title: The benefits of prophylactic treatment with APCC in patients with haemophilia and high-titre inhibitors: a retrospective case series.
abstract: Prophylactic infusion of factor concentrates is a safe, effective intervention for preventing arthropathy in patients with haemophilia; on-demand treatment is insufficient to prevent the orthopaedic complications and subsequent haemophilic arthropathy that stem from recurrent joint haemorrhages. The usefulness of prophylaxis in haemophilia patients without inhibitors suggests that patients with haemophilia and inhibitors could derive similar benefits. In patients with haemophilia and high-titre (>5 BU mL(-1)) inhibitors, bleeding episodes are treated with bypassing agents such as activated prothrombin complex concentrates (APCCs) and recombinant activated factor VII (rFVIIa, NovoSeven; Novo Nordisk A/S, Bagsvaerd, Denmark). It is possible to administer bypassing therapy regularly to prevent haemorrhages, with the goal of limiting arthropathy and serious life- and limb-threatening bleeding. The data evaluating the efficacy and safety of this approach in patients with inhibitors are limited, consisting of results from one prospective trial and retrospective case reports. This report describes our experience with the prophylactic use of the APCC Factor Eight Inhibitor Bypassing Activity, Anti-Inhibitor Coagulant Complex, Vapor Heated (FEIBA; Baxter AG, Vienna, Austria). Data from patients at one treatment centre were retrospectively evaluated. Case records of six patients with haemophilia A or B and high-titre inhibitors were identified. When APCC was administered regularly, most patients exhibited a reduction in the numbers of haemorrhages, an improvement in orthopaedic status, and an improvement in quality of life. Prophylaxis with APCC can reduce haemorrhages and halt further joint deterioration in patients with haemophilia and inhibitors.
authors.affiliation: Rush Hemophilia and Thrombophilia Center, Rush University Medical Center, Chicago, IL 60612-3833, USA. lvalentino@rush.edu
authors.forename: L A
authors.initials: LA
authors.lastname: Valentino
mesh_terms: D001177:Arthropathy, Neurogenic / Q000188:drug therapy* / Q000191:economics / Q000517:prevention & control; D019774:Blood Coagulation Factor Inhibitors / Q000191:economics / Q000627:therapeutic use*; D001779:Blood Coagulation Factors / Q000191:economics / Q000627:therapeutic use*; D002648:Child; D002675:Child, Preschool; D003362:Cost-Benefit Analysis; D004305:Dose-Response Relationship, Drug; D006395:Hemarthrosis / Q000188:drug therapy* / Q000191:economics / Q000517:prevention & control; D006467:Hemophilia A / Q000150:complications / Q000188:drug therapy* / Q000191:economics; D006801:Humans; D008297:Male; D012189:Retrospective Studies; D016896:Treatment Outcome; D055815:Young Adult
chemical_list: D019774:Blood Coagulation Factor Inhibitors; D001779:Blood Coagulation Factors; C025667:prothrombin complex concentrates