Haemophilia Clinic and Haemostasis Program, Division of Haematology/Oncology, Department of Paediatrics, Hospital for Sick Children, University of Toronto, Toronto, Ontario, Canada.
Hemophilia Treatment Centre, Aziza Othmana Hospital, University Tunis El Manar, Tunis, Tunisia.
Haemophilia. 2022 May;28 Suppl 4:26-34. doi: 10.1111/hae.14552.
Prophylaxis has become standard of care for persons with severe phenotype haemophilia (PWsH). However, 'standard prophylaxis' with either factor or non-factor therapies (emicizumab) is prohibitively expensive for much of the world. We sought to evaluate whether haemophilia care can be provided at a lower cost yet achieve good results using Lower dose/Lower frequency prophylaxis (LDP) and with increasing use of antifibrinolytics (Tranexamic acid and Epsilon amino caproic acid).
We identified 12 studies that collectively included 335 PWsH using LDP. Additionally, we undertook a literature search regarding the benefits of antifibrinolytics in haemophilia care.
Identified studies show that LDP is far superior to no prophylaxis (On demand [OD] therapy) resulting in significant patient benefits. Patients on LDP showed (in comparison to patients OD) on average: 72% less total bleeds; 75% less joint bleeds; 91% less days lost from school; 77% less hospital admission days; and improved quality of life measures. These benefits come at similar or only slightly higher (< 2-fold greater) costs than OD therapy. Antifibrinolytics are effective adjunctive agents in managing bleeds (oral, nasal, intracranial, possibly other) and providing haemostasis for surgeries (particularly oral surgeries). Antifibrinolytics can substitute for more expensive factor concentrates or can reduce the use of such concentrates. There is evidence to show that antifibrinolytics may be used in conjunction with factor concentrates/emicizumab for more effective/less costly prophylaxis.
The use of LDP along with appropriate and increased use of antifibrinolytics offers less resourced countries good options for managing patients with haemophilia.
对于严重表型血友病患者（PWsH），预防已成为标准治疗方法。然而，对于世界上大多数地区而言，因子或非因子治疗（依库珠单抗）的“标准预防”费用过高。我们试图评估使用低剂量/低频率预防（LDP）和增加使用抗纤维蛋白溶解剂（氨甲环酸和ε-氨基己酸）是否可以以更低的成本提供血友病治疗，同时获得良好的效果。
我们确定了 12 项研究，这些研究总共纳入了 335 名使用 LDP 的 PWsH。此外，我们还进行了一项关于抗纤维蛋白溶解剂在血友病治疗中益处的文献检索。
已确定的研究表明，LDP 远优于无预防（按需 [OD] 治疗），可显著改善患者的状况。与 OD 治疗相比，使用 LDP 的患者平均：总出血减少 72%；关节出血减少 75%；因上学而损失的天数减少 91%；住院天数减少 77%；生活质量得到改善。这些益处的成本与 OD 治疗相似或仅略高（增加不到 2 倍）。抗纤维蛋白溶解剂是管理出血（口服、鼻内、颅内、可能还有其他部位）和提供手术止血（特别是口腔手术）的有效辅助药物。抗纤维蛋白溶解剂可以替代更昂贵的因子浓缩物，也可以减少此类浓缩物的使用。有证据表明，抗纤维蛋白溶解剂可与因子浓缩物/依库珠单抗联合使用，以实现更有效/更经济的预防。
使用 LDP 并适当增加使用抗纤维蛋白溶解剂为资源较少的国家提供了管理血友病患者的良好选择。
