Response to DDAVP In mild Hemophilia A patients, in Search for dEterminants

Completed

Conditions: bleeding disorder
hemophilia A
10064477

Registration Number: NL-OMON43824

Lead Sponsor: Academisch Medisch Centrum

Brief Summary: Not available

Detailed Description: Not available

Recruitment & Eligibility

Status: Completed

Sex: Not specified

Target Recruitment: 281

Inclusion Criteria

Moderate/mild hemophilia: defined as a FVIII deficiency with a FVIII:C plasma concentration of 2-40 IU/DL.
Treated or tested with DDAVP (between 1980 and 2013), and documented FVIII and VWF plasma levels prior to DDAVP administration and at least for 1 time point after DDAVP administration.

Exclusion Criteria

Severe hemophilia A patients (factor VIII <1 IU/DL ) will be excluded from the study population.
The patient has other bleeding disorders, especially von Willebrand Disease (documented vWF plasma levels are available)
Type 2N von Willebrand Disease, or highly likely that patient has this disease since there are female bleeding patients in the pedigree
First DDAVP test is not taken at current hemophilia treatment center

Study & Design

Study Type: Observational invasive

Study Design: Not specified

Primary Outcome Measures

Name	Time	Method
<p>The main study parameters are:<br /><br>- Clinical factors that determine DDAVP response<br /><br>- Genetic factors that determine DDAVP response<br /><br>The main study endpoint is the response to DDAVP, which is defined as complete<br /><br>(1h after DDVP FVIII: C >0.50 IE/ml), partial (1h after DDVP FVIII: C 0.30-0.50<br /><br>IE/ml), or none (1h after DDVP FVIII: C < 0.30 IE/ml). Therapeutic response<br /><br>will also be evaluated. </p><br>

Secondary Outcome Measures