Long-Term Efficacy and Safety of Damoctocog Alfa Pegol Prophylaxis in Patients with Haemophilia A Aged 12–

Abstract

Plain Language SummaryThis research article gives further information about how well damoctocog alfa pegol works, and how safe it is, in those patients who were aged twelve to less-than-eighteen (“adolescents”) when they joined the PROTECT VIII clinical trial in patients 12–65 years with haemophilia A. People living with haemophilia A lack a part of their blood that normally allows clots to form. Damoctocog alfa pegol is a replacement for this missing “clotting factor”; it has also been altered to allow it to stay in the body for longer than other clotting factors, allowing for less frequent infusions. As children become adolescents and begin to manage their own factor replacement, rather than being helped by parents or others who take care of them, a treatment with less infusions is easier to follow. A clotting factor that stays in the body for longer, like damoctocog alfa pegol, might be useful for these adolescent patients. There were twelve people who were adolescents when they joined the PROTECT VIII trial. They spent an average of 4 years in the trial and had on average 1.8 bleeds every year. During the last 6 months of treatment, 8 patients had no bleeds. No study-drug-related serious adverse events were seen, no patient stopped treatment due to an adverse event, no deaths happened and no patients became immune to the treatment. This young, active patient group may benefit from the longer and more flexible dosing offered by damoctocog alfa pegol.