Clinical trial: The Von Willebrand Disease (VWD) International Prophylaxis Study (NCT00557908)

Eligibility criteria:
Inclusion Criteria:

        Type 1: eligible for participation if

          -  ≤20% RCo and/or ≤20% FVIII; and

          -  DDAVP non-responsive, defined as occurrence of bleeding episodes not responding
             satisfactorily to desmopressin, or deemed non-responsive a priori by the investigator;
             and

          -  Bleeding indication criteria are met

        Type 2: eligible for participation if

          -  DDAVP non-responsive, defined as occurrence of bleeding episodes not responding
             satisfactorily to desmopressin, or deemed non-responsive a priori by the investigator;
             or Type 2B;

          -  Bleeding indication criteria are met

        Type 3: eligible for participation if

          -  Bleeding indication criteria are met

        Bleeding Indication Criteria:

          -  Joint Bleeding: documentation of at least two apparently spontaneous bleeding episodes
             in the same joint in the six months prior to enrollment; or three or more apparently
             spontaneous bleeding episodes in different joints in the six months prior to
             enrollment.

          -  GI Bleeding: history of two or more severe GI bleeding episodes associated with either
             a drop in hemoglobin of ≥ 2 g/dl or requiring red blood cell transfusion or treatment
             with VWD concentrate.

          -  Failure to identify other causes of bleeding.

          -  Menorrhagia: a diagnosis of menorrhagia; prospectively completed Pictorial Blood
             Assessment Chart score >185 or required treatment with a VWD product for menstrual
             bleeding on one or more occasions in the year prior to enrollment.

          -  Normal cervical cytology (PAP) within the six months prior to enrollment for females ≥
             18 years of age.

          -  Epistaxis 1. Three or more bleeding episodes in a six-month period that required
             treatment with VWD concentrates or red cell transfusions.
No condition on gender to be admitted to the trial.
No healthy subjects accepted to join the trial.



Patient description (medical language): A 23-year-old female has prolonged oral bleeding immediately after a tooth extraction.  Despite several interventions, the bleeding persists for hours and stops only after desmopressin (DDAVP) administration.  The patient has heavy menstrual cycles each month.  She has no other medical problems and takes no medications.  Her mother and grandmother have also had excessive bleeding during menstrual period.  Review of systems is positive for mild bruising on his legs. Laboratory findings reveal a normal platelet count and an abnormal ristocetin cofactor assay, as well as CB <= 0.30 IU/mL and FVIII:C <= 0.40 IU/mL.

Patient description (patient's own words): I'm a 23 woman and I went to the ER because of non-stop bleeding after my tooth extraction. I tried to make the bleeding stop but it just kept going for hours and it finally stopped when I took some medication called DDAVP. I usually have heavy periods, just like my mom and grandma had. I don't have any other medical condition and I don't take any other medication. The doctor noticed the bruises on my legs. I had to do some lab tests. I had a normal platelet count but an abnormal ristocetin cofactor assay. I had low CB and FVIII:C levels.

Does the patient match the trial criteria? 1