Hemophilia A
Conditions
Brief summary
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.
Interventions
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.
Sponsors
Bayer
Study design
Observational model
COHORT
Time perspective
PROSPECTIVE
Eligibility
Sex/Gender
ALL
Healthy volunteers
No
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.
Design outcomes
Primary
| Measure | 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
| Measure | Time frame | Description |
|---|---|---|
| Occurrence of AEs. | Up to 36 months | 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. |
| 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) | From baseline to 12 months, 24 months and 36 months | HJHS:Hemophilia Joint Health Score |
| Joint status evaluation by ultrasound (HEAD-US score), if available or part of standard clinical practice. | Up to 36 months | HEAD-US:Hemophilia Early Arthropathy Detection with Ultrasound |
| 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. | Up to 36 months | Bleeds includes total, spontaneous, joint, and trauma. |
| 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. | Up to 36 months | AUC:Area under the plasma concentration versus time curve |
| 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 | From baseline to 12 months, 24 months and 36 months | 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). |
| Changes of Hemo-QoL (A and SF) score | From baseline to 12 months, 24 months and 36 months | 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. |
| Changes of WPAI score | From baseline to 12 months, 24 months and 36 months | WPAI:Work Productivity and Activity Impairment Scale。 Scores are expressed as percentages of impairment/ productivity loss, with higher scores indicating greater impairment. |
Countries
Belgium, Brazil, Canada, Colombia, Denmark, Germany, Greece, Italy, Japan, Kuwait, Netherlands, Norway, Saudi Arabia, Slovenia, Spain, Sweden, Switzerland, Taiwan, United Arab Emirates, United States
Outcome results
None listed