Haemophilia A Haematological Disorders Hereditary factor VIII deficiency
Conditions
Interventions
Cohort A: The participants with congenital severe haemophilia A without Factor VIII (FVIII) inhibitors who are undergoing emicizumab treatment as described in the Hemlibra® (Emicizumab) summary of pro
Sponsors
Roche (Germany)
Eligibility
Sex/Gender
Male
Inclusion criteria
Inclusion criteria: 1. Participants of any age with congenital severe haemophilia A with or without FVIII inhibitors 2. Participants undergoing treatment with emicizumab according to Summary of Product Characteristics (SPC) (start of treatment with emicizumab maximum 3 months prior to study entry) 3. Must sign informed consent by the legal representative or participant or both, as required 4. Selection criteria for Cohort A include participants diagnosed with severe congenital haemophilia A (<1% FVIII activity) and no present FVIII inhibitor at the start of emicizumab treatment, patients who completed successful ITI before the start of Emicizumab treatment are eligible. 5. Selection criteria for Cohort B include participants diagnosed with congenital haemophilia A (any severity) with FVIII inhibitor activity at the start of emicizumab treatment or ongoing ITI at the start of emicizumab treatment
Exclusion criteria
Exclusion criteria: 1. Participants having bleeding disorder other than congenital haemophilia A 2. Treatment with emicizumab outside of the SPC at study entry 3. Any contraindication for treatment with emicizumab according to current SPC 4. Current participation in an interventional study
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Annualized bleeding rates (ABRs) of treated bleeds estimated using nature and number of treated bleeds in the past 24 weeks prior to the emicizumab treatment, derived from participant’s files and treatment diaries from baseline until the end of the study (up to 5 years) | — |
Secondary
| Measure | Time frame |
|---|---|
| 1. Percentage of participants with zero treated bleeds estimated using nature and number of treated bleeds in the past 24 weeks prior to the emicizumab treatment, derived from participant’s files and treatment diaries from baseline until the end of the study (up to 5 years) 2. ABRs of treated spontaneous bleeds, treated joint bleeds, treated target joint bleeds estimated using nature and number of treated bleeds in the past 24 weeks prior to the emicizumab treatment, derived from participant’s files and treatment diaries from baseline until the end of the study (up to 5 years) 3. Percentage of participants with zero treated spontaneous bleeds, treated joint bleeds, treated target joint bleeds estimated using nature and number of treated bleeds in the past 24 weeks prior to the emicizumab treatment, derived from participant’s files and treatment diaries from baseline until the end of the study (up to 5 years) 4. Number of doses and frequency of haemostatic medication besides emicizumab used to treat bleeding events or for other purposes, derived from participant’s files and treatment diaries from baseline until the end of the study (up to 5 years) 5. Percentage of participants with different dosing regimens of emicizumab, derived from participant’s files and treatment diaries from screening up to the end of the study (up to 5 years) 6. Number of invasive surgical procedures, derived from participant’s files and treatment diaries from day 1 up to the end of the study (up to 5 years) 7. Percentage of participants that used pain medication, derived from participant’s files and treatment diaries from day 1 up to end of study (up to 5 years) 8. Percentage of participants with occupational disability related to haemophilia A, derived from participant’s files and treatment diaries from day 1 up to the end of the study (up to 5 years) 9. Number of events of using health resources (number of contacts to the site, unscheduled visits at the site, number of emergency room (er) | — |
Countries
Germany, Switzerland
Contacts
Public ContactMedical Information
Outcome results
None listed