Effectiveness and Tolerability of Eqwilate in Real-life Conditions

Study Overview
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Brief Summary
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MOdalities of use, effectiveNEss and TOlerability of Eqwilate® a balanced combInatiON of VWF and FVIII in von WillEbrand patients in real-life conditions: the ONE-TO-ONE study

Official Title
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MOdalities of Use, effectiveNEss and TOlerability of Eqwilate® a Balanced combInatiON of VWF and FVIII in Von WillEbrand Patients in Real-life Conditions: the ONE-TO-ONE Study

Conditions
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VWD - Von Willebrand's Disease

Intervention / Treatment
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* Biological: Eqwilate


Participation Criteria
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Eligibility Criteria
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Inclusion Criteria: The study will involve patients (≥ 6 years of age) from the participating centres treated with Eqwilate® (either on-demand, as perioperative prophylaxis, or as prophylaxis) during the time inclusion period will be opened. Patients will be of any VWD type, previously treated or untreated. Exclusion Criteria: N/A

Ages Eligible for Study
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Minimum Age: 6 Years

Sexes Eligible for Study
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All

Study Plan
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How is the study designed?
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Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Eqwilate<br> | Biological: Eqwilate<br>* Eqwilate® is a new generation, albumin-free, high-purity, double virus-inactivated, freezedried concentrate of VWF and FVIII<br>|

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Haemostatic Effectiveness of On-demand Treatment | Haemostatic Effectiveness of On-demand Treatment (excellent, good, moderate, none) none: severe uncontrolled bleeding or intensity of bleeding not changed (in case of non-severe bleeding episodes); moderate: moderate bleeding, or control of bleeding required additional product; good: slight oozing and adequate control of bleeding episode and did not require additional product; excellent: haemostasis achieved, cessation of bleeding episode. | 12 months | 
| Haemostatic Effectiveness of Perioperative Prophylaxis | Haemostatic Effectiveness of Perioperative Prophylaxis (excellent, good, moderate, none) none: severe uncontrolled bleeding or intensity of bleeding not changed (in case of non-severe bleeding episodes); moderate: moderate bleeding, or control of bleeding required additional product; good: slight oozing and adequate control of bleeding episode and did not require additional product; excellent: haemostasis achieved, cessation of bleeding episode. | 12 months | 
| Occurrence of Bleeding Episodes During Follow-Up | Occurrence of Bleeding Episodes During Follow-Up | 12 months | 

Secondary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| FVIII Levels | Clotting factor VII levels in blood | 12 months | 
| Productivity Loss | Days lost from school or work | 12 months | 
| Volume of Blood loss | Volume of Blood loss | up to 1 week | 
| Immunogenicity Presence of inhibitors against VWF | Presence of inhibitors against VWF found via blood draw analysis | 12 months |