Boston Children's Hospital
Alan D. Michelson
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.
Hemophilia A
| Study Type : | OBSERVATIONAL |
| Estimated Enrollment : | 36 participants |
| Official Title : | Decreased Platelet Function as a Cause of Increased Bleeding in Patients With Hemophilia A |
| Actual Study Start Date : | 2015-03 |
| Estimated Primary Completion Date : | 2018-06 |
| Estimated Study Completion Date : | 2020-02-01 |
Information not available for Arms and Intervention/treatment
| Ages Eligible for Study: | 2 Years to 18 Years |
| Sexes Eligible for Study: | MALE |
| Accepts Healthy Volunteers: |
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Not yet recruiting
Boston Children's Hospital, Boston Hemophilia Center
Boston, Massachusetts, United States, 02115