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Long‐term safety and efficacy of emicizumab for up to 5.8 years and patients’ perceptions of symptoms and daily life: A phase 1/2 study in patients with severe haemophilia A

Haemophilia Dec 13, 2020

Shima M, Nagao A, Taki M, et al. - In patients with severe hemophilia A with or without factor VIII inhibitors, the safety and efficacy results of phase 1 and phase 1/2 extension trials of the bispecific antibody emicizumab for up to 2.8 years were previously recorded, so researchers here assessed longer‐term data, including the perceptions of patients at the end of the study. At maintenance doses of 0.3, 1 or 3 mg/kg with potential up‐titration, emicizumab was administered subcutaneously once weekly. All patients were later moved to an approved 1.5 mg/kg maintenance dose. For up to 5.8 years, 18 patients received emicizumab. The majority of adverse effects were mild and not related to emicizumab. Long-term prophylaxis of emicizumab for up to 5.8 years has been safe and effective and can improve the everyday lives and feelings of patients, irrespective of inhibitor status. For bleeding severity and time until bleeding stops, all patients answered "improved" except one answering "slightly improved."

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