Zanon Ezio, Milan Marta, Pasca Samantha
Hemophilia Center, University Hospital of Padua, Padua, Italy.
Blood Coagul Fibrinolysis. 2018 Jul;29(5):465-468. doi: 10.1097/MBC.0000000000000736.
: The development of alloantibodies against the replacement of Factor VIII (FVIII) is the major complication in haemophilia A treatment. The gold standard to eradicate inhibitors is the immune tolerance induction (ITI), but in some cases it fails requiring another immune tolerance, defined ITI rescue (ITI-R), using a different concentrate, even though it is still debated. We report a successful case of a poor risk (titre of inhibitor at start of ITI > 10 BU/ml, peak titre on ITI > 200 BU/ml, >2 years since the inhibitor diagnosis) haemophilia A child treated with a high-dose regimen (200 UI/kg/day) turoctocog-alfa after a failed first-line ITI with octocog-alfa lasting 29 months. At 22 months of ITI-R, the inhibitor titre was undetectable, the FVIII recovery was 74%, of the expected level and the FVIII half-life more than 7 h. A complete successful ITI-R was then achieved with turoctocog-alfa.
针对凝血因子 VIII（FVIII）替代物产生同种异体抗体是甲型血友病治疗中的主要并发症。消除抑制物的金标准是免疫耐受诱导（ITI），但在某些情况下会失败，需要使用不同的浓缩物进行另一种免疫耐受，即定义为 ITI 挽救（ITI-R），尽管这仍存在争议。我们报告了一例低风险（ITI 开始时抑制物滴度>10 BU/ml，ITI 时峰值滴度>200 BU/ml，自抑制物诊断以来超过 2 年）的甲型血友病儿童，在使用 octocog-alfa 进行为期 29 个月的一线 ITI 失败后，采用高剂量方案（200 UI/kg/天）的 turoctocog-alfa 进行治疗。在 ITI-R 治疗 22 个月时，抑制物滴度检测不到，FVIII 回收率为预期水平的 74%，FVIII 半衰期超过 7 小时。然后使用 turoctocog-alfa 成功实现了完全成功的 ITI-R。
