Use of a TGA and TEM in the Assessment of the Efficacy of Treatment With APCC or rFVIIa
NCT ID: NCT01856751
Last Updated: 2015-12-18
Study Results
The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.
Basic Information
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UNKNOWN
80 participants
OBSERVATIONAL
2014-04-30
2016-06-30
Brief Summary
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Response to treatment with by-passing agents is patient-specific, and can vary in the same patient during subsequent bleedings. Some patients have good response to both products, however in other patients a better bleeding control is provided by one of the mentioned above agents (APCC or rFVIIa). There are clinical situations when severe bleedings requires an alternate use of both these agents.
Traditional methods of laboratory tests used post-treatment in patients with haemophilia without inhibitors are useless in the presence of inhibitor. Laboratory monitoring of therapy with by-passing agents is possible with the use of global tests for the coagulation process assessment, which are as follows: thrombin generation assay (TGA) and thromboelastometry (TEM).
Several studies revealed that TGA allows a monitoring of therapy with by-passing agents in patients with haemophilia A and inhibitor - the choice of the most effective treatment option - agent type and its dose, as well as laboratory assessment of treatment efficacy.
Up to date, laboratory tests assessing the efficacy of by-passing agents in patients with acquired haemophilia were not conducted.
In Factor VIII or IX deficiency conditions, fibrin's fibres generated by thrombin are morphologically thicker, and blood clots have increased susceptibility to fibrinolytic enzymes. Blood clot stability may be assessed with the use of thromboelastometry (TEM). We can hypothesize that simultaneous use of TGA and TEM methods may allow for an assessment of patient's individual response to therapy with by-passing agents. Clinical significance of the minimal dose of APCC and rFVIIa, needed to TGA and TEM normalization, requires further studies.
Tests' purpose: Examination of the hypothesis that simultaneous use of thrombin generation assay (TGA) and thromboelastometry (TEM) may facilitate the choice of optimal therapy with by-passing agents and laboratory monitoring of efficacy of those agents in patients with acquired haemophilia or haemophilia A with inhibitor.
Detailed Description
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Conditions
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Keywords
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Study Design
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COHORT
PROSPECTIVE
Study Groups
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haemophilia
Patients with acquired haemophilia. Patients with haemophilia A with inhibitor.
No interventions assigned to this group
Eligibility Criteria
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Inclusion Criteria
* patients with congenital haemophilia A with inhibitor
18 Years
ALL
No
Sponsors
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Stowarzyszenie Pomocy Chorym na Zakrzepicę i Skazy Krwotoczne Thrombus
OTHER
Responsible Party
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Principal Investigators
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Krystyna Zawilska, MD, PhD
Role: PRINCIPAL_INVESTIGATOR
Centrum Diagnostyczno - Lecznicze INTERLAB
Maria Podolak-Dawidziak, MD,PhD
Role: PRINCIPAL_INVESTIGATOR
Katedra i Klinika Hematologii, Nowotworow Krwi i Transplantacji Szpiku, Akademia Medyczna
Andrzej Mital, MD, PhD
Role: PRINCIPAL_INVESTIGATOR
Katedra i Klinika Hematologii i Transplantologii Gdanski Uniwersytet Medyczny
Jerzy Windyga, MD, PhD
Role: PRINCIPAL_INVESTIGATOR
Instytut Hematologii i Transfuzjologii w Warszawie
Krzysztof Chojnowski, MD, PhD
Role: PRINCIPAL_INVESTIGATOR
Klinika Hematologii Uniwersytetu Medycznego w Lodzi Wojewodzki Szpital Specjalistyczny im. M. Kopernika
Jacek Musial, MD, PhD
Role: PRINCIPAL_INVESTIGATOR
Szpital Uniwersytecki w Krakowie, Oddzial Kliniczny Alergii i Immunologii
Locations
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Katedra i Klinika Hematologii i Transplantologii Gdanski Uniwersytet Medyczny
Gdansk, , Poland
Szpital Uniwersytecki w Krakowie, Oddzial Kliniczny Alergii i Immunologii
Krakow, , Poland
Klinika Hematologii Uniwersytetu Medycznego w Lodzi Wojewodzki Szpital Specjalistyczny im. M. Kopernika
Lodz, , Poland
Centrum Diagnostyczno - Lecznicze INTERLAB
Poznan, , Poland
Instytut Hematologii i Transfuzjologii w Warszawie
Warsaw, , Poland
Katedra i Klinika Hematologii, Nowotworow Krwi i Transplantacji Szpiku, Akademia Medyczna
Wroclaw, , Poland
Countries
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Central Contacts
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Facility Contacts
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Andrzej Mital, MD, PhD
Role: primary
Jacek Musial, MD, PhD
Role: primary
Krzysztof Chojnowski, MD, PhD
Role: primary
Krystyna Zawilska, MD,PhD
Role: primary
Jerzy Windyga, MD, PhD
Role: primary
Maria Podolak-Dawidziak, MD, PhD
Role: primary
References
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Dargaud Y, Negrier C. Thrombin generation testing in haemophilia comprehensive care centres. Haemophilia. 2010 Mar;16(2):223-30. doi: 10.1111/j.1365-2516.2009.02082.x. Epub 2009 Aug 27.
Berntorp E. Differential response to bypassing agents complicates treatment in patients with haemophilia and inhibitors. Haemophilia. 2009 Jan;15(1):3-10. doi: 10.1111/j.1365-2516.2008.01931.x. Epub 2008 Nov 10.
Dargaud Y, Bordet JC, Lienhart A, Negrier C. Use of the thrombin generation test to evaluate response to treatment with recombinant activated factor VII. Semin Hematol. 2008 Apr;45(2 Suppl 1):S72-3. doi: 10.1053/j.seminhematol.2008.03.008. No abstract available.
Livnat T, Martinowitz U, Zivelin A, Seligsohn U. Effects of factor VIII inhibitor bypassing activity (FEIBA), recombinant factor VIIa or both on thrombin generation in normal and haemophilia A plasma. Haemophilia. 2008 Jul;14(4):782-6. doi: 10.1111/j.1365-2516.2008.01688.x. Epub 2008 Mar 21.
Other Identifiers
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TGA-TEM
Identifier Type: -
Identifier Source: org_study_id