Search results for "MTS1"
showing 8 items of 28 documents
Idiopathic thrombotic thrombocytopenic purpura: strongest risk factor for relapse from remission is having had a relapse
2016
BACKGROUND Thrombotic thrombocytopenic purpura (TTP) is a rare, episodic clinical syndrome involving the production of thrombi in the microvasculature accompanied by thrombocytopenia and symptoms of organ ischemia. Idiopathic TTP develops when a patient produces antibodies that react with the protease ADAMTS13. The course after an episode is unpredictable; patients may relapse frequently or never. There is no laboratory value that can reliably predict potential relapse. STUDY DESIGN AND METHODS To assess diagnostic and predictive values for risk of relapse, plasma samples from 27 patients with idiopathic TTP in remission were analyzed for anti-ADAMTS13 immunoglobulin (Ig)G, ADAMTS13 activit…
Best practices and recommendations for drug regimens and plasma exchange for immune thrombotic thrombocytopenic purpura.
2021
Introduction Thrombotic thrombocytopenic purpura (TTP) is a life-threatening thrombotic microangiopathy characterized by microangiopathic hemolytic anemia, thrombocytopenia, and organ injury. TTP pathophysiology is based on a severe ADAMTS13 deficiency, and is a medical emergency with fatal outcome if appropriate treatment is not initiated promptly. Areas covered Authors will review the best options currently available to minimize mortality, prevent relapses, and obtain the best clinical response in patients with immune TTP (iTTP). Available bibliography about iTTP treatment has been searched in Library's MEDLINE/PubMed database from January 1990 until April 2021. Expert opinion The general…
Narratives of Patients with Fatal Outcomes During the Phase 2 TITAN and Phase 3 HERCULES Studies
2019
Background: Acquired thrombotic thrombocytopenic purpura (aTTP) is a rare but life-threatening thrombotic microangiopathy, with an untreated mortality rate of >90%. Prompt treatment with therapeutic plasma exchange (TPE) and immunosuppression improves outcomes in patients with aTTP, but 10-20% of patients still die acutely from this disease. The aim of this analysis was to describe in more detail the characteristics and disease courses of the patients who died during the caplacizumab clinical development program. Methods: Patient narratives on all deaths occurring during the phase 2 TITAN and phase 3 HERCULES studies were extracted. Results: In the overall study periods, a total of 6 pat…
Efficacy and safety of open-label caplacizumab in patients with exacerbations of acquired thrombotic thrombocytopenic purpura in the HERCULES study.
2020
BACKGROUND Acquired thrombotic thrombocytopenic purpura (aTTP) is a rare, life-threatening autoimmune thrombotic microangiopathy. Caplacizumab, an anti-von Willebrand Factor Nanobody® , is effective for treating aTTP episodes and is well tolerated. OBJECTIVES AND METHODS In the phase 3 HERCULES trial (NCT02553317), patients with aTTP received double-blind caplacizumab or placebo during daily therapeutic plasma exchange (TPE) and for ≥30 days thereafter. Patients who experienced an exacerbation while on blinded study drug treatment switched to receive open-label caplacizumab plus re-initiation of daily TPE. Exacerbations were defined as recurrence of disease occurring within 30 days after ce…
Thrombotic Thrombocytopenic Purpura: Pathophysiology, Diagnosis, and Management
2021
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. TTP results from a severe deficiency of the specific von Willebrand factor (VWF)-cleaving protease, ADAMTS13 (a disintegrin and metalloprotease with thrombospondin type 1 repeats, member 13). ADAMTS13 deficiency is most commonly acquired due to anti-ADAMTS13 autoantibodies. It can also be inherited in the congenital form as a result of biallelic mutations in the ADAMTS13 gene. In adults, the condition is most often immune-mediated (iTTP) whereas congenital TTP (cTT…
Algoritmo diagnóstico-terapéutico de las microangiopatías trombóticas. A propósito de 2 casos
2022
Thrombotic microangiopathies (TMA) are a group of clinical syndromes associated with haemolytic anaemia, thrombocytopenia and organ dysfunction, mainly renal or neurological. They are associated with significant morbidity and mortality, so early diagnosis and treatment are essential. In this article we report two cases of TMA; a patient with thrombotic thrombocytopenic purpura (TTP) and a patient with atypical haemolytic uraemic syndrome (aHUS).
No Evidence for Classic Thrombotic Microangiopathy in COVID-19
2021
Background: Coronavirus disease-2019 (COVID-19) triggers systemic infection with involvement of the respiratory tract. There are some patients developing haemostatic abnormalities during their infection with a considerably increased risk of death. Materials and Methods: Patients (n = 85) with SARS-CoV-2 infection attending the University Medical Center, Mainz, from 3 March to 15 May 2020 were retrospectively included in this study. Data regarding demography, clinical features, treatment and laboratory parameters were analyzed. Twenty patients were excluded for assessment of disseminated intravascular coagulation (DIC) and thrombotic microangiopathy (TMA) due to lack of laboratory data. Resu…
Patterns of changes of anti‐ADAMTS13 after plasma exchange
2006
An enzyme-linked immunosorbent assay (ELISA) has recently been developed to detect antibodies against the von Willebrand factor-cleaving protease ADAMTS13 in patients with thrombotic thrombocytopenic purpura (TTP). The ELISA is based on incubation of plasma with immobilized recombinant ADAMTS13 followed by visualization of IgG and IgM antibodies by means of secondary enzyme-labeled antibodies [1]. In a recent study, anti-ADAMTS13 IgG antibodies were detected in most patients with TTP (97%) characterized by severe ADAMTS13 deficiency (< 10% of normal) [1]. The ELISA was more sensitive than the standard inhibitor assay based upon ADAMTS13 neutralizing activity, which gave positive results in …