Population pharmacokinetic modelling: a useful tool to predict response to DDAVP in mild Hemophilia A

Withdrawn

Conditions: bleeding disorder
hemophilia A
10064477

Registration Number: NL-OMON45526

Lead Sponsor: Academisch Medisch Centrum

Brief Summary: Not available

Detailed Description: Not available

Recruitment & Eligibility

Status: Withdrawn

Sex: Not specified

Target Recruitment: 20

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

* 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.

Study & Design

Study Type: Interventional

Study Design: Not specified

Primary Outcome Measures

Name	Time	Method
<p>The primary outcome parameter is the time profile of FVIII activity and antigen<br /><br>concentration and VWF antigen concentration in blood in a 24 hour period<br /><br>following DDAVP administration </p><br>

Secondary Outcome Measures