title: Immune tolerance therapy in paediatric haemophiliacs with factor VIII inhibitors: 14 years follow-up.
abstract: We report our clinical experience in the immune tolerance (IT) therapy of 21 paediatric haemophiliacs with FVIII inhibitor: high responders (16HR) received initially FVIII twice daily at a dosage of 50-300 U/kg/day, 11/16 received a concomitant treatment with activated prothrombin complex concentrate (100-200 U/kg/day). Low responders (five LR) received 20-100 FVIII U/kg every second or third day. Inhibitor elimination was achieved in 19/21 patients in a median time of 4 months in HR and 1.5 months in LR. The outcome and length of time needed to induce IT was significantly correlated with FVIII exposure between the first inhibitor detection and onset of IT therapy and to interruption of IT therapy. For a rapid elimination of FVIII inhibitors it is important to start continuous administration of high-dose FVIII (≥ 100 FVIII U/kg/day) before repeated exposure to FVIII, in order to prevent rebooster effects, prolongation of elimination time, and to reduce expense.
authors.affiliation: Department of Paediatrics, J. W. Goethe University Hospital, and Department of Internal Medicine, University Hospital, Frankfurt am Main, and Department of Paediatrics, Prof.-Hess-Hospital, Bremen, Germany.
authors.forename: W
authors.initials: W
authors.lastname: Kreuz
authors.affiliation: Department of Paediatrics, J. W. Goethe University Hospital, and Department of Internal Medicine, University Hospital, Frankfurt am Main, and Department of Paediatrics, Prof.-Hess-Hospital, Bremen, Germany.
authors.forename: S
authors.initials: S
authors.lastname: Ehrenforth
authors.affiliation: Department of Paediatrics, J. W. Goethe University Hospital, and Department of Internal Medicine, University Hospital, Frankfurt am Main, and Department of Paediatrics, Prof.-Hess-Hospital, Bremen, Germany.
authors.forename: M
authors.initials: M
authors.lastname: Funk
authors.affiliation: Department of Paediatrics, J. W. Goethe University Hospital, and Department of Internal Medicine, University Hospital, Frankfurt am Main, and Department of Paediatrics, Prof.-Hess-Hospital, Bremen, Germany.
authors.forename: G
authors.initials: G
authors.lastname: Auerswald
authors.affiliation: Department of Paediatrics, J. W. Goethe University Hospital, and Department of Internal Medicine, University Hospital, Frankfurt am Main, and Department of Paediatrics, Prof.-Hess-Hospital, Bremen, Germany.
authors.forename: D
authors.initials: D
authors.lastname: Mentzer
authors.affiliation: Department of Paediatrics, J. W. Goethe University Hospital, and Department of Internal Medicine, University Hospital, Frankfurt am Main, and Department of Paediatrics, Prof.-Hess-Hospital, Bremen, Germany.
authors.forename: J
authors.initials: J
authors.lastname: Joseph-Steiner
authors.affiliation: Department of Paediatrics, J. W. Goethe University Hospital, and Department of Internal Medicine, University Hospital, Frankfurt am Main, and Department of Paediatrics, Prof.-Hess-Hospital, Bremen, Germany.
authors.forename: T
authors.initials: T
authors.lastname: Beeg
authors.affiliation: Department of Paediatrics, J. W. Goethe University Hospital, and Department of Internal Medicine, University Hospital, Frankfurt am Main, and Department of Paediatrics, Prof.-Hess-Hospital, Bremen, Germany.
authors.forename: D
authors.initials: D
authors.lastname: Klarman
authors.affiliation: Department of Paediatrics, J. W. Goethe University Hospital, and Department of Internal Medicine, University Hospital, Frankfurt am Main, and Department of Paediatrics, Prof.-Hess-Hospital, Bremen, Germany.
authors.forename: I
authors.initials: I
authors.lastname: Scharrer
authors.affiliation: Department of Paediatrics, J. W. Goethe University Hospital, and Department of Internal Medicine, University Hospital, Frankfurt am Main, and Department of Paediatrics, Prof.-Hess-Hospital, Bremen, Germany.
authors.forename: B
authors.initials: B
authors.lastname: Kornhuber
keywords: children with haemophilia A; factor VIII inhibitors; immune tolerance therapy; requirements for successful FVIII inhibitor elimination