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Thrombotic Thrombocytopenic Purpura

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1. Caplacizumab-yhdp (Cablivi) - To treat adult patients with acquired thrombotic thrombocytopenic purpura (aTTP)

Caplacizumab-yhdp (Cablivi) - To treat adult patients with acquired thrombotic thrombocytopenic purpura (aTTP) Drug Approval Package: CABLIVI U.S. Department of Health and Human Services Search FDA Submit search Drug Approval Package: CABLIVI Company: Ablynx NV Application Number: 761112 Approval Date: 02/06/2019 Persons with disabilities having problems accessing the PDF files below may call (301) 796-3634 for assistance. FDA Approval Letter and Labeling (PDF) (PDF) FDA Application Review

2019 FDA - Drug Approval Package

3. Thrombotic thrombocytopenic purpura

Thrombotic thrombocytopenic purpura Thrombotic thrombocytopenic purpura - Symptoms, diagnosis and treatment | BMJ Best Practice You'll need a subscription to access all of BMJ Best Practice Search  Thrombotic thrombocytopenic purpura Last reviewed: February 2019 Last updated: October 2017 Summary This topic focuses on acquired (idiopathic) TTP. TTP is a potential diagnosis in any patient with haemolytic anaemia and thrombocytopenia - 95% of cases are fatal if left untreated. Symptoms (...) therapy is the mainstay of treatment for acute acquired (idiopathic) TTP. Renal and neurological dysfunctions are the main complications. Definition Thrombotic thrombocytopenic purpura (TTP) is a clinical syndrome characterised by microangiopathic haemolytic anaemia and thrombocytopenic purpura. Although the original descriptions included a pentad of microangiopathic haemolytic anaemia, thrombocytopenic purpura, neurological dysfunction, renal dysfunction, and fever, most patients do not have

2017 BMJ Best Practice

4. Solvent Detergent Plasma versus Standard Plasma for the Treatment of Thrombotic Thrombocytopenic Purpura: Comparative Clinical Effectiveness and Cost-Effectiveness

Solvent Detergent Plasma versus Standard Plasma for the Treatment of Thrombotic Thrombocytopenic Purpura: Comparative Clinical Effectiveness and Cost-Effectiveness Solvent Detergent Plasma versus Standard Plasma for the Treatment of Thrombotic Thrombocytopenic Purpura: Comparative Clinical Effectiveness and Cost-Effectiveness | CADTH.ca Find the information you need Solvent Detergent Plasma versus Standard Plasma for the Treatment of Thrombotic Thrombocytopenic Purpura: Comparative Clinical (...) Effectiveness and Cost-Effectiveness Solvent Detergent Plasma versus Standard Plasma for the Treatment of Thrombotic Thrombocytopenic Purpura: Comparative Clinical Effectiveness and Cost-Effectiveness Published on: August 15, 2017 Project Number: RA0920-000 Product Line: Research Type: Devices and Systems Report Type: Reference List Result type: Report Question What is the comparative clinical effectiveness of solvent detergent plasma versus standard plasma for the treatment of thrombotic thrombocytopenic

2017 Canadian Agency for Drugs and Technologies in Health - Rapid Review

5. Prospective Psychometric Evaluation Study of a Patient-reported Outcomes (PRO) Instrument for Congenital Thrombotic Thrombocytopenic Purpura (cTTP, Upshaw-Schulman Syndrome [USS], Hereditary Thrombotic Thrombocytopenic Purpura [hTTP]

Prospective Psychometric Evaluation Study of a Patient-reported Outcomes (PRO) Instrument for Congenital Thrombotic Thrombocytopenic Purpura (cTTP, Upshaw-Schulman Syndrome [USS], Hereditary Thrombotic Thrombocytopenic Purpura [hTTP] Prospective Psychometric Evaluation Study of a Patient-reported Outcomes (PRO) Instrument for Congenital Thrombotic Thrombocytopenic Purpura (cTTP, Upshaw-Schulman Syndrome [USS], Hereditary Thrombotic Thrombocytopenic Purpura [hTTP] - Full Text View (...) - ClinicalTrials.gov Hide glossary Glossary Study record managers: refer to the if submitting registration or results information. Search for terms x × Study Record Detail Saved Studies Save this study Warning You have reached the maximum number of saved studies (100). Please remove one or more studies before adding more. Prospective Psychometric Evaluation Study of a Patient-reported Outcomes (PRO) Instrument for Congenital Thrombotic Thrombocytopenic Purpura (cTTP, Upshaw-Schulman Syndrome [USS], Hereditary

2018 Clinical Trials

6. Caplacizumab Treatment for Acquired Thrombotic Thrombocytopenic Purpura. (PubMed)

Caplacizumab Treatment for Acquired Thrombotic Thrombocytopenic Purpura. In acquired thrombotic thrombocytopenic purpura (TTP), an immune-mediated deficiency of the von Willebrand factor-cleaving protease ADAMTS13 allows unrestrained adhesion of von Willebrand factor multimers to platelets and microthrombosis, which result in thrombocytopenia, hemolytic anemia, and tissue ischemia. Caplacizumab, an anti-von Willebrand factor humanized, bivalent variable-domain-only immunoglobulin fragment

2019 NEJM

7. Inherited thrombotic thrombocytopenic purpura mimicking immune thrombocytopenic purpura during pregnancy: a case report (PubMed)

Inherited thrombotic thrombocytopenic purpura mimicking immune thrombocytopenic purpura during pregnancy: a case report Thrombotic thrombocytopenic purpura is a very rare hereditary blood deficiency disorder of ADAMTS13 (von Willebrand factor-cleaving protease) and a life-threatening thrombotic microangiopathy characterized by thrombocytopenia and microangiopathic hemolytic anemia. The deficiency in ADAMTS13 metalloprotease, which cleaves the von Willebrand factor, may be congenital or acquired (...) . The congenital form is caused by inherited mutations in the ADAMTS13 gene. The diagnosis is challenging due to the nonspecific signs and symptoms, as well as the rarity of the disease.We present an unusual case of a 20-year-old feoderm woman from northeast region of Brazil who manifested thrombocytopenia during her pregnancy which was believed to be immune thrombocytopenic purpura.Considering the importance of a differential diagnosis of thrombotic microangiopathic disorders, congenital thrombotic

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2018 Journal of medical case reports

8. Post-Partum Thrombotic Thrombocytopenic Purpura (TTP) in a Patient with known Idiopathic (Immune) Thrombocytopenic Purpura: a case report and review of the literature (PubMed)

Post-Partum Thrombotic Thrombocytopenic Purpura (TTP) in a Patient with known Idiopathic (Immune) Thrombocytopenic Purpura: a case report and review of the literature Incidences of immune thrombocytopenic purpura occur in 1 in every 1000-10,000 pregnancies accounting for 3% of all thrombocytopenic pregnancies. A pre-existing immune thrombocytopenic purpura is known to be a risk factor for developing thrombocytopenia during pregnancy. We present here the treatment regime and management (...) of a patient with known immune thrombocytopenic purpura who developed postpartum thrombotic thrombocytopenia with atypical response to traditional therapy. Pregnant women are more vulnerable to immune thrombocytopenic purpura or thrombotic thrombocytopenia. Pregnancy or postpartum thrombotic thrombocytopenia accounts for 10-25% of all thrombotic thrombocytopenia.This case report deals with the treatment regime and management of a patient with known immune thrombocytopenic purpura who developed postpartum

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2018 Journal of medical case reports

9. The remarkable diversity of thrombotic thrombocytopenic purpura: a perspective (PubMed)

The remarkable diversity of thrombotic thrombocytopenic purpura: a perspective Understanding the autoimmune etiology of acquired thrombotic thrombocytopenic purpura (TTP) has provided precision for the diagnosis and a rationale for immunosuppressive treatment. These advances have also allowed recognition of the remarkable clinical diversities of patients' initial presentations and their long-term outcomes. These diversities are illustrated by the stories of patients from the Oklahoma TTP

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2018 Blood advances

10. External validation of the PLASMIC score: a clinical prediction tool for thrombotic thrombocytopenic purpura diagnosis and treatment

External validation of the PLASMIC score: a clinical prediction tool for thrombotic thrombocytopenic purpura diagnosis and treatment Essentials Severe ADAMTS-13 deficiency is key to thrombotic thrombocytopenic purpura (TTP) diagnosis. PLASMIC score predicts ADAMTS-13 deficiency in suspected TTP with high discrimination. PLASMIC score is more generalizable with fewer missing data than alternative clinical scores. PLASMIC score identifies a subgroup of patients lacking significant response (...) to plasma exchange.Background The PLASMIC score was recently published to distinguish patients with severe ADAMTS-13 deficiency from those without for early identification of thrombotic thrombocytopenia purpura (TTP). Objective We performed an independent external validation of the PLASMIC score for clinical prediction of severe ADAMTS-13 deficiency. Patients/Methods We studied an independent cohort of 112 consecutive hospitalized patients with suspected thrombotic microangiopathy and appropriate ADAMTS

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2018 EvidenceUpdates

11. Recombinant ADAMTS 13 in thrombotic thrombocytopenic purpura (PubMed)

Recombinant ADAMTS 13 in thrombotic thrombocytopenic purpura 29344548 2018 11 13 2331-4737 4 11-12 2017 Nov Oncoscience Oncoscience Recombinant ADAMTS 13 in thrombotic thrombocytopenic purpura. 160-161 10.18632/oncoscience.380 Scully Marie M Department of Haemotology; University College London Hospitals NHS Trust, Cardiometabolic Programme-NHR UCLH/UCL BRC, London, UK. Hibbard Christopher C Department of Haemotology; University College London Hospitals NHS Trust, Cardiometabolic Programme-NHR

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2017 Oncoscience

12. Thrombotic thrombocytopenic purpura: diagnostic criteria, clinical features, and long-term outcomes from 1995 through 2015 (PubMed)

Thrombotic thrombocytopenic purpura: diagnostic criteria, clinical features, and long-term outcomes from 1995 through 2015 Our objective was to describe new observations from the Oklahoma Thrombotic Thrombocytopenic Purpura (TTP) Registry experience (November 1995 through December 2015) on the diagnosis of TTP along with patients' clinical features and their outcomes. Among 363 patients with an initial episode of clinically suspected TTP, the diagnosis of TTP was supported by both ADAMTS13

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2017 Blood advances

13. Concurrent Presentation of Thrombotic Thrombocytopenic Purpura and Membranous Nephropathy (PubMed)

Concurrent Presentation of Thrombotic Thrombocytopenic Purpura and Membranous Nephropathy 29725652 2019 02 26 2468-0249 3 2 2018 Mar Kidney international reports Kidney Int Rep Concurrent Presentation of Thrombotic Thrombocytopenic Purpura and Membranous Nephropathy. 476-481 10.1016/j.ekir.2017.08.005 Al-Rabadi Laith L Renal Division, Department of Medicine, University of Utah Hospital, Salt Lake, Utah, USA. Quillen Karen K Department of Pathology & Laboratory Medicine, Boston University School

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2017 Kidney international reports

14. Cyclosporine or steroids as an adjunct to plasma exchange in the treatment of immune-mediated thrombotic thrombocytopenic purpura (PubMed)

Cyclosporine or steroids as an adjunct to plasma exchange in the treatment of immune-mediated thrombotic thrombocytopenic purpura Although steroids are routinely used as an adjunct to plasma exchange (PEX) therapy in the treatment of immune-mediated thrombotic thrombocytopenic purpura (iTTP), limited data regarding their efficacy or effect on ADAMTS13 biomarkers are available. We report the results of a prospective, randomized study that compared the effectiveness of prednisone or cyclosporine

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2017 Blood advances

15. Rituximab prophylaxis to prevent thrombotic thrombocytopenic purpura relapse: outcome and evaluation of dosing regimens (PubMed)

Rituximab prophylaxis to prevent thrombotic thrombocytopenic purpura relapse: outcome and evaluation of dosing regimens Acute antibody-mediated thrombotic thrombocytopenic purpura (TTP) is a thrombotic microangiopathy with high morbidity and mortality. Rituximab is highly effective as prophylaxis in patients at risk of acute TTP relapse, but the ideal dosing regimen is unknown. A multicenter retrospective cohort study evaluated outcomes of patients given rituximab prophylaxis to prevent TTP

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2017 Blood advances

16. Long-term Kidney Outcomes in Patients With Acquired Thrombotic Thrombocytopenic Purpura (PubMed)

Long-term Kidney Outcomes in Patients With Acquired Thrombotic Thrombocytopenic Purpura Severe acute kidney injury (AKI) and chronic kidney disease (CKD) are considered to be uncommon in patients with acquired thrombotic thrombocytopenic purpura. However, a recent case series from a tertiary care hospital indicated that 54 (59%) of 92 patients with thrombotic thrombocytopenic purpura presented with AKI; 14 (15%) required dialysis; and 12 (22%) of the 54 patients had CKD at follow-up.In (...) this prospective analysis of 78 patients diagnosed with their first episode of thrombotic thrombocytopenic purpura and enrolled in the Oklahoma Thrombotic Thrombocytopenic Purpura Registry from 1995 to 2015, we assessed AKI at diagnosis using Kidney Disease: Improving Global Outcomes criteria, and CKD at follow-up as defined by estimated glomerular filtration rate <60 ml/min per 1.73 m2 determined by the Chronic Kidney Disease-Epidemiology Collaboration equation.Forty-five (58%) patients had AKI; 8 (10%) had

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2017 Kidney international reports

17. Management of thrombotic thrombocytopenic purpura without plasma exchange: the Jehovah’s Witness experience (PubMed)

Management of thrombotic thrombocytopenic purpura without plasma exchange: the Jehovah’s Witness experience TTP in Jehovah's Witness patients has been managed successfully without PEX.This experience, plus new TTP treatments, may make it possible for patients who are not Jehovah's Witnesses to avoid PEX in the future.

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2017 Blood advances

18. Risk of diagnostic delay in congenital thrombotic thrombocytopenic purpura. (PubMed)

Risk of diagnostic delay in congenital thrombotic thrombocytopenic purpura. Essentials Congenital thrombotic thrombocytopenic purpura (cTTP) is a very rare thrombotic microangiopathy. Its rarity and great phenotype heterogeneity may account for misdiagnosis. We report the history of a middle-aged woman with cTTP, misdiagnosed until adulthood. Accurate clinical history is crucial for early diagnosis to prevent long-term sequelae. SUMMARY: Thrombotic thrombocytopenic purpura (TTP) is an acute

2019 Journal of Thrombosis and Haemostasis

19. New Treatment for Acquired Thrombotic Thrombocytopenic Purpura: Caplacizumab

New Treatment for Acquired Thrombotic Thrombocytopenic Purpura: Caplacizumab CasesBlog - Medical and Health Blog: New Treatment for Acquired Thrombotic Thrombocytopenic Purpura: Caplacizumab Health News Updated Daily by Internist and Allergist at Cleveland Clinic Florida Pages New Treatment for Acquired Thrombotic Thrombocytopenic Purpura: Caplacizumab Immune-mediated deficiency of the von Willebrand factor–cleaving protease ADAMTS13 allows unrestrained adhesion of von Willebrand factor (...) multimers to platelets and microthrombosis. This results in thrombocytopenia, hemolytic anemia, and tissue ischemia. These are the hallmarks of acquired thrombotic thrombocytopenic purpura (TTP). Caplacizumab is an anti–von Willebrand factor humanized. Caplacizumab is not a full antibody, but just a fragment if it, as you can see in the video below. It inhibits interaction between von Willebrand factor multimers and platelets. In this double-blind, controlled trial, 145 patients with TTP received

2019 CasesBlog - Medical and Health Blog

20. Prevention of relapse in patients with acquired thrombotic thrombocytopenic purpura undergoing elective surgery: a case series. (PubMed)

Prevention of relapse in patients with acquired thrombotic thrombocytopenic purpura undergoing elective surgery: a case series. Essentials Thrombotic thrombocytopenic purpura (TTP) is a life-threatening disease. Surgery is a possible trigger of acute TTP episodes and no guidelines are available. Six patients with severe ADAMTS-13 deficiency during remission underwent elective surgery. Patients were prophylactically treated to restore ADAMTS-13 activity and no relapses occurred. SUMMARY (...) : Background Severe ADAMTS-13 deficiency has been recognized as the main risk factor for recurrence of thrombotic thrombocytopenic purpura (TTP). Several conditions, including surgery, may influence the levels of ultra-large von Willebrand factor and ADAMTS-13, acting as a trigger for an acute TTP event. Objectives To report our experience of management of six patients with acquired TTP who underwent elective surgery after prophylactic treatment to restore ADAMTS-13 activity levels. Patients Six patients

2019 Journal of Thrombosis and Haemostasis

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