Thrombus: Use of a TGA and TEM in the Assessment of the Efficacy of Treatment With APCC or rFVIIa

Sponsor

Stowarzyszenie Pomocy Chorym na Zakrzepicę i Skazy Krwotoczne Thrombus (Other)

Overall Status

Unknown status

CT.gov ID

NCT01856751

Collaborator

(none)

Enrollment

Locations

Duration (Months)

13.3

Patients Per Site

0.5

Patients Per Site Per Month

Study Details

Study Description

Brief Summary

Occurrence of inhibitors to coagulation factor VIII is diagnosed in ~30% patients with haemophilia A. Presence of inhibitor with a titre >5 BU/ml requires the use of by-passing agents: recombinant activated Factor VIIa concentrate (rFVIIa) and/or activated prothrombin complex concentrate (APCC). Similarly, haemorrhagic complications in patients with acquired haemophilia and inhibitor titre >5 BU/ml should be treated with by-passing agents.

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.

Condition or Disease	Intervention/Treatment	Phase
Haemophilia

Detailed Description

This section is Not applicable

Study Design

Study Type:

Observational

Anticipated Enrollment :

80 participants

Observational Model:

Cohort

Time Perspective:

Prospective

Official Title:

Use of a TGA and TEM in the Assessment of the Efficacy of Treatment With APCC or rFVIIa Concentrate in Patients With Acquired Haemophilia and in Patients With Haemophilia A With Inhibitors

Study Start Date :

Apr 1, 2014

Anticipated Primary Completion Date :

Apr 1, 2016

Anticipated Study Completion Date :

Jun 1, 2016

Arms and Interventions

Arm	Intervention/Treatment
haemophilia Patients with acquired haemophilia. Patients with haemophilia A with inhibitor.

Outcome Measures

Primary Outcome Measures

Assessment of patient's individual response to therapy with by-passing agents by simultaneous use of TGA and TEM methods. [48 hours]
This is non-inverventional study as the protocol will not assign specific treatment to the particular subjects of the study. Patients will be treated with APCC or rFVIIa based on the experience of the study site. Patients are prescribed a treatment according to their physician's judgement or local clinical practice. This is observation of the everyday clinical practise on site.

Eligibility Criteria

Criteria

Ages Eligible for Study:

18 Years and Older

Sexes Eligible for Study:

All

Accepts Healthy Volunteers:

Inclusion Criteria:

patients with acquired haemophilia
patients with congenital haemophilia A with inhibitor

Exclusion Criteria:

Contacts and Locations

Locations

	Site	City	Country	Postal Code
1	Katedra i Klinika Hematologii i Transplantologii Gdanski Uniwersytet Medyczny	Gdansk	Poland	80-952
2	Szpital Uniwersytecki w Krakowie, Oddzial Kliniczny Alergii i Immunologii	Krakow	Poland	31-501
3	Klinika Hematologii Uniwersytetu Medycznego w Lodzi Wojewodzki Szpital Specjalistyczny im. M. Kopernika	Lodz	Poland	93-510
4	Centrum Diagnostyczno - Lecznicze INTERLAB	Poznan	Poland	61-505
5	Instytut Hematologii i Transfuzjologii w Warszawie	Warszawa	Poland	02-776
6	Katedra i Klinika Hematologii, Nowotworow Krwi i Transplantacji Szpiku, Akademia Medyczna	Wroclaw	Poland	50-367

Sponsors and Collaborators

Stowarzyszenie Pomocy Chorym na Zakrzepicę i Skazy Krwotoczne Thrombus

Investigators

Principal Investigator: Krystyna Zawilska, MD, PhD, Centrum Diagnostyczno - Lecznicze INTERLAB
Principal Investigator: Maria Podolak-Dawidziak, MD,PhD, Katedra i Klinika Hematologii, Nowotworow Krwi i Transplantacji Szpiku, Akademia Medyczna
Principal Investigator: Andrzej Mital, MD, PhD, Katedra i Klinika Hematologii i Transplantologii Gdanski Uniwersytet Medyczny
Principal Investigator: Jerzy Windyga, MD, PhD, Instytut Hematologii i Transfuzjologii w Warszawie
Principal Investigator: Krzysztof Chojnowski, MD, PhD, Klinika Hematologii Uniwersytetu Medycznego w Lodzi Wojewodzki Szpital Specjalistyczny im. M. Kopernika
Principal Investigator: Jacek Musial, MD, PhD, Szpital Uniwersytecki w Krakowie, Oddzial Kliniczny Alergii i Immunologii