title: Prophylactic treatment with activated prothrombin complex concentrate (FEIBA) reduces the frequency of bleeding episodes in paediatric patients with haemophilia A and inhibitors.
abstract: Orthopaedic complications are among the most disabling sequelae occurring in patients with haemophilia and inhibitors. Recurrent or refractory joint bleeds can lead to joint damage, limiting mobility and causing permanent disability. Activated prothrombin complex concentrates (aPCCs) are effective in controlling acute, intraoperative and postoperative bleeding in patients with haemophilia and inhibitors. The relatively long, dosing interval and safety profile distinguish aPCCs as a well-suited option for prophylaxis. Therefore, it is postulated that long-term routine aPCC administration will decrease the frequency of recurrent bleeds, prevent damage to normal joints, and slow the progression of existing joint disease in patients with inhibitors. To test this hypothesis, a retrospective chart audit was performed. In four treatment centres, five patients were identified who received aPCC [Factor Eight Inhibitor Bypassing Activity, Anti-Inhibitor Coagulant Complex (FEIBA); Baxter AG, Vienna, Austria] prophylactically for > or = 6 months to prevent or reduce further joint deterioration, reduce bleeding and prevent postsurgical bleeding. Median treatment duration was 15 months and included administration of >1300 doses of aPCC. Dosages ranged from 50 to 75 U kg(-1) three times per week in four patients; one patient received 100 U kg(-1) daily. Orthopaedic status was maintained in four patients and improved in one; the frequency of bleeding episodes was reduced in all patients. No adverse events or thrombotic complications were reported. This case series demonstrates that routine aPCC administration may be used safely and effectively to reduce the occurrence of bleeding episodes and to maintain or improve clinical joint status in some patients.
authors.affiliation: Section of Hematology/Oncology, Tulane University School of Medicine, New Orleans, LA 70112, USA. cleissi@tulane.edu
authors.forename: C A
authors.initials: CA
authors.lastname: Leissinger
authors.forename: D L
authors.initials: DL
authors.lastname: Becton
authors.forename: N P
authors.initials: NP
authors.lastname: Ewing
authors.forename: L A
authors.initials: LA
authors.lastname: Valentino
mesh_terms: D000293:Adolescent; D019774:Blood Coagulation Factor Inhibitors / Q000627:therapeutic use*; D001779:Blood Coagulation Factors / Q000627:therapeutic use*; D002648:Child; D002675:Child, Preschool; D006395:Hemarthrosis / Q000517:prevention & control*; D006467:Hemophilia A / Q000188:drug therapy*; D006470:Hemorrhage / Q000517:prevention & control*; D006801:Humans; D007223:Infant; D016896:Treatment Outcome
chemical_list: D019774:Blood Coagulation Factor Inhibitors; D001779:Blood Coagulation Factors; C025667:prothrombin complex concentrates