Hoots W K, Ebbesen L S, Konkle B A, Auerswald G K-H, Roberts H R, Weatherall J, Ferran J-M, Ljung R C R
Department of Pediatrics and Internal Medicine, University of Texas Medical School, Houston, TX 77030, USA.
Haemophilia. 2008 May;14(3):466-75. doi: 10.1111/j.1365-2516.2008.01654.x. Epub 2008 Feb 12.
Haemophilia patients with inhibitors characteristically have impaired joint function and reduced health-related quality of life (HRQoL). This analysis examined whether secondary prophylaxis with recombinant activated factor VII (rFVIIa) improves HRQoL vs. conventional on-demand therapy in patients with haemophilia with inhibitors and frequent bleeds. After a 3-month preprophylaxis period, 22 patients received daily rFVIIa prophylaxis (90 or 270 microg kg(-1)) for 3 months, followed by 3 months' postprophylaxis. Days of hospitalization, absence from school/work and mobility aids requirements were recorded. HRQoL was assessed by EuroQoL (EQ-5D) questionnaire, visual analogue scale (VAS), derived Time to Trade-Off (TTO) scores and Quality Adjusted Life Years (QALYs). rFVIIa prophylaxis significantly (P < 0.0001) reduced bleeding frequency vs. prior on-demand therapy. Hospitalization (5.9% vs. 13.5%; P = 0.0026) and absenteeism from school/work (16.7% vs. 38.7%; P = 0.0127) decreased during prophylaxis; these effects tended to be maintained during postprophylaxis. HRQoL (evaluated by EQ-5D) tended to improve during and after rFVIIa prophylaxis. Notably, pain decreased and mobility increased in 40.9% and 27.3% of patients, respectively, at the end of the postprophylaxis period vs. preprophylaxis. Median VAS score increased from 66 to 73 (P = 0.048), and TTO scores suggested better HRQoL (0.62 vs. 0.76; P = 0.054) during postprophylaxis than preprophylaxis. Small to moderate changes in effect sizes were reported for VAS and TTO scores. Median QALYs were 0.68 (VAS) and 0.73 (TTO). Reductions in bleeding frequency with secondary rFVIIa prophylaxis were associated with improved HRQoL vs. on-demand therapy.
有抑制物的血友病患者通常存在关节功能受损和健康相关生活质量（HRQoL）下降的情况。本分析研究了在有抑制物且频繁出血的血友病患者中，与传统按需治疗相比，重组活化凝血因子VII（rFVIIa）二级预防是否能改善HRQoL。在3个月的预防前期之后，22例患者接受了为期3个月的每日rFVIIa预防治疗（90或270微克/千克），随后是3个月的预防后期。记录住院天数、缺课/缺勤天数以及对行动辅助器具的需求。通过欧洲五维健康量表（EQ-5D）问卷、视觉模拟量表（VAS）、推导的时间权衡（TTO）分数和质量调整生命年（QALYs）来评估HRQoL。与先前的按需治疗相比，rFVIIa预防显著（P<0.0001）降低了出血频率。预防期间住院率（5.9%对13.5%；P=0.0026）和缺课/缺勤率（16.7%对38.7%；P=0.0127）下降；这些效果在预防后期倾向于维持。rFVIIa预防期间及之后，HRQoL（通过EQ-5D评估）倾向于改善。值得注意的是，与预防前期相比，在预防后期结束时，分别有40.9%和27.3%的患者疼痛减轻且活动能力增强。VAS评分中位数从66增加到73（P=0.048），TTO分数表明预防后期的HRQoL（0.62对0.76；P=0.054）优于预防前期。报告VAS和TTO分数的效应大小有小到中度的变化。QALYs中位数为0.68（VAS）和0.73（TTO）。与按需治疗相比，rFVIIa二级预防降低出血频率与改善HRQoL相关。
