Managing Severe Hemophilia A in Children: Pharmacotherapeutic Options

Authors: Regling, K; Callaghan, MU; Sidonio, R, JR

Affiliations: Pediatric Hematology Oncology, Children’s Hospital of Michigan, Detroit, MI, USA ; Central Michigan University School of Medicine, Mount Pleasant, MI, USA ; Agios Pharmaceuticals, Cambridge, MA, USA ; Emory University and Aflac Cancer and Blood Disorders, Atlanta, GA, USA.

Publication: Pediatric health, medicine and therapeutics ; 2022 ; 13. 27–35

Abstract: Hemophilia A is the most common severe inherited bleeding disorder in males. Initial treatment strategies focused on the use of factor concentrates to prevent joint bleeding and the development of long-term crippling arthropathy. The current standard of care has evolved from regular replacement of factor VIII concentrates which has significantly improved the quality of life for those with severe disease to include and consider novel therapies that augment or bypass the hemostatic pathway (ie, emicizumab, Mim8). Other pipeline therapies that suppress specific natural anticoagulant pathways (ie, antithrombin, TFPI) to reestablish hemostatic balance are under Phase 3 trial investigation. These novel therapeutics have allowed providers more variety in dosing regimens and ease of administration while also maintaining effective bleeding prevention. The possibility of « curative » gene therapy is under exploration, with ongoing clinical trials in adult males.