PB1239 - Beyond prophylaxis? Emicizumab in acute care management of inhibitor positive hemophilia A

Background: Emicizumab, currently the only licensed non-factor replacement is used as prophylaxis for both hemophilia A with or without inhibitors. A loading phase is required to maintain drug level in haemostatic range.

Aims: We describe our experience of using this drug in inhibitor positive Hemophilia A in acute care setting when standard treatment options were either not available or not effective.

Methods: Data of use of inhibitor-positive hemophilia A management betweenDec 2019- Dec 2023 (48 months) were evaluated. Due to resource constraints, Emicizumab was used only in HA patients with high titre inhibitors and poor joint status or life threatening bleeds. The results of patients in whom Emicizumab was started as a desperate measure in acute life or limb threatening settings is described here.

Results: 45 patients of HA with high-titre inhibitor receive Emicizumab at our center currently. 6 children with a median age of 11.5 years (3-17 years) received it in acute care settings which were either limb or life-threatening. The indications were intracranial bleeding (2), and 1 each ofmassive GI bleeding, acute hemarthrosis with cellulitis, compartment syndrome with impending gangrene and burns requiring tissue debridement. In all patients, although prior treatment with FVII and FEIBA was used, prophylaxis was not feasible. Both patients with intracranial bleeding were comatose with GCS of 6 and 7 respectively. Clinical response in the form of reduction of pain, swelling and overt bleeding was noted within 2 hours of use in non-CNS bleed patients. In those with CNS bleeding, improvement in sensorium was noted within 72 hours. All children survived the event and none have any long term sequelae.

Conclusion(s): There is promising data to show that in acute life-threatening bleeds in inhibitor positive hemophilia, there is good response to Emicizumab, although there is no time to achieve a haemostatic level.