Abstract Number: PB1025
Meeting: ISTH 2020 Congress
Background: A serious complication in hemophilia A is the formation of inhibitors to clotting factors. The primary means for eradicating inhibitors is immune tolerance induction (ITI) therapy. Antihemophilic factor (human) is indicated for the treatment of hemophilia.
Aims: This multicenter retrospective data collection project evaluated patient- and treatment-related factors associated with outcomes following primary or rescue ITI with an antihemophilic factor (human) in patients with hemophilia A and high titer inhibitors.
Methods: Medical records of 13 inhibitor patients treated with antihemophilic factor (human) for primary or rescue ITI therapy between January 1, 2012, and July 31, 2017, was conducted in five US hemophilia treatment centers. To be eligible, patients were required to have a diagnosis of hemophilia A of any severity level, inhibitor to FVIII at the time of treatment initiation with antihemophilic factor (human), and ongoing treatment with antihemophilic factor (human) for primary or rescue ITI. Data were de-identified and analyzed descriptively. Outcome measures were defined, per the International Immune Tolerance consensus recommendations, as “complete success”, “partial success”, or “failure”.
Results: All patients met the inclusion criteria. Ten of 13 patients (76.9%) had successful ITI; seven with complete success and three with partial success. Three patients failed ITI. As primary therapy, complete success was obtained with all but one of the six patients treated with antihemophilic factor (human). These six patients were all older than 7 at the initiation of ITI. Seven of the 13 patients had a combined previous 12 attempts with other products. Of these seven rescue patients, ITI with antihemophilic factor (human) was completely successful in two and partially successful in three.
Conclusions: While retrospective data has limitations, real-world evidence demonstrates that ITI with antihemophilic factor (human) concentrate can be successful or partially successful in diverse populations of moderately complex patients with hemophilia A and inhibitors.
To cite this abstract in AMA style:Escobar M, Shaffer L, Holguin M, McCavit T, Amega N, Rajan S. Immune Tolerance Induction (ITI) with a Plasma-derived Factor VIII for Patients with Hemophilia A and Inhibitors: A Retrospective Study [abstract]. Res Pract Thromb Haemost. 2020; 4 (Suppl 1). https://abstracts.isth.org/abstract/immune-tolerance-induction-iti-with-a-plasma-derived-factor-viii-for-patients-with-hemophilia-a-and-inhibitors-a-retrospective-study/. Accessed September 29, 2023.
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