Long-term Study Evaluating Joint Health in People With Haemophilia A Receiving Real-world Prophylactic Treatment With Efanesoctocog Alfa

This is a retrospective-prospective, low-interventional study to evaluate the long-term effectiveness of real-world prophylaxis with efanesoctocog alfa on joint health in people with haemophilia A. Participants in the study must be prescribed efanesoctocog alfa at the discretion of their physicians within 6 months prior to, or at the time of, enrolment. The decision to initiate treatment with efanesoctocog alfa shall be made independently from the decision to include the patients in the study. A broad haemophilia A population (e.g. all severities \[mild to severe\], all ages, patients with comorbidities and/or concomitant use of anticoagulant/antiplatelet drugs, patients with previous inhibitors to Factor FVIII \[FVIII\]) will be eligible to enrol. The study will require mandatory on-site visits with joint assessments. A retrospective data collection on patients' haemophilia, medical, and surgical history will be performed, including previous haemophilia treatment and treated bleeding episodes from 12 months prior to initiating treatment with efanesoctocog alfa until enrolment.

The study will start with the enrolment visit during a patient's routine visit to the hospital, to check whether the patient fulfils all the inclusion criteria and none of the exclusion criteria. Study participants will have yearly on-site visits for 24 months or 36 months, depending on the time of enrolment in relation to study closure. Patients will be followed in the study as long as they are treated with efanesoctocog alfa prophylaxis which would be at the physicians' discretion. Patients will also need to agree to document any bleeding episodes they may have during the study in a study-specific paper diary. Joint assessment, Haemophilia Joint Health Score (HJHS), and completion of Patient-reported outcomes (PROs) will be conducted during the on-site visits. For patients included in the Haemophilia Early Arthropathy Detection with Ultrasound (HEAD-US) group, ultrasound joint assessment will also be done at the yearly visits. The end of study (EoS) visit can be performed at the 24-month visit or the 36-month visit, depending on the time of enrolment.

The target is to enroll approximately 250 participants.

• Annualized bleeding rate (ABR) over the prospective observation period. [Срок оценки: Enrolment to Month 12, 24 and 36]
• Number of injections to control a bleeding event over the prospective observation period. [Срок оценки: Enrolment to Month 12, 24 and 36]
• Total dose to control bleeding event over the prospective observation period. [Срок оценки: Enrolment to Month 12, 24 and 36]
• Haemophilia Joint Health Score (HJHS) version 2.1 [Срок оценки: Enrolment to Month 12, 24 and 36]
• Target joint development on a patient and joint level over the prospective observation period. [Срок оценки: Month 12, 24 and 36]
• Target joint resolution on a patient and joint level over the prospective observation period. [Срок оценки: Month 12, 24 and 36]
• Target joint recurrency on a patient and joint level over the prospective observation period. [Срок оценки: Month 12, 24 and 36]
• Annualised injection frequency over the prospective observation period. [Срок оценки: Month 12, 24 and 36]
• Annualised efanesoctocog alfa consumption for prophylaxis and treatment of bleeds (excluding surgery) over the prospective observation period. [Срок оценки: Month 12, 24 and 36]
• Reason(s) for any changes in prescribed frequency and/or dose of efanesoctocog alfa prophylaxis over the prospective observation period. [Срок оценки: Enrolment to Month 12, 24 and 36]
• Change in ABR in the period of using efanesoctocog alfa vs prior haemophilia treatment. [Срок оценки: Prospective period vs Retrospective period]
• Change in annualised injection frequency during the period on efanesoctocog alfa vs the period on prior haemophilia treatment. [Срок оценки: Prospective period vs Retrospective period]