A chart review study of patients with haemophilia A with inhibitors treated with rFVIIIFc (Elocta®) for immune tolerance induction.
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.
Study Type
OBSERVATIONAL
Enrollment
44
Drug according to prescription
Swedish Orphan Biovitrum Research Site
Paris, France
Swedish Orphan Biovitrum Research Site
Rennes, France
ITI with rFVIIIFc: Main dose
Main dose will be assessed on the prescribed dose (IU/kg)
Time frame: From 2018 to 2022
ITI with rFVIIIFc: Main injection frequency
Main injection frequency will be assessed on the prescribed frequency
Time frame: From 2018 to 2022
ITI with rFVIIIFc: Duration
Number of treatment months
Time frame: From 2018 to 2022
ITI with rFVIIIFc: Concomitant by-passing agents
Product name and main dose will be used to describe any concomitant use of by-passing agents.
Time frame: From 2018 to 2022
Outcome of ITI with rFVIIIFc: Overall outcome
The Investigator will assess overall outcome as: success, partial success, failure, early withdrawal or other.
Time frame: From 2018 to 2022
Outcome of ITI with rFVIIIFc: Time to undetectable inhibitor titer
Treatment time to reach undetectable inhibitor levels (\<0.6 BU/ml)
Time frame: From 2018 to 2022
Outcome of ITI with rFVIIIFc: Time to normal recovery
Treatment time to reach normal recovery levels (≥66% of the expected value)
Time frame: From 2018 to 2022
Outcome of ITI with rFVIIIFc: Time to success
Treatment time to reach success (see outcome #5)
Time frame: From 2018 to 2022
This platform is for informational purposes only and does not constitute medical advice. Always consult a qualified healthcare professional.
Swedish Orphan Biovitrum Research Site
Tours, France
Swedish Orphan Biovitrum Research Site
Frankfurt, Germany
Swedish Orphan Biovitrum Research Site
Friedrichshain, Germany
Swedish Orphan Biovitrum Research Site
Hanover, Germany
Swedish Orphan Biovitrum Research Site
Mitte, Germany
Swedish Orphan Biovitrum Research Site
Dublin, Ireland
Swedish Orphan Biovitrum Research Site
Catania, Italy
Swedish Orphan Biovitrum Research Site
Catanzaro, Italy
...and 8 more locations
Outcome of ITI with rFVIIIFc: Inhibitor titer levels
BU/ml
Time frame: From 2018 to 2022
Outcome of ITI with rFVIIIFc: Half-life
FVIII half-life (hours)
Time frame: From 2018 to 2022
Outcome of ITI with rFVIIIFc: Recovery level
FVIII recovery level (%)
Time frame: From 2018 to 2022
Outcome of ITI with rFVIIIFc: Bleeds
Number of bleeds per month during ITI-treatment
Time frame: From 2018 to 2022
Long-term outcome after ITI with rFVIIIFc: Occurrence of relapse
Occurrence of relapse (Yes/No) will be assessed by the investigator.
Time frame: From 2018 to 2022
Long-term outcome after ITI with rFVIIIFc: Time to relapse
Time to occurrence of relapse (see outcome #13)
Time frame: From 2018 to 2022
Long-term outcome after ITI with rFVIIIFc: Treatment regimen
Treatment regimen will be described as: ITI, prophylaxis or on-demand; and also by product used.
Time frame: From 2018 to 2022
Long-term outcome after ITI with rFVIIIFc: Bleeds
Number of bleeds per month.
Time frame: From 2018 to 2022