TITLE:
Effective prophylaxis with rFVIIa in young haemophiliacs with inhibitors using a schedule similar to FVIII prophylaxis in non-inhibitor patients
AUTHORS:
María Fernanda López-Fernández, Carmen Altisent, Víctor Jiménez-Yuste, Faustino García-Candel, Carmen Sedano, Ana Cid, Eva Mingot, Ramiro Núñez, María José Paloma, Inmaculada Soto, Manuel Prieto
KEYWORDS:
rFVIIa; Haemarthroses; Haemophilia; Inhibitors; Prophylaxis
JOURNAL NAME:
Health,
Vol.5 No.7,
July
24,
2013
ABSTRACT: Objective: To assess
the role of early prophylaxis with recombinant activated factor VII (rFVIIa) in
young haemophiliacs with inhibitors and to determine whether it can reduce
bleeding episodes and prevent joint damage. Patients and Methods: Ten severe haemophiliacs, less of three
years old, with up to four joint bleeds and inhibitors to FVIII who started
early prophylaxis with rFVIIa, were included. A number of haemorrhages/year/patient
and haemarthroses/ year/patient were compared before the start of prophylaxis,
which include both the time before (initial period) and after inhibitor
diagnosis (inhibitor period), with those during prophylaxis (prophylaxis
period). Results: The mean time of inhibitor diagnosis was 15.6 months (range:
2.3-34.1). The mean time between inhibitor diagnosis and the start of
prophylaxis was 7.1 months (range: 0-23.2), shorter than the time of rFVIIa
prophylaxis (mean: 10.3 months; range: 4.1-32.0). Bleeding episodes for the
three time periods were 45, 36 and 17, respectively, or 0.29 and 0.51 haemorrhages/year/patient
in the two periods
prior to prophylaxis compared to 0.17 during prophylaxis. Total haemarthroses
for the three-time periods were 7, 5 and 5, respectively. The haemarthroses/year/patient
in the pre-prophylaxis period were 0.032 and 0.070, compared to0.049 inthe prophylaxis
period. rFVIIa schedules were either 90 μg/kg three times weekly or 90 μg/kg
daily. Conclusions: Early prophylaxis with rFVIIa may be efficacious in young
haemophiliacs with inhibitors, reducing joint bleeds. After the risky period of
inhibitor presence, they are able to continue rFVIII prophylaxis with success assured
to prevent arthropathy.