title: Prophylaxis with FEIBA in paediatric patients with haemophilia A and inhibitors.
abstract: The benefits shown with factor VIII (FVIII) prophylaxis relating to joint health and quality of life (QoL) provide the rationale for FEIBA prophylaxis in haemophilia A patients with persistent FVIII inhibitors. FEIBA has previously shown efficacy in preventing bleeds in inhibitor patients who failed to respond to, or were ineligible for immune tolerance induction (ITI). The study examined the outcome of paediatric patients undergoing long-term FEIBA prophylaxis. A retrospective chart review included severe haemophilia A patients with persistent inhibitors aged ≤13 years at the start of FEIBA prophylaxis. Baseline characteristics captured dose, frequency of prophylaxis, history of inhibitor development, including baseline titre, historical peak titre and history of ITI. Outcome measurements included annual bleed rate before and during FEIBA prophylaxis, joint status and school days missed. Sixteen cases of FEIBA prophylaxis from two centres are presented. The mean age of subjects at prophylaxis initiation was 7.5 ± 3.6 years and median baseline inhibitor titre was 23 (range 3.1-170) BU. Prior to prophylaxis initiation, median annual joint bleeds among all patients was 4 (0-48), which dropped significantly after the first year of prophylaxis, to a median annual joint bleed rate of 1 (0-7; P = 0.0179). Subsequent years (median = 9) of prophylaxis therapy demonstrated similarly low annual joint bleed rates. There were no life-threatening bleeds, no viral seroconversions or thrombotic events during FEIBA prophylaxis treatment. FEIBA prophylaxis was effective for preventing joint bleeds and subsequent joint damage, delaying arthropathy and improving outcomes in children with haemophilia A and inhibitors to FVIII, who failed or were ineligible for ITI.
authors.affiliation: City of Hope National Medical Center, Duarte, CA, USA.
authors.forename: N
authors.initials: N
authors.lastname: Ewing
authors.affiliation: HZRM - Hemophilia Centre Rhein Main, Moerfelden-Waldorf, Germany.
authors.forename: C
authors.initials: C
authors.lastname: Escuriola-Ettingshausen
authors.affiliation: HZRM - Hemophilia Centre Rhein Main, Moerfelden-Waldorf, Germany.
authors.forename: W
authors.initials: W
authors.lastname: Kreuz
mesh_terms: D019774:Blood Coagulation Factor Inhibitors / Q000097:blood / Q000276:immunology*; D001779:Blood Coagulation Factors / Q000008:administration & dosage / Q000009:adverse effects / Q000627:therapeutic use*; D002648:Child; D002675:Child, Preschool; D005169:Factor VIII / Q000276:immunology*; D005858:Germany; D006395:Hemarthrosis / Q000209:etiology / Q000517:prevention & control; D006467:Hemophilia A / Q000150:complications / Q000188:drug therapy* / Q000276:immunology*; D006801:Humans; D007223:Infant; D007518:Isoantibodies / Q000097:blood / Q000276:immunology*; D011292:Premedication; D012189:Retrospective Studies; D016896:Treatment Outcome; D014481:United States
chemical_list: D019774:Blood Coagulation Factor Inhibitors; D001779:Blood Coagulation Factors; D007518:Isoantibodies; D005169:Factor VIII; C065655:anti-inhibitor coagulant complex
keywords: FEIBA; FVIII inhibitor; haemophilia A; paediatric; prophylaxis