Pediatric Health, Medicine and Therapeutics (Feb 2022)
Managing Severe Hemophilia A in Children: Pharmacotherapeutic Options
Abstract
Katherine Regling,1,2 Michael U Callaghan,1– 3 Robert Sidonio Jr4 1Pediatric Hematology Oncology, Children’s Hospital of Michigan, Detroit, MI, USA; 2Central Michigan University School of Medicine, Mount Pleasant, MI, USA; 3Agios Pharmaceuticals, Cambridge, MA, USA; 4Emory University and Aflac Cancer and Blood Disorders, Atlanta, GA, USACorrespondence: Katherine Regling Pediatric Hematology Oncology, Children’s Hospital of Michigan, 3901 Beaubien Blvd, Detroit, MI, 48201, USA Tel +1 313 745 5515 Fax +1 313 745 5237 Email [email protected]: 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.Keywords: factor VIII, therapeutic management, coagulation, pediatrics
