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Poster Networking

Inherited Bleeding Disorders and Inhibitors

PB1218 - Use of a low maintenance dose of emicizumab in patients with hemophilia A: Experience in a Mexican pediatric center.

Tuesday, June 25, 2024
13:45 – 14:45 ICT
Room: Exhibition Hall
Background: Background. The role of emicizumab in the profilaxis for patients with hemophilia A is widely recognized. There are three stablished regimens for its administration that include a loading dose of 3mg/kg every week for four weeks and a subsecuent maintenance dose of 1.5mg/kg once weekly, 3mg/kg every 2 weeks, or 6mg/kg every 4 weeks must be used. In resource- limited countries it has been described a low dose maintenance dose of 3mg/kg every 4 weeks.

Aims: To present the experience in our pediatric center in the use of a low dose of emicizumab for the treatment of patients with hemophilia A with high-titer inhibitors.

Methods: Retrospective analysis of 3 patients who received a low maintenance dose from January 2021 to march 2022.

Results: In 2021 it was first introduced as a therapy in our pediatric hospital making it a reality for treatment in Mexican pediatric patients with high-titer inhibitors, however, in the first months after introducing profilaxis with emicizumab the resource and budget were not optimal so a low maintenance dose of 3mg/kg every 4 weeks, was used. The three selected patients received a weekly 3mg/kg dose for 4 weeks as a loading dose after which a 3mg/kg maintenance dose was used every 4 weeks instead of the standard 6mg/kg. From January 2021 to march 2022 this maintenance dose was applied with zero bleeding events reported. After march 2022 emicizumab became more available and patients were switched to standard dose of 6mg/kg every 4 weeks.

Conclusion(s): We present a real-world success experience in the use of a low maintenance dose of emicizumab which may be a reasonable profilaxis regimen in the context of centers with limited resources.