Surveillance of Safety and Efficacy of Wilate in Patients With Von Willebrand Disease

Study Overview
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Brief Summary
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This is an observational study, hence there is no study hypothesis

Official Title
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Surveillance of Safety and Efficacy of Wilate in Patients With Von Willebrand Disease

Conditions
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Von Willebrand Disease

Intervention / Treatment
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* Other: Patients using Wilate as standard of care


Participation Criteria
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Eligibility Criteria
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Inclusion Criteria: Patients with a diagnosis of von Willebrand Disease who have been prescribed Wilate
Sexes Eligible for Study
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All

Accepts Healthy Volunteers
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No

Study Plan
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How is the study designed?
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Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Patients using Wilate as standard of care treatment<br>This patient population is being treated with Wilate as standard of care treatment | Other: Patients using Wilate as standard of care<br>* Patients with von Willebrand Disease using Wilate for a period of 2 years.<br>|

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Incidence of Adverse Drug Reactions (ADRs) (%) | Medical Dictionary for Regulatory Activities (MedDRA) primary system organ class preferred term. Incidence rate = number of patients reporting the event / number of patients * 100 | Throughout the duration of each patient's participation in the study (mean [± standard deviation (SD)]: 575 days [±326]; median [range]: 731 days [2-1185]) | 
| Tolerability Assessment of Wilate Infusions by Reason for Administration | Tolerability was assessed using a 3-point verbal rating scale (excellent; satisfactory; unsatisfactory) according to overall feeling during and after Wilate therapy and occurrence of ADRs. Tolerability was assessed for infusions given for on-demand and prophylactic treatment, but not for infusions administered for surgeries or for the purpose of thrombogenicity assessment. In some instances, however, investigators also recorded the tolerability of infusions given for surgical prophylaxis. For infusions administered for surgeries, only those with available tolerability assessments are presented. Wilate infusion may have been administered to a patient for more than one reason and may be included in more than one category (e.g., if a patient was under Wilate prophylaxis, they could also receive Wilate for the treatment of a bleeding episode [BE] or surgery or menstruation). | During and immediately after each infusion of Wilate during the study. | 

Secondary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Patient and Investigator Efficacy Analysis Assessment of the Treatment of Bleeding Episodes (BEs) | Document the efficacy of Wilate in the treatment of acute BEs, breakthrough BEs in patients receiving prophylactic treatment, and menstrual BEs. Efficacy was rated on a 4-point scale (excellent; good; moderate; none) according to overall haemostasis. | During and immediately after treatment of each BE. | 
| Efficacy Analysis for the Prevention of Breakthrough Bleeds During Prophylaxis | Efficacy was rated on a 4-point scale (excellent; good; moderate; none) according to the number of breakthrough bleeds per month. The treatment regimen for prophylactic treatment was at the discretion of the investigator and differed for each patient, as did the duration of prophylactic treatment. The prophylaxis efficacy population (EFF-P) was subdivided into 2 groups, prophylaxis on a continuous basis (EFF-PC population) and prophylaxis on an intermittent basis (EFF-PI population). In total, 25 patients received Wilate for prophylaxis and of these, 17 patients received prophylaxis on a continuous basis, which was defined as: (1) patients having received continuous prophylaxis over a period of at least 3 months, with no treatment gaps longer than 14 days; or (2) patients having received continuous prophylaxis for at least 1 year with an average of 1 infusion/per week (these patients may have had gaps of more than 14 days). | At the end of the study for each patient (study duration: mean [±SD]: 805 days [±247]; median [range]: 797 days [144-1185]). | 
| Efficacy Analysis of Surgical Prophylaxis | Efficacy was rated on a 4-point scale (excellent; good; moderate; none) according to overall haemostasis during and after surgery. | During and immediately after each surgery. | 
| Overall Efficacy Assessment by Patient and Physician at the End of the Treatment Period | Patient and investigator assessment of the overall efficacy of Wilate performed at the end of the study for each patient. Efficacy was rated on a 4-point scale (excellent; good; moderate; none); criteria for assessment were not defined. | At the end of the study for each patient (study duration: mean [±SD]: 596 days [±336]; median [range]: 732 days [2-1185]). |