title: Impact of choice of treatment for bleeding episodes on inhibitor outcome in patients with mild/moderate hemophilia a and inhibitors.
abstract: Patients with mild/moderate hemophilia A (MHA) may develop inhibitors to factor VIII (FVIII). In this condition, FVIII clotting activity (FVIII:C) baseline levels may remain stable for some patients, but may be reduced to less than 0.01 U/mL for others. Several risk factors for the development of inhibitors in MHA have been proposed. Genetic factors, such as mutations in the FVIII gene, may play a central role; however, other influences, such as intensive treatment with FVIII products, may also be important. Optimal treatment regimens have yet to be determined, not only for the eradication of inhibitors, but also for the management or surgical prophylaxis of hemorrhages associated with this condition. Several treatment options for the control of bleeding in patients with MHA and inhibitors (MHAI) are currently available, and the choice of therapeutic strategy should be given careful consideration; some treatments may produce an anamnestic response, thus delaying the return to FVIII:C baseline levels and adversely affecting the duration of the severe bleeding phenotype. To increase our knowledge of MHAI, a retrospective collection of data is currently being performed among hemophilia centers in France and Belgium. Based on five examples of patients with MHAI collated from preliminary study data, we illustrate the impact on inhibitor outcome of the therapeutic choices used to treat bleeding episodes in these patients.
authors.affiliation: Hemophilia Center, APHP Bicêtre University Hospital, Le Kremlin-Bicêtre, France. rosaline.doiron@bct.ap-hop-paris.fr
authors.forename: Roseline
authors.initials: R
authors.lastname: d'Oiron
authors.forename: F
authors.initials: F
authors.lastname: Volot
authors.forename: J
authors.initials: J
authors.lastname: Reynaud
authors.forename: K
authors.initials: K
authors.lastname: Peerlinck
authors.forename: J
authors.initials: J
authors.lastname: Goudemand
authors.forename: C
authors.initials: C
authors.lastname: Guérois
authors.forename: C
authors.initials: C
authors.lastname: Rothschild
authors.forename: H
authors.initials: H
authors.lastname: Chambost
authors.forename: A
authors.initials: A
authors.lastname: Borel-Derlon
authors.forename: V
authors.initials: V
authors.lastname: Roussel-Robert
authors.forename: A
authors.initials: A
authors.lastname: Marquès-Verdier
authors.forename: A
authors.initials: A
authors.lastname: Lienhart
authors.forename: A M
authors.initials: AM
authors.lastname: Berthier
authors.forename: P
authors.initials: P
authors.lastname: Moreau
authors.forename: T
authors.initials: T
authors.lastname: Lambert
mesh_terms: D019774:Blood Coagulation Factor Inhibitors / Q000235:genetics; D002648:Child; D002675:Child, Preschool; D005169:Factor VIII / Q000008:administration & dosage* / Q000235:genetics; D005260:Female; D006467:Hemophilia A / Q000150:complications / Q000188:drug therapy* / Q000235:genetics; D006470:Hemorrhage / Q000188:drug therapy* / Q000209:etiology / Q000235:genetics; D006801:Humans; D007223:Infant; D008297:Male; D016896:Treatment Outcome
chemical_list: D019774:Blood Coagulation Factor Inhibitors; D005169:Factor VIII