Volume 13 (2026): Issue 1

Real-world Treatment Patterns and Outcomes of Switching to Efanesoctocog Alfa in Children with Haemophilia A: A Single-Centre Experience from the United Arab Emirates

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Muhammad Faisal Khanani ORCID iD
Najam Awan
Haydar Jawad Alrufaey
Manal Rayyan
Layla Al Reheymi

Abstract

Background: Haemophilia A management has advanced significantly with the introduction of extended half-life (EHL) recombinant factor VIII (FVIII) therapies, notably efmoroctocog alfa.

Aims: This report evaluated treatment patterns and outcomes of switching from efmoroctocog alfa to efanesoctocog alfa prophylaxis in children with haemophilia A in the United Arab Emirates (UAE).

Methods: This retrospective, single-centre report evaluated 22 paediatric, male haemophilia A patients (median age =9.0 years [IQR: 7.3–15.5]) transitioning from efmoroctocog alfa to efanesoctocog alfa prophylaxis at Tawam Hospital, UAE. All patients received efmoroctocog alfa prophylaxis during the pre-switch period (January 2023 to July 2024) before transitioning to efanesoctocog alfa in August 2024 and were followed up until March 2025 (observation period).

Results: Twenty-one patients (95.5%) had severe disease. During the pre-switching period, half of the cohort experienced joint bleeding episodes; eight patients (36.4%) experienced one joint bleed, and three patients (13.6%) experienced two or more bleeds. The knee was the most frequently affected joint by bleed (54.6%), followed by the elbow (18.2%). The median prophylactic dose of efanesoctocog alfa was 57.1 IU/kg (IQR: 50.0–63.9). Patients received efanesoctocog alfa for eight months. During the observation period, 100% of patients achieved a zero ABR, with no reported treatment-emergent adverse events or discontinuations. All patients demonstrated 100% adherence to the prescribed once-weekly efanesoctocog alfa regimen. No active inhibitors or treatment-emergent adverse events were observed.

Conclusions: Switching from efmoroctocog alfa to once-weekly efanesoctocog alfa prophylaxis demonstrated highly effective bleeding prevention and a favourable safety profile in paediatric patients.

Plain Language Summary

Haemophilia A is an inherited bleeding disorder in which the body does not have enough working factor VIII, a protein needed for normal blood clotting. Children with haemophilia A can have repeated bleeding, especially into joints such as the knees, elbows, and ankles. Over time, these bleeds can cause pain, joint damage, reduced movement, and difficulties with daily life, school, and physical activity. Regular preventive treatment is therefore used to reduce bleeding before it happens.


This study looked at children with haemophilia A treated at Tawam Hospital in the United Arab Emirates (UAE) who changed from one long-acting factor VIII treatment, efmoroctocog alfa, to a newer once-weekly treatment, efanesoctocog alfa. The research was needed because clinical trials have shown that efanesoctocog alfa can reduce bleeding, but real-world information from children in routine care, especially in the UAE, is still limited.


The study included 22 boys with haemophilia A. Most had severe haemophilia. Before switching treatment, half of the children had experienced joint bleeding, most often in the knee. After switching to once-weekly efanesoctocog alfa and being followed for eight months, all children had no recorded treated bleeds. No child developed an active inhibitor, no treatment-related side effects were recorded, and no child stopped treatment. All children also followed the prescribed once-weekly schedule.


These findings suggest that switching to efanesoctocog alfa may provide strong bleed protection with fewer injections for children with haemophilia A. This may be important for children and families because better prevention of bleeding can help protect joints, reduce treatment burden, and support more stable daily life. Larger studies with longer follow-up are still needed to confirm long-term safety and joint-health benefits.

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