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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

4. Immune thrombocytopenic purpura

Immune thrombocytopenic purpura Immune thrombocytopenia - Symptoms, diagnosis and treatment | BMJ Best Practice You'll need a subscription to access all of BMJ Best Practice Search  Immune thrombocytopenia Last reviewed: February 2019 Last updated: March 2019 Summary Immune thrombocytopenia (ITP), also known as immune thrombocytopenic purpura, is defined as an autoimmune haematological disorder characterised by isolated thrombocytopenia in the absence of an identifiable cause. Typically found (...) immune thrombocytopenia (ITP) is a haematological disorder characterised by isolated thrombocytopenia (platelet count <100 × 10⁹/L [<100 × 10³/microlitre]) in the absence of an identifiable cause. Rodeghiero F, Stasi R, Gernsheimer T, et al. Standardization of terminology, definitions and outcome criteria in immune thrombocytopenic purpura of adults and children: report from an international working group. Blood. 2009 Mar 12;113(11):2386-93. http://bloodjournal.hematologylibrary.org/cgi/content/full

2018 BMJ Best Practice

5. Henoch-Schonlein purpura

Henoch-Schonlein purpura Henoch-Schonlein purpura - Symptoms, diagnosis and treatment | BMJ Best Practice You'll need a subscription to access all of BMJ Best Practice Search  Henoch-Schonlein purpura Last reviewed: February 2019 Last updated: March 2018 Summary Most common vasculitis of childhood. Rash of palpable purpura is present in all cases. Most cases resolve with symptomatic treatment. Complications can occur, with renal failure being the most common cause of death. Definition Henoch (...) -Schonlein purpura (HSP) is the most common vasculitis of childhood and affects the small vessels. HSP is characterised by the classic tetrad of rash, abdominal pain, arthritis/arthralgia, and glomerulonephritis. Roberts PF, Waller TA, Brinker TM, et al. Henoch-Schonlein purpura: a review article. South Med J. 2007;100:821-824. http://www.ncbi.nlm.nih.gov/pubmed/17713309?tool=bestpractice.com History and exam presence of risk factors arthralgias abdominal pain rash signs of renal disease hx of taking

2018 BMJ Best Practice

6. 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

7. 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

8. Inherited thrombotic thrombocytopenic purpura mimicking immune thrombocytopenic purpura during pregnancy: a case report (Full text)

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 (...) thrombocytopenic purpura may mimic the signs and symptoms of pre-eclampsia/eclampsia, hemolysis with elevated liver enzymes and low platelet count syndrome, and atypical hemolytic-uremic syndrome. It should be considered in suspect cases in patients with an ADAMTS13 activity at 5% without ADAMTS13 antibodies.

2018 Journal of medical case reports PubMed

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

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

2018 Journal of medical case reports PubMed

10. 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

11. Atypical purpura location in a pediatric patient with Henoch-Schönlein purpura: A case report. (PubMed)

Atypical purpura location in a pediatric patient with Henoch-Schönlein purpura: A case report. The presence of purpura is a compulsory criteria for the diagnosis of Henoch-Schönlein purpura (HSP). Typical purpura of HSP is distributed symmetrically over the extensor surfaces of the lower limbs, buttocks, and forearms with the occasional involvement of trunk and face in children. It occurs only involving the bottom of the feet has never been reported.A 7-year-old girl was admitted (...) to the hospital with abdominal pain, vomiting, and fever.Combining clinical manifestations with results of radiologic examinations, acute appendicitis was suspected and a laparotomy was considered. Purpura was found on the bottom of her feet when she was in the operating room and HSP was diagnosed.The patient was treated with glucocorticosteroids, antibiotics, cimetidine, and restriction of feeding.The abdominal pain and purpura resolved at discharge and there were no recurrences in the subsequent 3-, 6

2018 Medicine

12. Fostamatinib for chronic immune thrombocytopenic purpura

Fostamatinib for chronic immune thrombocytopenic purpura Fostamatinib for chronic immune thrombocytopenic purpura Fostamatinib for chronic immune thrombocytopenic purpura NIHR HSRIC Record Status This is a bibliographic record of a published health technology assessment from a member of INAHTA. No evaluation of the quality of this assessment has been made for the HTA database. Citation NIHR HSRIC. Fostamatinib for chronic immune thrombocytopenic purpura. Birmingham: NIHR Horizon Scanning (...) Research&Intelligence Centre. Horizon Scanning Review. 2016 Authors' conclusions Idiopathic thrombocytopenic purpura (ITP) occurs when the immune system destroys platelets, which are cells in the blood that are necessary for normal blood clotting. Persons with the disease have too few platelets in the blood which causes bleeding. Adults tend to get a chronic (long-term) form of the disease. Fostamatinib is a new drug for the treatment of ITP that is given as a tablet. Some studies have suggested

2016 Health Technology Assessment (HTA) Database.

13. 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

14. Thrombopénie sévère chez un nouveau né de mère splénectomisé pour purpura thrombopénique idiopathique: Severe thrombopenia in an infant born to mother splenectomized for idiopathic thrombopenic purpura (Full text)

Thrombopénie sévère chez un nouveau né de mère splénectomisé pour purpura thrombopénique idiopathique: Severe thrombopenia in an infant born to mother splenectomized for idiopathic thrombopenic purpura Neonatal thrombopenia is the most common hemostatic abnormality in newborns. It is defined as a platelet count below 150.000/mm3. 40% of newborns to mothers with a history of autoimmune thrombopenia are at risk of developing neonatal thrombopenia while 10-15% of them are at risk (...) of developing severe thrombopenia. We here report the case of a 20 days old newborn to mother splenectomized for idiopathic thrombopenic purpura in order to highlight the relationship between the severity of maternal disease and the severity of the neonatal thrombopenia and thereby to avoid the risk of intracranial hemorrhage resulting in death or neurological sequelae.

2017 The Pan African medical journal PubMed

15. Systematic review of the role of rituximab in treatment of antineutrophil cytoplasmic autoantibody-associated vasculitis, hepatitis C virus-related cryoglobulinemic vasculitis, Henoch-Schönlein purpura, ankylosing spondylitis, and Raynaud's phenomenon. (Full text)

Systematic review of the role of rituximab in treatment of antineutrophil cytoplasmic autoantibody-associated vasculitis, hepatitis C virus-related cryoglobulinemic vasculitis, Henoch-Schönlein purpura, ankylosing spondylitis, and Raynaud's phenomenon. Rituximab (RTX) is established for the treatment of rheumatoid arthritis. This systematic review of the literature since 2006 summarizes evidence for the use of RTX in the treatment of additional rheumatological diseases: antineutrophil (...) cytoplasmic autoantibody-associated vasculitis (AAV), hepatitis C virus-related cryoglobulinemic vasculitis, Henoch-Schönlein purpura, ankylosing spondylitis, and Raynaud's phenomenon. Data from randomized controlled trials are available only for AAV, confirming efficacy for remission induction, including in disease resistant to conventional treatment, and maintenance of remission. Further studies are required to confirm optimal maintenance regimens in AAV, important questions needing to be addressed

2018 Open access rheumatology : research and reviews PubMed

16. The remarkable diversity of thrombotic thrombocytopenic purpura: a perspective (Full text)

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

2018 Blood advances PubMed

17. Persistence of endothelial thrombomodulin in a patient with infectious purpura fulminans treated with protein C concentrate (Full text)

Persistence of endothelial thrombomodulin in a patient with infectious purpura fulminans treated with protein C concentrate 30396911 2018 12 07 2473-9537 2 21 2018 Nov 13 Blood advances Blood Adv Persistence of endothelial thrombomodulin in a patient with infectious purpura fulminans treated with protein C concentrate. 2917-2921 10.1182/bloodadvances.2018024430 Bendapudi Pavan K PK Division of Hematology and Blood Transfusion Service, Massachusetts General Hospital, Boston, MA. Division

2018 Blood advances PubMed

18. Non-specific skin purpura (Full text)

Non-specific skin purpura Essential thrombocythemia is one of the myeloproliferative neoplasms. Palpable purpura is a rare manifestation that may delay diagnosis and treatment. We report a case of essential thrombocythemia in a 50-year-old man, who presented with recurrent thigh pain for the past one year with nonspecific localized purpura. His full blood count revealed isolated thrombocytosis of 880,000/µL with an impression of myeloproliferative disorder from peripheral blood film. He

2018 Malaysian family physician : the official journal of the Academy of Family Physicians of Malaysia PubMed

19. Interventions for preventing and treating kidney disease in Henoch-Schönlein Purpura (HSP). (PubMed)

Interventions for preventing and treating kidney disease in Henoch-Schönlein Purpura (HSP). Henoch-Schönlein purpura (HSP) is the most common vasculitis of childhood but may occur in adults. This small vessel vasculitis is characterised by palpable purpura, abdominal pain, arthritis or arthralgia and kidney involvement. This is an update of a review first published in 2009.To evaluate the benefits and harms of different agents (used singularly or in combination) compared with placebo

2015 Cochrane

20. External validation of the PLASMIC score: a clinical prediction tool for thrombotic thrombocytopenic purpura diagnosis and treatment (Full text)

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

2018 EvidenceUpdates PubMed

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