Clinical Trial WATCH |
Efanesoctocog Alfa Prophylaxis for Children with Severe Hemophilia
Time to read: 0 minutes, 56 seconds.Source: NEJM
Date Originally Published: 17 July 2024
Type of article: Clinical Trial Watch
MedED Catalogue Reference: MPLCT0012
Category: Paediatrics & Neonatology
Category Cross-reference: Haematology, Critical Care, Emergency Room Medicine
Keywords: haemophilia, FVIII, paediatrics, neonatology, haemophilia A,Efanesoctocog alfa (ALUTVIIIO)
This summary is intended to provide a snapshot of the original research. It is in no way a substitute for the original research article, nor is it intended to be a complete reflection of the original research.
Originally published in NEJM,17 July 2024
Once weekly, efanesoctocog alfa offers superior bleeding prevention and high sustained factor VIII activity in patients aged 12 or older with severe haemophilia A. However, data on its effectiveness in children under 12 are limited.
Seventy-four previously treated male patients with severe haemophilia (38 aged <6 years, 36 aged 6 to <12 years) were enrolled in this phase 3 open-label study.
Patients received once-weekly prophylaxis with efanesoctocog alfa (50 IU/kg) for 52 weeks. The primary endpoint was factor VIII inhibitor occurrence, with secondary endpoints including bleeding rates, treatment, safety, and pharmacokinetics.
Among the patients, 47 (64%) had no treated bleeding episodes, 65 (88%) had no spontaneous bleeding episodes, and 61 (82%) had no joint bleeding episodes. Of 43 bleeding episodes, 41 (95%) resolved with a single injection of efanesoctocog alfa.
The researchers conclude that the data showed that in cases of severe haemophiliac in patients aged twelve years and younger, once weekly, efanesoctocog alfa maintained high factor VIII levels, effectively preventing bleeding with mostly nonserious adverse events.
ClinicalTrials.gov( NCT04759131)
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