Severe Hemophilia A With Inhibitor Clinical Trial
Official title:
Thrombin Generation Assay (TGA) as Predictive Test for Haemostatic Effectiveness of Factor VIII (FVIII) Concentrates in Patients Affected by Inherited Haemophilia A With FVIII Inhibitors High and Low Anamnestic Response.
This is an observational, prospective, longitudinal, multicenter, cohort study designed with the scope to verify whether or not TGA may predict effectiveness of different FVIII concentrates class (devoid or rich of VWF) in patient affected by severe or moderately severe inherited haemophilia A and inhibitors.
Rationale:
Hemophilia A is a serious and common hereditary bleeding disorder caused by deficiency of
coagulation factor VIII (FVIII). Patients with this disease are treated with recombinant
factor VIII or factor VIII concentrates derived from plasma.
Administration of exogenous FVIII in 15-35% of cases, cause the formation of antibodies to
FVIII (inhibitors) that neutralize the activity of factor VIII, making the treatment
ineffective.The development of inhibitors of factor VIII (FVIII) is the most serious and
challenging complication of the treatment of hemophilia A and represents the highest
economic burden for a chronic disease. Therefore, research is making great efforts to
optimize the best therapeutic approach for the disease.
It has been observed that FVIII inhibitors display a wide range of immunoreactivity when
tested against different classes of FVIII concentrates (with/without von Willebrand factor
-VWF). It has been demonstrated that the different inhibitors reactivity may correlate with
different ability of inhibitors to impair thrombin generation, as tested by Thrombin
Generation Assay (TGA). In these patients TGA assay might be a tool to predict which FVIII
concentrate has the greater haemostatic effectiveness.
It is also uncertain if the different classes of FVIII used in ITI protocols may have a
different effectiveness in reducing the occurrence of BT bleedings and if this may correlate
to lower reactivity, epitope specificity, VWF content and may be predicted by TGA. It would
be very helpful to be able to give an evidence based diagnostic and prognostic instrument,
the TGA, to aid physician to optimize the therapy for all inhibitors patients.
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Observational Model: Cohort, Time Perspective: Prospective
| Status | Clinical Trial | Phase | |
|---|---|---|---|
| Recruiting |
NCT05802836 -
Dynamics of the Anti-factor VIII Antibody Signature During Treatment With Emicizumab
|