Shrestha Anshu, Su Jun, Li Nanxin, Barnowski Christopher, Jain Nisha, Everson Katie, Jena Anupam Bapu, Batt Katharine
Precision Health Economics and Outcomes Research Los Angeles CA USA.
Sanofi Cambridge MA USA.
Res Pract Thromb Haemost. 2020 Dec 30;5(1):94-103. doi: 10.1002/rth2.12437. eCollection 2021 Jan.
Few have assessed physical activity (PA) and annual bleed rates (ABRs) among people with hemophilia on extended half-life (EHL) factors (recombinant factor VIII Fc [rFVIIIFc]/recombinant factor IX Fc [rFIXFc]) and conventional factors (recombinant factor VIII [rFVIII]/recombinant factor IX [rFIX]).
To assess changes in PA and ABR at consecutive annual visits in individuals with severe hemophilia A and B (HA/HB) on prophylactic treatment with rFVIIIFc/rFIXFc versus rFVIII/rFIX.
PATIENTS/METHODS: We conducted a retrospective chart review of 344 people with severe HA/HB (ages 6-35) receiving prophylaxis with rFVIIIFc/rFIXFc (EHL factors) or rFVIII/rFIX (conventional factors) for ≥6 months in 2014-2015. Differences in changes in outcomes from 2014 to 2015 were compared across the treatment groups.
Baseline characteristics and adherence to the prophylactic regimen were similar across the treatment groups. Greater increase in weekly PA frequency and duration were observed among all EHL groups, except for children treated with rFIXFc. The increase in PA frequency was greater among the children on rFVIIIFc group, adults on rFVIIIFc group, and adults on rFIXFc group by 1.2, 1.2, and 1.4 events/week, respectively, compared to their rFVIII/rFIX counterparts. The increases in PA duration were 44, 60, and 80 min/wk greater among the children on rFVIIIFc, adults on rFVIIIFc, and adults on rFIXFc groups, respectively. Larger reductions in total ABR were observed in children and adults treated with rFVIIIFc compared to rFVIII (0.4 and 0.7 fewer bleeds). Larger reductions were also observed in spontaneous ABR in adult rFVIIIFc and rFIXFc groups (0.8 and 0.3 fewer bleeds, respectively).
This study suggests that rFVIIIFc/FIXFc agents can positively impact PA while maintaining low ABRs.
很少有人评估过接受延长半衰期(EHL)因子(重组凝血因子VIII Fc[rFVIIIFc]/重组凝血因子IX Fc[rFIXFc])和传统因子(重组凝血因子VIII[rFVIII]/重组凝血因子IX[rFIX])治疗的血友病患者的身体活动(PA)和年出血率(ABR)。
评估接受rFVIIIFc/rFIXFc与rFVIII/rFIX预防性治疗的重度甲型和乙型血友病(HA/HB)患者在连续年度随访时PA和ABR的变化。
患者/方法:我们对2014 - 2015年期间接受rFVIIIFc/rFIXFc(EHL因子)或rFVIII/rFIX(传统因子)预防性治疗≥6个月的344例重度HA/HB患者(年龄6 - 35岁)进行了回顾性病历审查。比较了各治疗组2014年至2015年结局变化的差异。
各治疗组的基线特征和预防性治疗方案的依从性相似。除接受rFIXFc治疗的儿童外,所有EHL组的每周PA频率和持续时间均有更大增加。与接受rFVIII/rFIX治疗的儿童、成人相比,接受rFVIIIFc治疗的儿童组、接受rFVIIIFc治疗的成人组和接受rFIXFc治疗的成人组的PA频率增加分别更大,每周增加1.2、1.2和1.4次。接受rFVIIIFc治疗的儿童组、接受rFVIIIFc治疗的成人组和接受rFIXFc治疗的成人组的PA持续时间分别每周增加44、60和80分钟。与rFVIII相比,接受rFVIIIFc治疗的儿童和成人的总ABR降低幅度更大(出血次数减少0.4次和减少0.7次)。在接受rFVIIIFc和rFIXFc治疗的成人组中,自发性ABR也有更大幅度降低(分别减少0.8次和减少0.3次)。
本研究表明,rFVIIIFc/FIXFc药物在维持低ABR的同时可对PA产生积极影响。