Efficacy, Safety & Utilisation of Nuwiq, Octanate and Wilate in Previously Untreated & Minimally Treated Haemophilia A Patients

Study Overview
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Brief Summary
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International, post-authorisation non-interventional study to evaluate real-life effectiveness, safety and utilisation patterns of Octapharma's FVIII concentrates Nuwiq, Octanate, and Wilate in previously untreated and minimally treated severe haemophilia A patients in routine clinical practice.

Detailed Description
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Octapharma's FVIII concentrates have been tested in clinical trials and registered for treatment of haemophilia A; however, as haemophilia A is a rare disease, the numbers of patients treated in studies so far are limited. For previously untreated patients (PUPs), who are typically young children, and for minimally treated patients (MTPs), who have been exposed to only minimal FVIII dosages, there is a general interest to increase the body of data on treatment effectiveness and safety, particularly related to inhibitor development. Also, specifically for PUPs, treatment algorithms are not standardized, e.g. with respect to utilisation, dosage, frequency or optimal start age of FVIII prophylaxis. Real world evidence derived from a non-interventional study (NIS) can describe product utilisation and demonstrate value over a product's life cycle and facilitate benefit-risk assessments. The purpose of this study is thus to evaluate product utilisation, effectiveness and safety, including inhibitor development information, in severe haemophilia A PUPs and MTPs, who have been prescribed Octapharma's FVIII concentrates.

Official Title
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Practical Utilisation of Octapharma FVIII Concentrates in Previously Untreated & Minimally Treated Haemophilia A Patients Entering Routine Clinical Treatment With Nuwiq, Octanate or Wilate - Efficacy & Safety Observational Study-Protect-NOW

Conditions
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Haemophilia A

Participation Criteria
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Eligibility Criteria
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Inclusion Criteria: Male and female patients of any age and ethnicity Severe haemophilia A (FVIII:C<1%) Decision to prescribe Octapharma's FVIII concentrate before enrollment into the study Either No previous treatment with FVIII concentrates or other blood products containing FVIII (PUPs) OR Less than 5 Exposure Days (EDs) to FVIII concentrates or other blood products containing FVIII (MTPs), if data are available on all previous treatment, AND they did not develop an inhibitor at any time point, OR they developed an inhibitor during treatment with an Octapharma FVIII concentrate AND continue treatment with THIS Octapharma FVIII concentrate (in the presence or absence of emicizumab). Voluntarily given, fully informed written and signed consent obtained before any study-related data documentation is conducted (obtained from the patient's parent/legal guardian) Exclusion Criteria: Diagnosis with a coagulation disorder other than haemophilia A Concomitant treatment with any systemic immunosuppressive drug Participation in an interventional clinical trial during the time period evaluated Participation in another non-interventional study of Octapharma
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 |
| --- | --- |
| Nuwiq<br>All patients receiving Nuwiq (recombinant FVIII) | |
| Octanate<br>All patients receiving Octanate (plasma derived FVIII) | |
| Wilate<br>All patients receiving Wilate (plasma derived FVIII/von Willebrand factor [VWF]) | |

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Annualised rate of breakthrough bleeds to assess efficacy in prophylactic treatment | Annualised rate of all bleeding events (BEs), including all spontaneous, traumatic and joint BEs | 100 exposure days | 
| Incidence of Adverse Drug Reactions (ADRs) | Adverse drug reactions (ADRs) including hypersensitivity reactions will be recorded in by patients in treatment diaries, which will be reviewed at each Follow-up Visit. | 100 exposure days | 

Secondary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Dosage of FVIII concentrates | For each individual FVIII injection the dose will be recorded. | 100 exposure days | 
| Overall assessment of the effectiveness of surgical prophylaxis by the treating physicians | At the end of the postoperative period, treating physicians will assess the effectiveness of surgical prophylaxis using a scale including the four items: 'excellent,' 'good,' moderate,' and 'none'. | 100 exposure days |