■需要使用efmotocogalfa(重组因子[F]VIIIFc融合蛋白[rFVIIIFc])和eftrenacogalfa(重组因子IXFc融合蛋白[rFIXFc])的实际经验来弥合证据空白。
■描述rFVIIIFc/rFIXFc在24个月的预期期内的使用情况和有效性。
■预防(NCT03055611),一项针对25个德国血友病治疗中心的非干预性研究,纳入前/纳入时曾接受过rFVIIIFc/rFIXFc个体化预防的A和B血友病患者(所有年龄/严重程度).主要终点包括年出血率(ABR),注入频率(IF),和要素消耗(FC)。此外,我们收集了长达12个月的FVIII/FIX回顾性数据.医生和患者满意度,还评估了安全性结果.
■总的来说,150例患者接受≥1rFVIIIFc剂量,47例患者接受≥1rFIXFc剂量,中位前瞻性随访时间为20.6个月和21.0个月,分别。rFVIIIFc/rFIXFc显示低的中位ABR(0.5/1.7),年化IF(121.8/52.2注射/年),和FC(4611.7/2423.9IU/kg)符合产品标签。与以前的FVIII/FIX相比,rFVIIIFc的ABR降低了56.0%(比率,0.44;95%CI,0.31-0.64),rFIXFc没有变化(比率,0.93;95%CI,0.66-1.31);rFVIIIFc/rFIXFc降低了年化IF(rFVIIIFc,平均差异,-31.7;95%CI,-40.3至-23.1;rFIXFc,平均差异,-37.3;95%CI,-46.9至-27.8),而FC保持稳定(rFVIIIFc,+374.1;95%CI,+46.8至+701.3;rFIXFc,+503.9;95%CI,+95.4至+912.4)。大多数医生和患者对rFVIIIFc/rFIXFc感到满意或高度满意。rFVIIIFc/rFIXFc耐受性良好,无抑制剂开发或治疗相关的严重不良事件。
■Real-worldPREVENT数据补充了3期试验,表明个体化rFVIIIFc/rFIXFc预防可在低IF和维持FC的情况下提供稳定的出血保护。与以前的FVIII相比,使用rFVIIIFc时ABR大大降低,具有稳定的年化FC。对于rFIXFc,与先前的FIX相比,维持了出血保护,同时降低了年化IF。
UNASSIGNED: Real-world experience with efmoroctocog alfa (a recombinant factor [F]VIII Fc fusion protein [rFVIIIFc]) and eftrenonacog alfa (a recombinant factor IX Fc fusion protein [rFIXFc]) is needed to bridge evidence gaps.
UNASSIGNED: To describe rFVIIIFc/rFIXFc usage and effectiveness over a 24-month prospective period.
UNASSIGNED: PREVENT (NCT03055611), a noninterventional study across 25 German hemophilia treatment centers, enrolled previously treated persons with hemophilia A and B (all ages/severities) on individualized rFVIIIFc/rFIXFc prophylaxis before/at enrollment. Primary endpoints included annualized bleeding rate (ABR), injection frequency (IF), and factor consumption (FC). Additionally, up to 12 months of retrospective FVIII/FIX data were collected. Physician and patient satisfaction, and safety outcomes were also assessed.
UNASSIGNED: Overall, 150 patients received ≥1 rFVIIIFc dose and 47 patients received ≥1 rFIXFc dose, with median prospective follow-up of 20.6 and 21.0 months, respectively. rFVIIIFc/rFIXFc demonstrated low median ABR (0.5/1.7), annualized IF (121.8/52.2 injections/y), and FC (4611.7/2423.9 IU/kg) in line with product labels. Compared with previous FVIII/FIX, there was a 56.0% reduction in ABR for rFVIIIFc (rate ratio, 0.44; 95% CI, 0.31-0.64), with no change for rFIXFc (rate ratio, 0.93; 95% CI, 0.66-1.31); rFVIIIFc/rFIXFc reduced annualized IF (rFVIIIFc, mean difference, -31.7; 95% CI, -40.3 to -23.1; rFIXFc, mean difference, -37.3; 95% CI, -46.9 to -27.8), while FC remained stable (rFVIIIFc, +374.1; 95% CI, +46.8 to +701.3; rFIXFc, +503.9; 95% CI, +95.4 to +912.4). Most physicians and patients were satisfied or highly satisfied with rFVIIIFc/rFIXFc. rFVIIIFc/rFIXFc were well tolerated, with no inhibitor development or treatment-related serious adverse events.
UNASSIGNED: Real-world PREVENT data complement phase 3 trials and show that individualized rFVIIIFc/rFIXFc prophylaxis provided stable bleed protection with low IF and maintained FC. Compared with previous FVIII, ABR was considerably reduced with rFVIIIFc, with stable annualized FC. For rFIXFc, bleed protection was maintained vs previous FIX while reducing annualized IF.