Damoctocog alfa pegol in patients aged 7 to<12; an interim analysis of the Alfa-PROTECT extension
Makaleler > Tam Makale · 2026
Yazarlar: A K Chan, M C Ozelo, M Luciani, H Glosli, K Kavakli, N Samji, M Maas Enriquez, G Makris, P Kaur, L C Oliveira, S Gupta, M G Arbesú, M Davoli, M E Mancuso, S El Hadouchi
Hämostaseologie, Uluslararası, Wos, 2026, cilt 46, ss S38-S39
Özet
Introduction The safety and efficacy of damoctocog alfa pegol for the prevention and treatment of bleeding in previously treated children with severe hemophilia A has been re-examined in the Alfa-PROTECT trial (NCT05147662) and its 18-month extension. Here, we aim to evaluate the safety and efficacy of damoctocog alfa pegol in children aged 7 to<12 years during the ongoing extension. Method Alfa-PROTECT is a phase 3, multicenter, open-label, single-arm study comprising a 6-month,≥50 exposure days (EDs), main study (completed) and an 18-month extension. As of August 31, 2024, of 35 patients enrolled in the main study, 32 completed the main study and entered the extension. Of those, 30 were included in the modified intent-to-treat (mITT) group. The median (range) treatment duration was 360 (104–504) days with a median (range) of 97 (20–145) EDs (mITT). Participant demographics are shown in [ Table 1 ]. Results Overall, 22/32 patients (69%) experienced at least one adverse event (AE); all but two were mild or moderate. One patient (3%) had a drug-related AE (infusion-site pain). No AEs of special interest were reported, and there were no deaths. Of 21 total treated bleeds in the mITT population, the mean (SD) number of infusions to control a bleed was 1.5 (2.4). Bleed characteristics are shown in [ Table 2 ]. Treatment response was reported as ‘excellent’ or ‘good’ in 18/21 cases (86%). During the extension, 4/32 patients (13.3%) switched from twice-weekly prophylaxis to every-5-days. One patient (3.3%) switched from every-5-days to twice-weekly. The median (range) FVIII dose for prophylaxis was 59.3 (41–72) IU/kg per infusion. The median (range) annual utilization was 5808 (4352–7251) IU/kg/year. Conclusion Data from this analysis confirm the good safety profile and efficacy of damoctocog alfa pegol prophylaxis in children aged 7 to<12 years with severe hemophilia A. Publication History Article published online: 17 February 2026 © 2026. Thieme. All rights reserved. Georg Thieme Verlag KG Oswald-Hesse-Straße 50, 70469 Stuttgart, Germany