Immune Tolerance Induction With a Recombinant Factor VIII Fc in Haemophilia A: Data From a Chart Review Study

Area of Science:

• Hematology
• Immunology
• Pharmacology

Background:

• Hemophilia A is a genetic bleeding disorder caused by factor VIII deficiency.
• Immune tolerance induction (ITI) is a treatment strategy to overcome inhibitors in hemophilia A.
• Recombinant factor VIII Fc fusion protein (rFVIIIFc) is a long-acting factor VIII concentrate.

Purpose of the Study:

• To evaluate the efficacy and safety of rFVIIIFc for ITI in patients with hemophilia A.
• To report real-world data on first-time and rescue ITI using rFVIIIFc.
• To analyze ITI outcomes and treatment regimens associated with rFVIIIFc.

Main Methods:

• Retrospective and prospective real-world data collection from a non-interventional, multicenter study.
• Inclusion of patients treated with rFVIIIFc for ITI.
• Assessment of investigator-defined ITI treatment outcomes and regimens.

Main Results:

• First-time ITI: 81.3% success rate in 16 patients, with median rFVIIIFc consumption of 300 IU/kg/week, mostly non-daily.
• Rescue ITI: 50% success/partial success in 16 patients, with median consumption of 536 IU/kg/week, often daily.
• Forty-one patients from 16 sites were included in the study.

Conclusions:

• First-time ITI with rFVIIIFc demonstrates a high likelihood of success in hemophilia A patients.
• rFVIIIFc is an effective therapeutic option for patients requiring rescue ITI after previous ITI failure.