Haemophilia A
Conditions
Brief summary
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
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.
Interventions
None listed
Sponsors
Octapharma
Study design
Observational model
COHORT
Time perspective
OTHER
Eligibility
Sex/Gender
ALL
Healthy volunteers
No
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
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Annualised rate of breakthrough bleeds to assess efficacy in prophylactic treatment | 100 exposure days | Annualised rate of all bleeding events (BEs), including all spontaneous, traumatic and joint BEs |
| Incidence of Adverse Drug Reactions (ADRs) | 100 exposure days | 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. |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| Dosage of FVIII concentrates | 100 exposure days | For each individual FVIII injection the dose will be recorded. |
| Overall assessment of the effectiveness of surgical prophylaxis by the treating physicians | 100 exposure days | 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'. |
Countries
Argentina, Azerbaijan, Belarus, Belgium, Canada, Croatia, Estonia, France, Germany, Hungary, Italy, Lithuania, Mexico, Russia, Spain, Turkey (Türkiye), United Kingdom, United States
Contacts
CONTACTCristina Solomon, MD
STUDY_DIRECTORSigurd Knaub, PhD
Octapharma
Outcome results
None listed