Abstract：
BACKGROUND: The real-world effectiveness of the efmoroctocog alfa (recombinant FVIII Fc fusion protein, a rFVIIIFc) has been investigated in numerous studies, however, currently, there exists no comprehensive collection of the existing real-world evidence (RWE) on the performance of prophylactic use of rFVIIIFc.
OBJECTIVE: The aims of this systematic literature study were to identify, review, evaluate and collate the RWE of prophylactic rFVIIIFc for patients with haemophilia A reported in Europe.
METHODS: We searched Medline and Embase from 2014 to February 2022 to identify publications reporting the effectiveness of rFVIIIFc in patients with haemophilia A. The outcomes of interest were annualised bleeding rates (ABR, AjBR, AsBR), injection frequency, factor consumption, adherence, development of inhibitors and quality-of-life measures.
RESULTS: 46 eligible publications (eight full-text articles) were included. rFVIIIFc showed a low ABR in patients with haemophilia A. Studies assessing treatment switching from a standard half-life (SHL) treatment to rFVIIIFc found that the ABR and consumption were reduced in most patients. Studies assessing rFVIIIFc effectiveness reported a median ABR between 0.0 and 2.0 with median injections per week ranging between 1.8 and 2.4 and median doses between 60 and 105 IU/kg/week. Of the studies assessing inhibitor development, only one study reported an incidence of a low titre inhibitor, and no patients developed clinically significant inhibitors.
CONCLUSIONS: rFVIIIFc prophylaxis treatment results in a low ABR across studies in patients with haemophilia A in a European real-world setting, which correlates with findings from clinical trials assessing the efficacy of rFVIIIFc in patients with haemophilia A.
OBJECTIVE: The aims of this systematic literature study were to identify, review, evaluate and collate the RWE of prophylactic rFVIIIFc for patients with haemophilia A reported in Europe.
METHODS: We searched Medline and Embase from 2014 to February 2022 to identify publications reporting the effectiveness of rFVIIIFc in patients with haemophilia A. The outcomes of interest were annualised bleeding rates (ABR, AjBR, AsBR), injection frequency, factor consumption, adherence, development of inhibitors and quality-of-life measures.
RESULTS: 46 eligible publications (eight full-text articles) were included. rFVIIIFc showed a low ABR in patients with haemophilia A. Studies assessing treatment switching from a standard half-life (SHL) treatment to rFVIIIFc found that the ABR and consumption were reduced in most patients. Studies assessing rFVIIIFc effectiveness reported a median ABR between 0.0 and 2.0 with median injections per week ranging between 1.8 and 2.4 and median doses between 60 and 105 IU/kg/week. Of the studies assessing inhibitor development, only one study reported an incidence of a low titre inhibitor, and no patients developed clinically significant inhibitors.
CONCLUSIONS: rFVIIIFc prophylaxis treatment results in a low ABR across studies in patients with haemophilia A in a European real-world setting, which correlates with findings from clinical trials assessing the efficacy of rFVIIIFc in patients with haemophilia A.
摘要：
背景：efmoroctocogalfa（重组FVIIIFc融合蛋白，rFVIIIFc)已经在许多研究中进行了研究，然而,目前,目前尚无关于rFVIIIFc预防性使用表现的现有真实世界证据(RWE)的全面收集.
目的：本系统文献研究的目的是确定，review,评估和整理欧洲报道的A型血友病患者预防性rFVIIIFc的RWE。
方法：我们搜索了2014年至2022年2月的Medline和Embase，以确定报告rFVIIIFc在血友病A患者中有效性的出版物。感兴趣的结果是年度出血率（ABR，AjBR,AsBR),注入频率,要素消耗，坚持,抑制剂和生活质量措施的发展。
结果：纳入46篇合格出版物（8篇全文文章）。rFVIIIFc在血友病A患者中显示低ABR。评估从标准半衰期（SHL）治疗到rFVIIIFc治疗的治疗转换的研究发现，大多数患者的ABR和消耗减少。评估rFVIIIFc有效性的研究报告，ABR中位数在0.0和2.0之间，每周注射中位数在1.8和2.4之间，中位剂量在60和105IU/kg/周之间。在评估抑制剂发展的研究中，只有一项研究报告了低滴度抑制剂的发生率，并且没有患者出现临床上显著的抑制剂。
结论：rFVIIIFc预防治疗导致在欧洲现实世界中A型血友病患者的ABR较低，这与评估rFVIIIFc在血友病患者中的疗效的临床试验结果相关。
目的：本系统文献研究的目的是确定，review,评估和整理欧洲报道的A型血友病患者预防性rFVIIIFc的RWE。
方法：我们搜索了2014年至2022年2月的Medline和Embase，以确定报告rFVIIIFc在血友病A患者中有效性的出版物。感兴趣的结果是年度出血率（ABR，AjBR,AsBR),注入频率,要素消耗，坚持,抑制剂和生活质量措施的发展。
结果：纳入46篇合格出版物（8篇全文文章）。rFVIIIFc在血友病A患者中显示低ABR。评估从标准半衰期（SHL）治疗到rFVIIIFc治疗的治疗转换的研究发现，大多数患者的ABR和消耗减少。评估rFVIIIFc有效性的研究报告，ABR中位数在0.0和2.0之间，每周注射中位数在1.8和2.4之间，中位剂量在60和105IU/kg/周之间。在评估抑制剂发展的研究中，只有一项研究报告了低滴度抑制剂的发生率，并且没有患者出现临床上显著的抑制剂。
结论：rFVIIIFc预防治疗导致在欧洲现实世界中A型血友病患者的ABR较低，这与评估rFVIIIFc在血友病患者中的疗效的临床试验结果相关。
