既往未经治疗的重度甲型血友病患者从血浆源性因子VIII转换为重组因子VIII后的抑制剂发生情况：PUP-SWITCH研究
Inhibitor development upon switching from plasma-derived to recombinant factor VIII in previously untreated patients with severe hemophilia A: the PUP-SWITCH study.
作者信息
Miri Syna, Rosendaal Frits R, Kavakli Kaan, Eshghi Peyman, Moghaddam Soha Mohammadi, Scardo Sara, Habibpanah Behnaz, Elalfy Mohsen, Halimeh Susan, Nicolò Gabriella, Gökçebay Dilek, Özbek Namık, Celkan Tiraje, Mohammadi Ahmad, Karimi Mehran, Shahsavani Amin, Yılmaz Bariş, Albayrak Canan, Gunes Burcak, Kaya Zühre, Ay Yilmaz, Akbayram Sinan, Sarper Nazan, Mannucci Pier Mannuccio, Peyvandi Flora
机构信息
Angelo Bianchi Bonomi Hemophilia and Thrombosis Center, Fondazione IRCCS Ca' Granda, Ospedale Maggiore Policlinico, Milan, Italy.
Department of Clinical Epidemiology, Leiden University Medical Center, Leiden, the Netherlands.
出版信息
Res Pract Thromb Haemost. 2024 Oct 18;8(8):102595. doi: 10.1016/j.rpth.2024.102595. eCollection 2024 Nov.
BACKGROUND
The SIPPET randomized clinical trial showed that in previously untreated patients (PUPs) with severe hemophilia A, treatment with plasma-derived factor (F)VIII (pdFVIII) within the first 50 exposure days (EDs) was associated with a lower cumulative incidence of inhibitors than with recombinant FVIII (rFVIII). Switching to rFVIII beyond 50 EDs with pdFVIII is a treatment often implemented by many centers. The question is whether or not this switch may induce a risk of inhibitor development.
OBJECTIVES
We investigated if in PUPs with severe hemophilia A switched after 50 EDs from pdFVIII to rFVIII, a novel inhibitor peak appears.
METHODS
The PUP-SWITCH observational retrospective study was designed to investigate the cumulative incidence of novel inhibitors after switching PUPs to rFVIII after 50 and before 150 EDs. Hemophilia centers that routinely switched PUPs from pdFVIII to rFVIII within this exposure time frame were invited to participate. Patients were followed up for at least 50 EDs after the switch.
RESULTS
Ninety-seven patients were evaluated, and 87 were included according to eligibility criteria between 2020 and 2022. Only one of them developed an inhibitor 20 EDs after switching, so the cumulative incidence was 1.15% (95% CI, 0.03%-6.24%).
CONCLUSION
PUP-SWITCH, a study focusing on PUPs undergoing a product class switch from pdFVIII to rFVIII after 50 EDs, showed that switching appears to be safe pertaining to the risk of development of new inhibitors.
背景
SIPPET随机临床试验表明，在既往未接受治疗的重度甲型血友病患者（PUPs）中，在最初50个暴露日（EDs）内使用血浆源性凝血因子VIII（pdFVIII）治疗，与重组凝血因子VIII（rFVIII）相比，抑制剂的累积发生率更低。许多中心经常采用在使用pdFVIII超过50个EDs后改用rFVIII的治疗方法。问题在于这种转换是否会引发抑制剂产生的风险。
目的
我们研究了在50个EDs后从pdFVIII转换为rFVIII的重度甲型血友病PUPs中，是否会出现新的抑制剂峰值。
方法
PUP-SWITCH观察性回顾性研究旨在调查PUPs在50个EDs至150个EDs之间转换为rFVIII后新抑制剂的累积发生率。邀请了在此暴露时间范围内常规将PUPs从pdFVIII转换为rFVIII的血友病中心参与。转换后对患者进行至少50个EDs的随访。
结果
评估了97例患者，根据纳入标准，2020年至2022年期间有87例被纳入。其中只有1例在转换后20个EDs出现了抑制剂，因此累积发生率为1.15%（95%CI，0.03%-6.24%）。
结论
PUP-SWITCH研究聚焦于在50个EDs后从pdFVIII转换为rFVIII的PUPs，结果显示就新抑制剂产生的风险而言，这种转换似乎是安全的。
Zotero 插件