Dtsch Arztebl Int
-
AAV (adeno-associated virus)-based gene therapy is a new treatment for hemophilia and has recently received approval for the treatment of severe hemophilia A. It does not suffer from the limitations of the current standard treatment (regular prophylactic intravenous injections of the missing clotting factor; subcutaneous injection of a bispecific antibody in hemophilia A) and can, it is hoped, raise the concentration of the missing clotting factor over the long term. AAV-based gene therapy can only be performed once, however, because of the generation of antibodies to AAV. ⋯ Gene therapy for hemophilia holds out the prospect of freedom from hemorrhage without the need for regular treatment with drugs. The various steps that need to be carried out in gene therapy should be coordinated in a graded and partly overlapping integrated care model (a so-called hub-and-spoke model). Electronic platforms should be used for data acquisition and transmission.
-
The proper management of patients being treated with platelet aggregation inhibitors or anticoagulant drugs is a common clinical problem for both elective and emergency procedures in gastroenterology and visceral surgery. The essential matters that must be kept in mind in this situation are the hemorrhagic risk of the procedure, the indication for anticoagulation, and the pharmacology of anticoagulant drugs and platelet aggregation inhibitors. ⋯ Knowledge of the current recommendations on the management of anticoagulants before gastroenterological and visceral surgical procedures gives the clinician a well-founded means of dealing with this complex and common clinical situation.