Clinical trial: Bleeding Incidence in VWD Patients Undergoing On-Demand Treatment (NCT04053699)

Eligibility criteria:
Inclusion Criteria:

        Patients who meet all of the following criteria are eligible for the study:

          -  Male or female patients aged ≥5.5 years at the time of enrolment

          -  VWD type 1 (baseline von Willebrand factor activity [VWF:RCo], <30 IU/dL), 2A, 2B, 2M,
             or 3 according to medical history requiring substitution therapy with a VWF-containing
             product to control bleeding

          -  Currently receiving frequent on-demand treatment with a VWF-containing product

          -  In female patients of child-bearing potential using hormonal contraception, the
             medication class should remain unchanged for the duration of their study participation

          -  Voluntarily given, fully informed written and signed consent obtained before
             collection of any patient data

        Exclusion Criteria:

        Patients who meet any of the following criteria are not eligible for the study:

          -  Patients currently on prophylaxis for VWD (except for perioperative prophylaxis) as
             well as patients having received treatment once a month for menstrual bleeding, but
             not for any other bleeds

          -  Patients whose VWD treatment is planned to be switched from on-demand to prophylactic
             treatment in the next 6 months

          -  History, or current suspicion, of VWF or FVIII inhibitors

          -  Medical history of a thromboembolic event within 6 months before enrolment

          -  Severe liver or kidney diseases as described in the medical records

          -  Female patients with an existing or suspected pregnancy or who are breast-feeding at
             the time of enrolment

          -  Change in hormonal contraception within 6 months before enrolment

          -  Cervical or uterine conditions causing abnormal uterine bleeding (including infection
             or dysplasia)

          -  Other coagulation disorders or bleeding disorders due to anatomical reasons

          -  Participation in an interventional clinical study during the 6-month of study period

          -  Inability to complete the patient diary to reliably evaluate the type, frequency, and
             treatment of BEs during the 6-month study period
No condition on gender to be admitted to the trial.
No healthy subjects accepted to join the trial.
Subject must be at least 66 Months old.


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? Entailment