Human fibrinogen concentrate (HFC) has favorable pharmacokinetics for adult, adolescent, and pediatric patients with congenital fibrinogen deficiency (CFD), according to the results of open-label studies presented at the 2021 American Society of Pediatric Hematology/Oncology (ASPHO) meeting.
The prospective studies FORMA-01, FORMA-02, and FORMA-04 looked at efficacy, safety, and dosing of HFC in different age groups. HFC was used for on-demand treatment of bleeding episodes and as surgical prophylaxis for patients with CFD.
In patients 12 years and older, there were 89 bleeding episodes and 12 surgeries. In patients younger than 12 years old, there were 10 bleeding episodes and 3 surgeries. Patients younger than 12 received a higher total dose of HFC per bleeding episode than patients older than 12. The median loading dose in older patients was 70 mg/kg and 75 mg/kg in younger patients. The median total dose per surgery was 85.8 mg/kg for older patients and 108.10 mg/kg in patients younger than 12.
Overall, the hemostatic efficacy was rated successful for 98.9% of bleeding events and 100% of surgeries across age groups. The authors concluded that HFC has favorable pharmacokinetics similar to other coagulation factors for adults, adolescents and pediatric patients. The authors suggested an in vivo recovery of 1.8 for adults and adolescents and 1.4 for pediatric patients for dose calculations.
Djambas Khayat C, Lohade SD, D’Souza F, et al. Analysis of fibrinogen concentrate pharmacokinetics and dosing in adult, adolescent, and pediatric patients with congenital fibrinogen deficiency. Poster presented at: 2021 American Society of Pediatric Hematology/Oncology meeting; April 21-23, 2021; virtual.