Platelet Function in Patients With Hemophilia A
| Status: | Enrolling by invitation |
|---|---|
| Conditions: | Anemia, Hematology |
| Therapuetic Areas: | Hematology |
| Healthy: | No |
| Age Range: | 2 - 18 |
| Updated: | 10/19/2018 |
| Start Date: | March 2015 |
| End Date: | June 2019 |
Decreased Platelet Function as a Cause of Increased Bleeding in Patients With Hemophilia A
Abnormalities in the gene encoding Factor VIII (FVIII) results in hemophilia A, an X-linked
recessive bleeding disorder with a prevalence of 1 in 5000 males. Hemophilia A patients are
classified into 3 different categories based on residual FVIII activity compared to normal:
mild (6-40%), moderate (1-5%) and severe (<1%). This categorization correlates to some degree
with bleeding phenotype, but does not completely define it. Some patients with hemophilia A
bleed less often than others despite identical plasma FVIII levels. The cause(s) of this
phenotype heterogeneity in hemophilia A remains largely unknown, despite a number of studies
of possible factors.
Activated platelets, in addition to their role in primary hemostasis, play a major role in
secondary hemostasis (coagulation) by providing a phospholipid surface to which coagulation
factors bind. A role for platelets in the hemorrhagic propensity of hemophilia A has been
suggested in the past, but only a small number of studies have been performed with
limitations in assays performed and numbers of patients. The purpose of the present study is
to determine whether platelet reactivity in severe hemophilia A patients is associated with
past bleeding frequency and/or predicts future bleeding frequency.
recessive bleeding disorder with a prevalence of 1 in 5000 males. Hemophilia A patients are
classified into 3 different categories based on residual FVIII activity compared to normal:
mild (6-40%), moderate (1-5%) and severe (<1%). This categorization correlates to some degree
with bleeding phenotype, but does not completely define it. Some patients with hemophilia A
bleed less often than others despite identical plasma FVIII levels. The cause(s) of this
phenotype heterogeneity in hemophilia A remains largely unknown, despite a number of studies
of possible factors.
Activated platelets, in addition to their role in primary hemostasis, play a major role in
secondary hemostasis (coagulation) by providing a phospholipid surface to which coagulation
factors bind. A role for platelets in the hemorrhagic propensity of hemophilia A has been
suggested in the past, but only a small number of studies have been performed with
limitations in assays performed and numbers of patients. The purpose of the present study is
to determine whether platelet reactivity in severe hemophilia A patients is associated with
past bleeding frequency and/or predicts future bleeding frequency.
Inclusion Criteria:
- Patients with severe hemophilia A who are being prophylactically treated with FVIII.
- Age of at least 2 years.
- Bleeding history of at least 6 months.
- IRB-approved informed consent.
Exclusion Criteria:
- Presence of FVIII inhibitors.
- Greater than 7 days since active bleeding.
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