rFVIIIFc (Elocta®) ITI Chart Review in Patients With Haemophilia A
- Conditions
- Hemophilia A With Inhibitor
- Interventions
- Other: rFVIIIFc
- Registration Number
- NCT03951103
- Lead Sponsor
- Swedish Orphan Biovitrum
- 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.
Recruitment & Eligibility
- Status
- COMPLETED
- Sex
- All
- Target Recruitment
- 44
- 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.
- Current participation in any investigational medicinal product trial.
Study & Design
- Study Type
- OBSERVATIONAL
- Study Design
- Not specified
- Arm && Interventions
Group Intervention Description Hemophili A patients rFVIIIFc Patients treated with rFVIIIFc for ITI
- Primary Outcome Measures
Name Time Method Outcome of ITI with rFVIIIFc: Recovery level From 2018 to 2022 FVIII recovery level (%)
ITI with rFVIIIFc: Duration From 2018 to 2022 Number of treatment months
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 normal recovery From 2018 to 2022 Treatment time to reach normal recovery levels (≥66% of the expected value)
ITI with rFVIIIFc: Main dose From 2018 to 2022 Main dose will be assessed on the prescribed dose (IU/kg)
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)
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: Occurrence of relapse From 2018 to 2022 Occurrence of relapse (Yes/No) will be assessed by the investigator.
ITI with rFVIIIFc: Main injection frequency From 2018 to 2022 Main injection frequency will be assessed on the prescribed frequency
Long-term outcome after ITI with rFVIIIFc: Time to relapse From 2018 to 2022 Time to occurrence of relapse (see outcome #13)
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: Half-life From 2018 to 2022 FVIII half-life (hours)
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: Bleeds From 2018 to 2022 Number of bleeds per month during ITI-treatment
Long-term outcome after ITI with rFVIIIFc: Bleeds From 2018 to 2022 Number of bleeds per month.
- Secondary Outcome Measures
Name Time Method
Trial Locations
- Locations (3)
Swedish Orphan Biovitrum Research Site
🇨🇭Bern, Switzerland
Swedish Orphan Biovitrum Research Site (a)
🇸🇦Riyadh, Saudi Arabia
Swedish Orphan Biovitrum Research Site (p)
🇸🇦Riyadh, Saudi Arabia