血友病
医学
血友病B
血友病A
凝血因子
儿科
因子IX
关节病
凝结
外科
内科学
替代医学
骨关节炎
病理
作者
Víctor Jiménez‐Yuste,María Teresa Álvarez‐Román
标识
DOI:10.1016/s2352-3026(23)00057-1
摘要
Haemophilia is a congenital X-linked bleeding disorder caused by deficiency of coagulation factors VIII (haemophilia A) or IX (haemophilia B). Recurrent joint bleeding, leading to the development of arthropathy, is the hallmark of haemophilia and is more pronounced in people with moderate to severe forms of the disease (<1–5% of normal factor VIII or IX concentrations). 1 Srivastava A Santagostino E Dougall A et al. WFH Guidelines for the Management of Hemophilia, 3rd edition. Haemophilia. 2020; 26: 1-158 Crossref PubMed Scopus (651) Google Scholar Prophylaxis is the mainstay therapy for enhancing haemostasis sufficiently to prevent and control bleeding events in people with moderate to severe haemophilia. The development of neutralising antibodies (inhibitors) against factor VIII or factor IX that render factor replacement therapy ineffective is the most serious complication of haemophilia treatment. 2 Giangrande PLF Hermans C O'Mahony B et al. European principles of inhibitor management in patients with haemophilia. Orphanet J Rare Dis. 2018; 13: 66 Crossref PubMed Scopus (28) Google Scholar Fitusiran prophylaxis in people with severe haemophilia A or haemophilia B without inhibitors (ATLAS-A/B): a multicentre, open-label, randomised, phase 3 trialIn participants with haemophilia A or B without inhibitors, fitusiran prophylaxis resulted in significant reductions in annualised bleeding rate compared with on-demand clotting factor concentrates and no bleeding events in approximately half of participants. Fitusiran prophylaxis shows haemostatic efficacy in both haemophilia A and haemophilia B, and therefore has the potential to be transformative in the management of all people with haemophilia. Full-Text PDF
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