دورية أكاديمية
أعرض في EDS

Thrombotic thrombocytopenic purpura: pathogenesis, diagnosis and potential novel therapeutics.

التفاصيل البيبلوغرافية
العنوان: Thrombotic thrombocytopenic purpura: pathogenesis, diagnosis and potential novel therapeutics.
المؤلفون: Saha M; Division of Nephrology, Departments of Medicine, The University of Alabama at Birmingham, Birmingham, AL, USA., McDaniel JK; Division of Hematology/Oncology, Department of Pediatrics, The University of Alabama at Birmingham, Birmingham, AL, USA., Zheng XL; Division of Laboratory Medicine, Department of Pathology, The University of Alabama at Birmingham, Birmingham, AL, USA.
المصدر: Journal of thrombosis and haemostasis : JTH [J Thromb Haemost] 2017 Oct; Vol. 15 (10), pp. 1889-1900. Date of Electronic Publication: 2017 Jul 27.
نوع المنشور: Journal Article; Review; Research Support, N.I.H., Extramural
اللغة: English
بيانات الدورية: Publisher: Elsevier Country of Publication: England NLM ID: 101170508 Publication Model: Print-Electronic Cited Medium: Internet ISSN: 1538-7836 (Electronic) Linking ISSN: 15387836 NLM ISO Abbreviation: J Thromb Haemost Subsets: MEDLINE
أسماء مطبوعة: Publication: 2023- : [New York] : Elsevier
Original Publication: Oxford : Blackwell Pub.
مواضيع طبية MeSH: Genetic Therapy* , Hemostasis* , Immunotherapy* , Plasma Exchange*, ADAMTS13 Protein/*therapeutic use , Purpura, Thrombotic Thrombocytopenic/*diagnosis , Purpura, Thrombotic Thrombocytopenic/*therapy, ADAMTS13 Protein/blood ; ADAMTS13 Protein/genetics ; ADAMTS13 Protein/immunology ; Anemia, Hemolytic/blood ; Anemia, Hemolytic/diagnosis ; Anemia, Hemolytic/therapy ; Autoantibodies/blood ; Diagnosis, Differential ; Humans ; Predictive Value of Tests ; Purpura, Thrombotic Thrombocytopenic/blood ; Purpura, Thrombotic Thrombocytopenic/epidemiology ; Recombinant Proteins/therapeutic use ; Risk Factors ; Thrombocytopenia/blood ; Thrombocytopenia/diagnosis ; Thrombocytopenia/therapy ; Treatment Outcome
مستخلص: Thrombotic thrombocytopenic purpura (TTP), a potentially fatal clinical syndrome, is primarily caused by autoantibodies against the von Willebrand factor (VWF)-cleaving metalloprotease ADAMTS-13. In general, severe deficiency of plasma ADAMTS-13 activity (< 10 IU dL -1 ) with or without detectable inhibitory autoantibodies against ADAMTS-13 supports the diagnosis of TTP. A patient usually presents with thrombocytopenia and microangiopathic hemolytic anemia (i.e. schistocytes, elevated serum lactate dehydrogenase, decreased hemoglobin and haptoglobin) without other known etiologies that cause thrombotic microangiopathy (TMA). Normal to moderately reduced plasma ADAMTS-13 activity (> 10 IU dL -1 ) in a similar clinical context supports an alternative diagnosis such as atypical hemolytic uremic syndrome (aHUS) or other types of TMA. Prompt differentiation of TTP from other causes of TMA is crucial for the initiation of an appropriate therapy to reduce morbidity and mortality. Although plasma infusion is often sufficient for prophylaxis or treatment of hereditary TTP due to ADAMTS-13 mutations, daily therapeutic plasma exchange remains the initial treatment of choice for acquired TTP with demonstrable autoantibodies. Immunomodulatory therapies, including corticosteroids, rituximab, vincristine, cyclosporine, cyclophosphamide and splenectomy, etc., should be considered to eliminate autoantibodies for a sustained remission. Other emerging therapeutic modalities, including recombinant ADAMTS-13, adeno-associated virus (AAV) 8-mediated gene therapy, platelet-delivered ADAMTS-13, and antagonists targeting the interaction between platelet glycoprotein 1b and VWF are under investigation. This review highlights the recent progress in our understanding of the pathogenesis and diagnosis of, and current and potential novel therapies for, hereditary and acquired TTP.
(© 2017 International Society on Thrombosis and Haemostasis.)
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معلومات مُعتمدة: R01 HL115187 United States HL NHLBI NIH HHS; R01 HL126724 United States HL NHLBI NIH HHS; T32 DK007545 United States DK NIDDK NIH HHS
فهرسة مساهمة: Keywords: diagnosis; pathology; therapeutics; thrombotic microangiopathies; von Willebrand factor
المشرفين على المادة: 0 (Autoantibodies)
0 (Recombinant Proteins)
EC 3.4.24.87 (ADAMTS13 Protein)
EC 3.4.24.87 (ADAMTS13 protein, human)
تواريخ الأحداث: Date Created: 20170630 Date Completed: 20180723 Latest Revision: 20230829
رمز التحديث: 20240628
مُعرف محوري في PubMed: PMC5630501
DOI: 10.1111/jth.13764
PMID: 28662310
قاعدة البيانات: MEDLINE
الوصف
تدمد:1538-7836
DOI:10.1111/jth.13764