von Willebrand disease type III. Angiohaemophilia, Factor VIII deficiency with vascular defect, Vascular haemophilia
Conditions
Interventions
Intervention 1: Wilate® (Octapharma), 500 IU vial (500 international units of FVIII, 500 international units of vWF:RCo), 35 international units of vWF:RCo per kilogram of patient's weight per dose is
Treatment - Drugs
Wilate® (Octapharma), 500 IU vial (500 international units of FVIII, 500 international units of vWF:RCo), 35 international units of vWF:RCo per kilogram of patient's weight per dose is administered. T
Humate-P® (CSL Behring), 500 IU vial (500 international units of FVIII, 500 international units of vWF:RCo), 35 international units of vWF:RCo per kilogram of patient's weight per dose is administered
Sponsors
Pourateb Company
Eligibility
Sex/Gender
All
Inclusion criteria
Inclusion criteria: Inclusion criteria: patients with definitive diagnosis of vWD type III based on their plasma level of vWF:RCo and vWF:Ag; regardless of their age, sex, and previous treatments; who need on-demand treatment with vWF/FVIII concentrates; patients with spontaneous hemorrhages* from nose, mouth, teeth, menorrhagia, and hemarthrosis; patients with stable hemodynamic condition and without need for packed red blood cells to treat bleeding episodes. (only mild and moderate bleedings); who has given their verbal and written consent to take part in the study. Exclusion criteria: women of reproductive age with clinical history or signs or laboratory evidence of pregnancy; breastfeeding women; patients with current or past history of positive inhibitor activity against vWF or factor VIII; patients with a history of hypersensitivity to or intolerance of blood or plasma-derived products; patients with severe diseases like liver or kidney disease; patients who have recieved a vWF/FVIII concentrate during the past seven days (no matter they had been included in the study or not); patients with gastrointestinal hemorrhages; patients with severe bleeding episodes, which make the patient hemodynamically unstable or require urgent transfusion with packed red blood cells; patients who need vWF/FVIII concentrates for the prophylaxis of bleeding or a surgical operation.
Exclusion criteria
Exclusion criteria:
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Qualitative (subjective) effectiveness in bleeding control. Timepoint: After intervention. Method of measurement: Four-point effectiveness scale (Excellent, Good, Intermediate, Poor) scored by patient.;Quantitative (objective) effectiveness in bleeding control. Timepoint: After intervention. Method of measurement: It is scored by physician based on a quantitative scale (0, +1, +2, +3, +4), which is designed according to the different types of bleeding and their own criteria of response to therapy.;Cost-effectiveness in bleeding control. Timepoint: After intervention. Method of measurement: Calculation of incremental cost effectiveness ratio (ICER). | — |
Secondary
| Measure | Time frame |
|---|---|
| Tolerability. Timepoint: On patient's discharge. Method of measurement: 3-point Verbal Rating Scale (Excellent, Satisfactory, Undesirable), by patient (subjectively).;Adverse effect. Timepoint: After intervention to the time of discharge, two weeks after intervention, whenever an adverse event occurs between these two times, or if not possible, the next time when the patient comes for follow-up (two weeks after intervention). Method of measurement: Clinician's diagnosis and determination of its relationship to the concentrate (probably related, possibly related, probably not related, and not related).;Serious adverse effect. Timepoint: After intervention to the time of discharge, two weeks after intervention, whenever an adverse event occurs between these two times, or if not possible, the next time when the patient comes for follow-up (two weeks after intervention). Method of measurement: Clinician's diagnosis and determination of its relationship to the concentrate (probably related, possibly related, probably not related, and not related). | — |
Countries
Iran (Islamic Republic of)
Contacts
Public ContactDr Gholamreza Toogeh
Tehran University of Medical Sciences
Outcome results
None listed