Yi Weijia, Guo Yuxia, Zeng Xing, et al. Clinical Retrospective Analysis of 5 Children with Hemophilia A Complicated with Recombinant Factor Ⅷ Inhibitor Formation. [J]. 28(10):37-41(2022)
DOI:
Yi Weijia, Guo Yuxia, Zeng Xing, et al. Clinical Retrospective Analysis of 5 Children with Hemophilia A Complicated with Recombinant Factor Ⅷ Inhibitor Formation. [J]. 28(10):37-41(2022) DOI: 10.13407/j.cnki.jpp.1672-108X.2022.10.011.
Clinical Retrospective Analysis of 5 Children with Hemophilia A Complicated with Recombinant Factor Ⅷ Inhibitor Formation
Objective: To retrospectively analyze the clinical data of 5 cases of hemophilia A (HA) complicated with recombinant factor Ⅷ (FⅧ) inhibitor formation
so as to guide the treatment of HA. Methods: The clinical characteristics of 5 cases of HA complicated with FⅧ inhibitor formation were retrospectively analyzed
and the mutation of hemophilia-related genes was detected by polymerase chain reaction (PCR) technology and DNA sequencing analysis. Results: All of the 5 patients were male and received gene FⅧ on-demand treatment in other hospitals. Five cases were tested for HA genes in the children and mothers
and all were due to maternal inheritance. The median age at inhibitor discovery was 24 months. Five cases were unwilling to induce immune tolerance therapy after the bleeding was stopped. Case 1 received low-dose emecizumab prophylaxis
case 3 and 4 received on-demand treatment with four-factor prothrombin complex concentrate
case 2 continued standard prophylaxis with third-generation recombinant FⅧ
and case 5 received low-dose prophylaxis with third-generation recombinant FⅧ. The follow-up period was (7.60±1.75) months until Jan. 2021. The inhibitor in 3 children with high titer was still >5 BU/mL
and the inhibitor in 2 children had returned to normal state. Conclusion: Inhibitors may occur in children with severe HA treated with FⅧ
and the production of inhibitor is closely related to both genetic and therapeutic factors. After bleeding events in HA children with inhibitor
the appropriate hemostatic scheme should be selected as far as possible according to the bleeding situation of the child and the specific situation of the family
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