Introduction

Given the relatively small number of patients with haemophilia A, head-to-head comparisons between recombinant FVIII (rFVIII) products are difficult to conduct. This study compared the efficacy and consumption of rVIII-SingleChain (lonoctocog alfa, AFSTYLA^®) with rAHF-PFM (octocog alfa, Advate^®) and rFVIIIFc (efmoroctocog alfa, Elocta^®), for the prophylaxis and treatment of bleeding episodes in previously treated adolescents/adults with severe haemophilia A, through a matching-adjusted indirect comparison (MAIC).

Methods

A systematic literature review identified published clinical trials for rAHF-PFM and rFVIIIFc. Individual patient data for rVIII-SingleChain were used to match baseline patient characteristics to those from published trials, using an approach similar to propensity score weighting. After matching, annualized bleeding rates (ABR), percentage of patients with zero bleeds, and rFVIII consumption were compared across trial populations.

Results

Published data were identified from two rAHF-PFM trials and one rFVIIIFc trial. rVIII-SingleChain had similar ABR (risk ratio [RR]: 0.74 [0.16; 3.48]; RR: 1.18 [0.85; 1.65]) and percentage of patients with zero bleeds (odds ratio [OR]: 1.34 [0.56; 3.22]; OR: 0.78 [0.47; 1.31]) versus rAHF-PFM and rFVIIIFc, respectively. Annual rVIII-SingleChain consumption was significantly lower than rAHF-PFM (mean difference: − 1507.66 IU/kg/year [− 2011.71; − 1003.61]) and equivalent to rFVIIIFc (RR: 0.96 [0.62; 1.49]).

Conclusion

Although limited to published information for comparator trials, these results suggest that with an annualized rFVIII consumption comparable to rFVIIIFc, but significantly lower than rAHF-PFM, routine prophylaxis with rVIII-SingleChain is able to maintain a similar ABR and percentage of patients with zero bleeds, attesting to the long-acting nature of rVIII-SingleChain.

中文翻译：

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rVIII-SingleChain 与用于预防性治疗成人/青少年 A 型严重血友病的两种重组 FVIII 产品的疗效和消耗量的匹配调整间接比较

介绍

鉴于 A 型血友病患者数量相对较少，重组 FVIII (rFVIII) 产品之间的头对头比较难以进行。本研究比较了 rVIII-SingleChain (lonoctocog alfa, AFSTYLA ^® ) 与 rAHF-PFM (octocog alfa, Advate ^® ) 和 rFVIIIFc (efmoroctocog alfa, Elocta ^® ) 预防和治疗先前治疗的出血事件的疗效和用量通过匹配调整间接比较 (MAIC) 对患有严重血友病 A 的青少年/成人进行评估。

方法

系统文献回顾确定了已发表的 rAHF-PFM 和 rFVIIIFc 临床试验。rVIII-SingleChain 的个体患者数据用于将基线患者特征与已发表试验的患者特征相匹配，使用类似于倾向评分加权的方法。匹配后，比较试验人群的年化出血率 (ABR)、零出血患者百分比和 rFVIII 消耗量。

结果

已发表的数据来自两项 rAHF-PFM 试验和一项 rFVIIIFc 试验。rVIII-SingleChain 具有相似的 ABR（风险比 [RR]：0.74 [0.16；3.48]；RR：1.18 [0.85；1.65]）和零出血患者百分比（优势比 [OR]：1.34 [0.56；3.22]； OR: 0.78 [0.47; 1.31]) 与 rAHF-PFM 和 rFVIIIFc 分别相比。rVIII-SingleChain 的年消耗量显着低于 rAHF-PFM（平均差异：- 1507.66 IU/kg/年 [- 2011.71；- 1003.61]），与 rFVIIIFc 相当（RR：0.96 [0.62；1.49]）。

结论

尽管仅限于比较试验的已发表信息，但这些结果表明，与 rFVIIIFc 相当但显着低于 rAHF-PFM 的年化 rFVIII 消耗量，使用 rVIII-SingleChain 进行常规预防能够维持相似的 ABR 和零出血患者百分比，证明了 rVIII-SingleChain 的长效特性。