Global Hemostatic Methods in Hemophilia and Von Willebrand's Disease

NCT02061033 | Unknown

• 1 other identifier: GHMJA2014
• Study Type: observational
• Target: 180
• Locations: 1 country
• Sites: 1

Brief Summary

Patients with hemophilia who have the same level of deficient factor(s) may express different severity of clinical presentation and bleeding tendency. Therefore a test which could determine overall hemostasis rather than simple concentration of a single deficient factor may correlate better with clinical phenotype in these patients. The investigators will therefore study the usefulness of global hemostatic methods (endogenous thrombin potential (ETP), overall hemostatic potential (OHP), fibrin clot structure) and microparticles in the prediction of severity of bleeding and estimation of response to the treatment in patients with hemophilia. Since hemophilia patients on prophylactic treatment virtually do not bleed, additional patients who are treated on demand only will be included enabling to study possible modulatory effects of different hemostatic factors (particularly prothrombotic and thrombin activatable fibrinolysis inhibitor (TAFI)) on clinical presentation. The investigators will correlate both those factors and clinical severity with global hemostatic methods. The investigators expect to prove that individual tailoring of the treatment, which may enable lowering the prophylactic dose of factor concentrate without increasing the risk of bleeding, is justified in some hemophilia patients. This approach would reduce the amount of necessary factor concentrate in certain patients and decrease the cost (which represents extensive burden for health care systems) of treatment without potential risk for the patients.

Conditions

Hemophilia A; Hemophilia B; Von Willebrand's Disease

Keywords

Hemophilia A; Hemophilia B; Von Willebrand's Disease; Endogen thrombin potential; Overall hemostatic potential; Microparticles; Fibrin clot

Outcome Measures

Primary Outcomes (1)

• Number of microparticles

  5 years

Eligibility Criteria

• Age: 10 Years+
• Sex: all
• Healthy Volunteers: Yes
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)
• Sampling Method: Non-Probability Sample

Study Population

100 patients with moderate and severe hA, 30 patients with moderate and severe hB and 50 patients with VWD (primarily severe type I and type III) from hemophilia centers Stockholm, Sweden and Belgrade, Serbia.

You may qualify if:

• patients with bleeding disorders

You may not qualify if:

• none

Sponsors & Collaborators

• Karolinska University Hospital: lead
• The Swedish Society of Medicine: collaborator
• Karolinska Institutet: collaborator

Study Sites (1)

Karolinska University Hospital

Stockholm, Sweden

RECRUITING

Related Publications (2)

• Antovic JP, Mikovic D, Elezovic I, Holmstrom M, Wilkens M, Elfvinge P, Mahmoud Hourani Soutari N, Antovic A. Two global haemostatic assays as additional tools to monitor treatment in cases of haemophilia A. Thromb Haemost. 2012 Jul;108(1):21-31. doi: 10.1160/TH11-11-0811. Epub 2012 Apr 26.

  PMID: 22534727 RESULT

• Mobarrez F, Mikovic D, Antovic A, Antovic JP. Is a decrease of microparticles related to improvement of hemostasis after FVIII injection in hemophilia A patients treated on demand? J Thromb Haemost. 2013 Apr;11(4):697-703. doi: 10.1111/jth.12103.

  PMID: 23231463 RESULT

Biospecimen

Retention: SAMPLES WITH DNA

Citrated plasma and whole blood DNA samples

MeSH Terms

Conditions

Hemophilia A; Hemophilia B; von Willebrand Diseases

Condition Hierarchy (Ancestors)

Blood Coagulation Disorders, Inherited; Blood Coagulation Disorders; Hematologic Diseases; Hemic and Lymphatic Diseases; Coagulation Protein Disorders; Hemorrhagic Disorders; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Genetic Diseases, X-Linked; Blood Platelet Disorders

Central Study Contacts

Jovan P Antovic, MD, PhD

CONTACT

+46 734 294447; Jovan.Antovic@ki.se

Study Design

• Study Type: observational
• Observational Model: CASE CONTROL
• Time Perspective: CROSS SECTIONAL
• Sponsor Type: OTHER
• Responsible Party: PRINCIPAL INVESTIGATOR
• PI Title: Associate professor, Consultant

Study Record Dates

• First Submitted: February 7, 2014
• First Posted: February 12, 2014
• Study Start: March 1, 2013
• Primary Completion: December 1, 2017
• Study Completion: December 1, 2018
• Last Updated: August 31, 2016

Record last verified: 2016-08

Locations

SE (1)