Evaluating Effectiveness and Long Term Safety of Damoctocog Alfa Pegol in Patients, Who Have Been Diagnosed With Hemophilia A

Study Overview
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Brief Summary
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The aim of the HEM-POWR study is to understand better how Damoctocog alfa pegol (Jivi) is used to treat people with Hemophilia A in day-to-day life, how well the treatment is tolerated and how satisfied patients and physicians are with the treatment.

Official Title
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Observational Study Evaluating Effectiveness and Safety of Real-World Treatment With Damoctocog Alfa Pegol in Previously Treated Patients With Hemophilia A

Conditions
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Hemophilia A

Intervention / Treatment
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* Drug: Damoctocog alfa pegol (Jivi, Bay94-9027)


Participation Criteria
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Eligibility Criteria
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Inclusion Criteria: Diagnosis of hemophilia A. Patients previously treated for Hemophilia A. Patients without previous history of inhibitors or patients with previous history of inhibitors on standard prophylaxis therapy for at least 1 year prior to study entry. No current evidence of FVIII inhibitor or clinical suspicion of FVIII inhibitor. Initiation of or currently on damoctocog alfa pegol with any kind of treatment modality (on-demand, prophylaxis, or intermittent prophylaxis). Signed informed consent/assent. Exclusion Criteria: Concurrent participation in an investigational program with interventions outside of routine clinical practice. Diagnosis of any other bleeding/coagulation disorder other than hemophilia A. Contra-indications according to the local marketing authorization. Patient on immune tolerance induction (ITI) treatment at the time of enrollment.
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 |
| --- | --- |
| Previously treated patient (PTPs) with hemophilia A<br>Previously treated patients with hemophilia A receiving damoctocog alfa pegol with any kind of treatment modality (on-demand, prophylaxis, or intermittent prophylaxis)。 | Drug: Damoctocog alfa pegol (Jivi, Bay94-9027)<br>* Follow clinical practice. BAY94-9027 is a B-domain-deleted recombinant factor VIII (rFVIII) product site-specifically conjugated to a single (dual 30-kDa branched) 60-kDa polyethylene glycol (PEG) molecule. BAY 94-9027 is intended for prophylaxis and treatment of bleeds in patients with hemophilia A aged ≥12 years, with a narrow, predictable weekly dose that allows for the treatment regimen to be tailored to individual patient needs.<br>|

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Mean annualized number of reported total bleeds in patients with hemophilia A |  | Up to 36 months | 
| Median annualized number of reported total bleeds in patients with hemophilia A |  | Up to 36 months | 

Secondary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Occurrence of AEs. | Adverse Events (AEs) includes: AEs of special interest, Adverse Event, Serious Adverse Event, Adverse reaction. AEs of special interest includes: hypersensitivity reactions, loss of drug effect, renal impairment, neurocognitive disorders, inhibitor development. | Up to 36 months | 
| Duration of AEs. |  | Up to 36 months | 
| Treatment of AEs. |  | Up to 36 months | 
| Severity of AEs. |  | Up to 36 months | 
| Outcome of AEs. |  | Up to 36 months | 
| Number of infusions and FVIII consumption to achieve hemostasis during surgery |  | Up to 36 months | 
| Change in joint scores (HJHS) | HJHS:Hemophilia Joint Health Score | From baseline to 12 months, 24 months and 36 months | 
| Joint status evaluation by ultrasound (HEAD-US score), if available or part of standard clinical practice. | HEAD-US:Hemophilia Early Arthropathy Detection with Ultrasound | Up to 36 months | 
| Change of number of affected joints by patient |  | From baseline to 12 months, 24 months and 36 months | 
| Annualized number of spontaneous, joint, and trauma bleeds |  | Up to 36 months | 
| Number of reported bleeds during the study compared with number of reported bleeds for previous FVIII products in the 12 months prior to enrollment into the study. | Bleeds includes total, spontaneous, joint, and trauma. | Up to 36 months | 
| Proportion of patients with 0 bleeds, and the difference in proportion comparing to previous prophylaxis treatment. |  | Up to 36 months | 
| AUC for previous FVIII products versus damoctocog alfa pegol. | AUC:Area under the plasma concentration versus time curve | Up to 36 months | 
| Half-life [t½] for previous FVIII products versus damoctocog alfa pegol. |  | Up to 36 months | 
| FVIII trough for previous FVIII products versus damoctocog alfa pegol. |  | Up to 36 months | 
| FVIII peak levels for previous FVIII products versus damoctocog alfa pegol. |  | Up to 36 months | 
| In-vivo recovery for previous FVIII products versus damoctocog alfa pegol. |  | Up to 36 months | 
| Number of infusions to control for a bleed |  | Up to 36 months | 
| Changes of Hemo-SAT A score | Hemo-SAT A:Hemophilia Treatment Satisfaction Questionnaire for adults。The Hemo-SAT questionnaire version for adults (Hemo-SAT A) consists of 34 items pertaining to 6 dimensions (Ease & Convenience, Efficacy, Burden, Specialist/Nurses, Center/Hospital, General Satisfaction). | From baseline to 12 months, 24 months and 36 months | 
| Changes of Hemo-QoL (A and SF) score | Hemo-QoL-A:Hemophilia Quality of Life Measure for adults Hemo-QoL-SF:Hemophilia Quality of Life short form for children Hemo-QoL-A is a hemophiliaspecific quality of life questionnaire for adults aged 18 years and above. The questionnaire has 41 items covering 6 domains: Physical Functioning, Role Functioning, Worry, Consequences of Bleeding, Emotional Impact, and Treatment Concerns. For patients younger than 18 years, the Hemo-QoL-SF Questionnaire for children and adolescents (12 to 17 years) is used. The Hemo-QoL-SF contains 35 questions covering 9 domains: Physical Health, View of Yourself, Family, Friends, Others, Sports, Dealing, and Treatment. | From baseline to 12 months, 24 months and 36 months | 
| Changes of WPAI score | WPAI:Work Productivity and Activity Impairment Scale。 Scores are expressed as percentages of impairment/ productivity loss, with higher scores indicating greater impairment. | From baseline to 12 months, 24 months and 36 months |