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.