Clinical trial: Efficacy and Safety of IL-11 in DDAVP Unresponsive (NCT00994929)

Eligibility criteria:
Inclusion Criteria:

          -  Males or females >= 18 years of age.

          -  A diagnosis of hemophilia A, moderate (FVIII:C 0.01-0.04 U/ml) or mild (FVIII:C >=
             0.05 U/ml); or a diagnosis of VWD, defined by a low VWF:RCo and past bleeding history.

          -  For those with VWD, an inability to use DDAVP due to i) unresponsiveness defined as
             VWF:RCo or FVIII:C level lower than 50 IU/dl or less than a 3-fold increase after 0.3
             microgram/kg DDAVP; ii) allergic reactions or seizures; or iii) a contraindication to
             DDAVP.

          -  Willingness to have blood drawn.

          -  Willingness to sign informed consent.

        Exclusion Criteria:

          -  Presence of other bleeding disorders, acquired Von Willebrand disease, primary
             thrombocytopenia.

          -  Use of immunomodulatory or experimental drugs, or diuretics.

          -  Pregnant or lactating women.

          -  Previous cardiac disease, congestive failure, arrhythmia (e.g. atrial fibrillation,
             atrial flutter), hypertension, MI, stroke, or thrombosis.

          -  Past allergic reaction to Neumega.

          -  Surgery within the past 8 weeks.

          -  Inability to comply with study protocol requirements.

          -  Concomitant use of antiplatelet drugs, anticoagulants, dextran, aspirin or NSAIDs.

          -  Treatment with DDAVP, cryoprecipitate, whole blood, plasma and plasma derivatives
             containing substantial quantities of FVIII and/or VWF within five days of study.

          -  Baseline safety and/or hematology lab values outside the normal limits and/or an EKG
             indicating an arrhythmia.
No condition on gender to be admitted to the trial.
No healthy subjects accepted to join the trial.
Subject must be at least 18 Years 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