The rationale for this study is to further understand and describe the long-term prophylactic effectiveness of efanesoctocog alfa in preventing joint bleeds in a real-life setting.
Male or female patient with a diagnosis of haemophilia A.
Capable of giving signed informed consent, which includes compliance with the requirements and restrictions listed in the informed consent form (ICF) and in this protocol.
Prescribed with efanesoctocog alfa prophylactic treatment within 6 months prior to study enrolment or at the latest at the enrolment visit, in accordance with local regulations.
Must have received prophylactic treatment(s) with any haemophilia product(s) for at least 12 months prior to being prescribed with efanesoctocog alfa treatment.
Acquired haemophilia A and other blood clotting disorders than hereditary haemophilia A.
Any positive FVIII inhibitor result (defined as inhibitor titre ≥0.6 Bethesda unit [BU]/mL) from the medical records in connection to the switch to efanesoctocog alfa until the enrolment visit.
Enrolment in a concurrent clinical interventional study, or intake of an investigational medicinal product (IMP), including for haemophilia prophylaxis, within 3 months prior to enrolment in this study.
Patient not suitable for participation, whatever the reason, as judged by the Investigator, e.g., patient is not able or willing to perform the study assessments.
Single group
Efanesoctocog alfa
DrugParticipant in this study must be prescribed efanesoctocog alfa prophylactic treatment per their standard of care from their physician. No drug will be provided by the Sponsor.
Annualized joint bleeding rate (AjBR) over the prospective observation period.
Annualized bleeding rate (ABR) over the prospective observation period.
Number of injections to control a bleeding event over the prospective observation period.
Total dose to control bleeding event over the prospective observation period.
Cumulative amount of medication administered to effectively manage bleeding episodes.
Haemophilia Joint Health Score (HJHS) version 2.1
Change from enrolment in total and domain scores of HJHS per patient.
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Swedish Orphan Biovitrum
Lead sponsor
PSI CRO
Collaborator
