Castaman Giancarlo
a Center for Bleeding Disorders , Careggi University Hospital , Florence , Italy.
Expert Rev Hematol. 2018 Aug;11(8):673-683. doi: 10.1080/17474086.2018.1489719. Epub 2018 Jul 18.
The health benefits of prophylactic dosing regimens for clotting factor therapy in patients with hemophilia include reduced joint damage and improved quality of life; as such, prophylaxis is recommended in treatment guidelines. However, many patients with hemophilia B are treated on demand, and prophylaxis has been utilized less frequently than in hemophilia A. Areas covered: This review discusses the opportunities and evidence for prophylaxis in hemophilia B, in the context of treatment guidelines and with regard to factor IX (FIX) replacement therapies, including long-acting recombinant FIX (rFIX). Expert commentary: Long-acting rFIX concentrates may increase uptake of and adherence to prophylaxis regimens through attainment of higher trough levels with longer dosing intervals. In this new era of hemophilia B treatment, physicians may be able to achieve better clinical outcomes for their patients and reconsider treatment goals. Maintaining higher FIX trough levels will undoubtedly have long-term benefits for patients, such as preserving joint function. The long-acting rFIX concentrates support robust prophylaxis regimens and offer physician's flexibility in treating patients to best suit their needs, whether to enable an active lifestyle, to achieve higher trough levels for better bleed protection, or simply to decrease the burden of treatment by reducing injection frequency.
预防性给药方案用于血友病患者凝血因子治疗的健康益处包括减少关节损伤和改善生活质量；因此，治疗指南中推荐采用预防疗法。然而，许多B型血友病患者接受的是按需治疗，与A型血友病相比，预防疗法的使用频率较低。涵盖领域：本综述在治疗指南的背景下，讨论了B型血友病预防疗法的机会和证据，并涉及凝血因子IX（FIX）替代疗法，包括长效重组FIX（rFIX）。专家评论：长效rFIX浓缩剂通过延长给药间隔达到更高的谷浓度，可能会增加预防方案的采用率和依从性。在B型血友病治疗的新时代，医生或许能够为患者取得更好的临床疗效，并重新考虑治疗目标。维持较高的FIX谷浓度无疑将给患者带来长期益处，比如保留关节功能。长效rFIX浓缩剂支持强有力的预防方案，并为医生治疗患者提供灵活性，以最适合患者的需求，无论是实现积极的生活方式、达到更高的谷浓度以更好地预防出血，还是仅仅通过减少注射频率来减轻治疗负担。
