Hemophilia A With Inhibitor
Conditions
Keywords
Elocta, rFVIIIFc
Brief summary
A chart review study of patients with haemophilia A with inhibitors treated with rFVIIIFc (Elocta®) for immune tolerance induction.
Detailed description
A multicenter, international, non- interventional, retrospective and prospective medical chart review study. Data will be collected from medical records for patients diagnosed with haemophilia A who have been, or who are currently, treated with rFVIIIFc for ITI . The study will be descriptive in nature and report on baseline characteristics, treatment and outcomes for patients who have been, or who are currently, treated with rFVIIIFc for ITI.
Interventions
OTHERrFVIIIFc
Drug according to prescription
Sponsors
Swedish Orphan Biovitrum
Study design
Observational model
COHORT
Time perspective
OTHER
Eligibility
Sex/Gender
ALL
Healthy volunteers
No
Inclusion criteria
* Patients diagnosed with haemophilia A who have been, or who are currently, treated with rFVIIIFc for ITI. * Signed and dated informed consent provided by the patient, or the patient's legally acceptable representative for patients under the legal age, before any study-related data collection are undertaken. Assent should be obtained from paediatric patients according to local regulations.
Exclusion criteria
* Current participation in any investigational medicinal product trial.
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| ITI with rFVIIIFc: Main dose | From 2018 to 2022 | Main dose will be assessed on the prescribed dose (IU/kg) |
| ITI with rFVIIIFc: Main injection frequency | From 2018 to 2022 | Main injection frequency will be assessed on the prescribed frequency |
| ITI with rFVIIIFc: Duration | From 2018 to 2022 | Number of treatment months |
| ITI with rFVIIIFc: Concomitant by-passing agents | From 2018 to 2022 | Product name and main dose will be used to describe any concomitant use of by-passing agents. |
| Outcome of ITI with rFVIIIFc: Overall outcome | From 2018 to 2022 | The Investigator will assess overall outcome as: success, partial success, failure, early withdrawal or other. |
| Outcome of ITI with rFVIIIFc: Time to undetectable inhibitor titer | From 2018 to 2022 | Treatment time to reach undetectable inhibitor levels (\<0.6 BU/ml) |
| Outcome of ITI with rFVIIIFc: Time to normal recovery | From 2018 to 2022 | Treatment time to reach normal recovery levels (≥66% of the expected value) |
| Outcome of ITI with rFVIIIFc: Time to success | From 2018 to 2022 | Treatment time to reach success (see outcome #5) |
| Outcome of ITI with rFVIIIFc: Inhibitor titer levels | From 2018 to 2022 | BU/ml |
| Outcome of ITI with rFVIIIFc: Half-life | From 2018 to 2022 | FVIII half-life (hours) |
| Outcome of ITI with rFVIIIFc: Recovery level | From 2018 to 2022 | FVIII recovery level (%) |
| Outcome of ITI with rFVIIIFc: Bleeds | From 2018 to 2022 | Number of bleeds per month during ITI-treatment |
| Long-term outcome after ITI with rFVIIIFc: Occurrence of relapse | From 2018 to 2022 | Occurrence of relapse (Yes/No) will be assessed by the investigator. |
| Long-term outcome after ITI with rFVIIIFc: Time to relapse | From 2018 to 2022 | Time to occurrence of relapse (see outcome #13) |
| Long-term outcome after ITI with rFVIIIFc: Treatment regimen | From 2018 to 2022 | Treatment regimen will be described as: ITI, prophylaxis or on-demand; and also by product used. |
| Long-term outcome after ITI with rFVIIIFc: Bleeds | From 2018 to 2022 | Number of bleeds per month. |
Countries
France, Germany, Ireland, Italy, Kuwait, Norway, Saudi Arabia, Switzerland
Outcome results
None listed