bleeding disorder hemophilia A
Conditions
Interventions
DDAVP will be administrated intravenously (0.3 *g/kg). Six serial blood samples
will be taken before and at 5 time points after administration.
Sponsors
Academisch Medisch Centrum
Eligibility
Age
18 Years to 99 Years
Inclusion criteria
Inclusion criteria: * Mild hemophilia A: defined as a FVIII:C plasma concentration of 0.06-0.40 IU/ml; * Administration of DDAVP for a test (intraveneous administration with 0.3 ug/ kg) between 2000 and 2015 with documented FVIII and von Willebrand plasma levels prior to and FVIII levels after DDAVP administration; * Administration of DDAVP in past was performed in AMC; * Known F8 gene mutation, preferably limited to two large mutation groups; * >19 and <70 years of age.
Exclusion criteria
Exclusion criteria: * Severe or moderate hemophilia A patients (factor VIII *0.05 IU/ml); * Von Willebrand Factor plasma levels are unknown (von Willebrand disease (VWD) cannot be excluded); * Type 2 Normandy (2N) VWD is identified, or patient is highly likely to have type 2N VWD since there are female patients with bleeding symptoms in the pedigree; * Other bleeding disorders; * Presence of antibodies for FVIII (an inhibitor) at the time of DDAVP administration; * F8 genotype that never showed a >2 fold increase in VWF levels upon DDAVP administration; * Contraindication DDAVP use: contusio cerebri, cardiovascular disease (history, multiple cardiovascular risk factors and age > 70 years), electrolyte deficiencies, hematuria, von Willebrand disease type 2b, TTP, reduced kidney function, kidney hemorrhage.
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| The primary outcome parameter is the time profile of FVIII activity and antigen concentration and VWF antigen concentration in blood in a 24 hour period following DDAVP administration | — |
Countries
Netherlands
Outcome results
None listed