Effective prophylaxis with rFVIIa in young haemophiliacs with inhibitors using a schedule similar to FVIII prophylaxis in non-inhibitor patients

作     者：Maria Fernanda Lopez-Fernandez Carmen Altisent Victor Jimenez-Yuste Faustino Garcia-Candel Carmen Sedano Ana Cid Eva Mingot Ramiro Nunez Maria Jose Paloma Inmaculada Soto Manuel Prieto 

作者机构：HaematologyComplejo Hospitalario Universitario A CorunaA CorunaSpain Haemophilia UnitHospital Universitario Vall d’HebronBarcelonaSpain Haemophilia UnitHospital UniversitarioMadridSpain Haemophilia UnitHospital Universitario Virgen de la ArreixacaMurciaSpain HaematologyComplejo Hospitalario Universitario Marques de ValdecillaSantanderSpain Congenital Coagulophaties UnitHospital Universitario La FeValenciaSpain HaematologyHospital Universitario Carlos HayaMalagaSpain HaematologyHospital Universitario Virgen del RocíoSevillaSpain HaematologyHospital Virgen del CaminoPamplonaSpain HaematologyHospital General de AsturiasOviedoSpain HaematologyHospital General YagüeBurgosSpain 

出 版 物：《Health》 (健康（英文）)

年 卷 期：2013年第5卷第7期

页      面：1151-1157页

学科分类：1002[医学-临床医学] 10[医学] 

主　　题：rFVIIa Haemarthroses Haemophilia Inhibitors Prophylaxis 

摘      要：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.