH formed a multidisciplinary guideline panel to issue recommendations about treatment of TTP.

https://www.ncbi.nlm.nih.gov/books/NBK470585/
Thrombotic thrombocytopenic purpura (TTP) is a rare microangiopathic hemolytic anemia that causes blood clots in small blood vessels. TTP is characterized by fever, hemolytic anemia, thrombocytopenia, and renal and neurologic dysfunction. TTP results from either a congenital or acquired absence or decrease of the von Willebrand factor-cleaving protease ADAMTS13 (a disintegrin and

https://www.fda.gov/news-events/press-announcements/fda-approves-first-treatment-patients-rare-inherited-blood-clotting-disorder
The efficacy of Adzynma in the prophylactic treatment of patients with cTTP was evaluated in 46 patients who were randomized to receive 6 months of treatment with either Adzynma or plasma based

https://www.nhlbi.nih.gov/health/thrombotic-thrombocytopenic-purpura
This may cause bleeding and bruising. "Thrombotic" refers to the blood clots that form. "Thrombocytopenic" means the blood has a lower-than-normal platelet count. "Purpura" refers to purple bruises caused by bleeding under your skin. TTP usually occurs suddenly and lasts for days or weeks, but it can continue for months.

https://www.jthjournal.org/article/S1538-7836(22)01180-1/fulltext
The panel discussed 12 treatment questions related to immune‐mediated TTP (iTTP) and hereditary or congenital TTP (cTTP). The panel used the Grading of Recommendations Assessment, Development, and Evaluation approach, including evidence‐to‐decision frameworks, to appraise evidence and formulate recommendations.

https://ashpublications.org/blood/article/116/20/4060/28083/How-I-treat-patients-with-thrombotic
Names matter. The name of a syndrome is clinically important because it can trigger treatment. For example, the name TTP implies a disorder that is fatal without effective treatment, 3 whereas the name HUS implies that supportive care may be sufficient. 4 Table 1 presents my current understanding of the common usage of the terms thrombotic microangiopathy (TMA), TTP, and HUS.

https://www.takeda.com/newsroom/newsreleases/2023/takeda-adzynma-approved-by-fda-as-the-first-and-only-recombinant-adamts13-enzyme-replacement-therapy-for-the-treatment-of-congenital-thrombotic-thrombocytopenic-purpura/
, including information for patients. ABOUT cTTP. cTTP is an ultra-rare, chronic and debilitating clotting disorder associated with life-threatening acute events and debilitating chronic symptoms, or TTP manifestations. 6,7 TTP has an estimated prevalence of 2-6 cases/million. The inherited form of the disease, cTTP, accounts for ≤5% of TTP patients. 7,8,9 It develops due to deficiency in

https://www.ncbi.nlm.nih.gov/pmc/articles/PMC6055500/
Thrombotic thrombocytopenic purpura (TTP) is characterized by microangiopathic hemolytic anemia and a consumptive thrombocytopenia, as a result of severe deficiency of ADAMTS 13.The standard of care of the acute episode is treatment with plasma exchange and immunosuppression. After the acute episode is resolved, patients face a significant risk of relapse and long‐term complications

https://www.drugs.com/news/fda-approves-first-congenital-thrombotic-thrombocytopenic-purpura-116056.html
MONDAY, Nov. 13, 2023 -- The U.S. Food and Drug Administration has approved Adzynma, the first recombinant protein product indicated for prophylactic or on-demand enzyme replacement therapy (ERT) in adult and pediatric patients with congenital thrombotic thrombocytopenic purpura (cTTP).

https://ashpublications.org/blood/article/124/2/211/32922/Thrombotic-thrombocytopenic-purpura-and-pregnancy
Thrombotic thrombocytopenic purpura (TTP) is an acute, rare, potentially life-threatening disorder, presenting with thrombocytopenia, hemolytic anemia, and clinical consequences of microvascular thrombosis, caused by deficiency of ADAMTS13. 1,2 The majority of acute cases are acquired, autoantibody mediated, and characterized by low ADAMTS13 activity (<10%) and the presence of anti-ADAMTS13

https://www.ncbi.nlm.nih.gov/pmc/articles/PMC8185636/
Thrombotic thrombocytopenic purpura (TTP) is a thrombotic microangiopathy (TMA), characterized by the spontaneous formation of thrombi in the microcirculation. 1 The diagnosis of TMA relies on the association of hemolytic mechanical anemia, peripheral thrombocytopenia and various signs of visceral ischemia ascribable to microvessel thrombosis.