title: Prophylaxis in 10 patients with severe haemophilia A and inhibitor: different approaches for different clinical situations.
abstract: The effect of bypassing agents is not as predictable as replacement therapy with the deficient factor in inhibitor patients. Consequently, these patients have more levels of arthropathy than patients without inhibitors. Prophylaxis for inhibitor patients has gained attention over the last decade and some papers have reported that bypassing agents could work in the prevention of arthropathy. However, there is a lack data to support any specific agent or regimen or even to recommend their use in different clinical conditions. We report ten patients with haemophilia A and inhibitors treated prophylacticaly with bypassing agents (5 with FEIBA and 5 with NovoSeven). The variable conditioning the choice of one agent or the other was the intention to initiate of immune tolerance induction therapy (ITI) in the future. In 8/10 patients (4 in FEIBA group and 4 in rFVIIa group) there was a decrease of bleeding episodes while 9/10 maintained or increased their joint range of motion (ROM). In the rFVIIa prophylaxis group, prophylaxis can be considered primary since all of them had had less than one joint bleed before prophylaxis. Economic analysis showed that prophylaxis is an expensive treatment. In our experience both agents seem to be safe and effective in reducing the number of bleeds in patients with inhibitors. The anamnestic response provoked by FEIBA could be an issue while awaiting a decline in titres before ITI can be initiated and so rFVIIa may be the best option for prophylaxis in patients with inhibitors who have not yet begun ITI.
authors.affiliation: Haematology Department, Hospital Universitario La Paz, Madrid, Spain.
authors.forename: V
authors.initials: V
authors.lastname: Jiménez-Yuste
authors.forename: M T
authors.initials: MT
authors.lastname: Alvarez
authors.forename: M
authors.initials: M
authors.lastname: Martín-Salces
authors.forename: M
authors.initials: M
authors.lastname: Quintana
authors.forename: C
authors.initials: C
authors.lastname: Rodriguez-Merchan
authors.forename: C
authors.initials: C
authors.lastname: Lopez-Cabarcos
authors.forename: F
authors.initials: F
authors.lastname: Velasco
authors.forename: F
authors.initials: F
authors.lastname: Hernández-Navarro
mesh_terms: D000328:Adult; D001779:Blood Coagulation Factors / Q000009:adverse effects / Q000191:economics / Q000627:therapeutic use*; D002648:Child; D002675:Child, Preschool; D016527:Drug Costs / Q000706:statistics & numerical data; D004341:Drug Evaluation / Q000379:methods; D005169:Factor VIII / Q000276:immunology*; D015942:Factor VIIa / Q000009:adverse effects / Q000191:economics / Q000627:therapeutic use*; D006395:Hemarthrosis / Q000209:etiology / Q000517:prevention & control; D006467:Hemophilia A / Q000150:complications / Q000188:drug therapy* / Q000191:economics / Q000276:immunology; D006470:Hemorrhage / Q000209:etiology / Q000517:prevention & control*; D006801:Humans; D007108:Immune Tolerance; D007223:Infant; D007518:Isoantibodies / Q000097:blood; D008297:Male; D016059:Range of Motion, Articular / Q000187:drug effects; D011994:Recombinant Proteins / Q000009:adverse effects / Q000191:economics / Q000627:therapeutic use; D012189:Retrospective Studies; D055815:Young Adult
chemical_list: D001779:Blood Coagulation Factors; D007518:Isoantibodies; D011994:Recombinant Proteins; D005169:Factor VIII; C103587:recombinant FVIIa; C065655:anti-inhibitor coagulant complex; D015942:Factor VIIa