Brief Title
Global Haemostatic Methods Following Administration of Bypassing Agents to Patients With Haemophilia With Inhibitors
Official Title
Global Haemostatic Methods to Measure the Treatment Effect Following Administration of Bypassing Agents to Patients With Haemophilia With Inhibitors
Brief Summary
Background
The treatment of haemophilia A and B has been revolutionized by the use of factor
concentrate, both as prophylaxis and to treat bleeding episodes (on-demand treatment).
However, despite its advantages, repeated treatment with factor concentrate can lead to
development of inhibitors (antibodies) towards the coagulation factor in the concentrate.
Another patient group in which the bleeding symptoms are difficult to treat because of
inhibitors towards coagulation factors, most commonly FVIII, is patients with acquired
haemophilia. Patients with high antibody titers exhibit a deficient or no response to factor
concentrates and usually need treatment with bypassing agents, namely factor eight inhibitor
bypassing agent (FEIBA®, Baxter) och recombinant activated factor VII (rFVIIa, Novo-Seven®,
Novo Nordisk). The effect of the treatment cannot be accurately monitored by traditional
coagulation tests.
The aim of the study is to evaluate the utility of the global haemostatic methods in patients
with haemophilia with inhibitors. The objective is to improve the monitoring of the treatment
effect and thus increase the safety of the patient and the effectiveness of the treatment.
Patients and methods
Patients
The primary cohort will consist of fifteen patients with inherited haemophilia with
inhibitors as well as five adult patients with acquired haemophilia who are followed up at
the Coagulation Department of the Karolinska University Hospital, Stockholm, Sweden.
Blood samples will be collected from those patients at specific time points (see Design of
the study) during the course of two years (for each patient). The treatment (type, dose,
duration) will be determined by the treating physician.
Methods (selection)
- Thrombin generation (Calibrated Automated Thrombogram, CAT® and a commercial kit from
Siemens®).
- Overall haemostatic potential (OHP)
Design of the study
Timeframe for blood sampling: i) baseline (inclusion in the study), and ii) prior and after
administration of bypassing agents to either treat bleeding symptoms or before an invasive
procedure or as prophylaxis.
Data analysis
The variations in coagulation markers measured as described above (Methods) will be
associated to the clinical symptoms (bleeding), the level of coagulation factors (if
measurable) and the titers of the inhibitors.
Study Type
Observational [Patient Registry]
Primary Outcome
Changes in coagulation markers, such as thrombin generation markers (Endogenous Thrombin Potential in nano molar thrombin*minute and peak thrombin in nano molar and fibrin aggregation curves, following administration of bypassing agents.) composite
Condition
Hemophilia
Publications
* Includes publications given by the data provider as well as publications identified by National Clinical Trials Identifier (NCT ID) in Medline.
Recruitment Information
Estimated Enrollment
20
Start Date
March 2015
Completion Date
August 2025
Primary Completion Date
June 2025
Eligibility Criteria
Inclusion Criteria:
- informed consent
- meets the study population description
Exclusion Criteria:
- no informed consent age<7 years
Gender
Male
Ages
7 Years - N/A
Accepts Healthy Volunteers
No
Contacts
Roza Chaireti, MD, PhD, +46 738 5170974, [email protected]
Location Countries
Sweden
Location Countries
Sweden
Administrative Informations
NCT ID
NCT02453542
Organization ID
202100-2973
Responsible Party
Principal Investigator
Study Sponsor
Karolinska Institutet
Study Sponsor
Roza Chaireti, MD, PhD, Principal Investigator, Karolinska Institutet
Verification Date
March 2022