Abstract Number: PB1047
Meeting: ISTH 2020 Congress
Theme: Hemophilia and Rare Bleeding Disorders » Hemophilia - Clinical
Background: As the only clinically proven approach to eradicate inhibitors, immune tolerance induction (ITI) is the primary treatment strategy in patients with severe haemophilia A (HA) who develop inhibitors to FVIII.
Aims: The aim of this analysis was to evaluate ITI outcomes in patients with inhibitors who were treated with simoctocog alfa (Nuwiq®, a human cell line-derived recombinant FVIII) according to routine clinical practice in 3 UK centres.
Methods: Ten patients with severe HA and inhibitors were treated with simoctocog alfa for ITI. The ITI regimen was at the discretion of the treating physician. ITI outcome was assessed based on the following success criteria: undetectable inhibitor titre (< 0.6 BU/mL); FVIII recovery ≥ 66%; and FVIII half-life ≥ 6 hours. Tolerability, safety and the occurrence of bleeding episodes were assessed.
Results: Median age at ITI start was 18 months (range 9-193). Eight patients were high responders, 2 were low responders. The starting ITI regimen for 7 patients was 100 IU/kg daily, while one patient each received 100, 90 and 50 IU/kg every other day. Inhibitors were eradicated in 8 patients (80%). Two patients achieved complete success (all 3 criteria) after 6 and 14.5 months. Six patients achieved partial success (2 criteria), of whom 4 continue to receive ITI. During ITI, 6 patients (60%) experienced no spontaneous bleeds, 3 patients experienced 1 bleed, and 1 patient had 2 bleeds. Simoctocog alfa was well tolerated, with no adverse drug reactions reported.
Conclusions: In this ongoing cohort, 8 of 10 patients receiving ITI with simoctocog alfa have achieved an undetectable inhibitor titre and 6 patients have not experienced any spontaneous bleeds. These data indicate that ITI with simoctocog alfa is effective and well tolerated in patients with HA and inhibitors.
To cite this abstract in AMA style:
Hall GW, Wilkinson A, Mathias M, Bhatnagar N, Biss T, Liesner RJ. Immune Tolerance Induction with Simoctocog Alfa in Patients with Haemophilia A: An Ongoing Multicentre Case Series from the UK [abstract]. Res Pract Thromb Haemost. 2020; 4 (Suppl 1). https://abstracts.isth.org/abstract/immune-tolerance-induction-with-simoctocog-alfa-in-patients-with-haemophilia-a-an-ongoing-multicentre-case-series-from-the-uk/. Accessed October 1, 2023.« Back to ISTH 2020 Congress
ISTH Congress Abstracts - https://abstracts.isth.org/abstract/immune-tolerance-induction-with-simoctocog-alfa-in-patients-with-haemophilia-a-an-ongoing-multicentre-case-series-from-the-uk/