INSERM, UMR 1059, Dysfonction Vasculaire et de l'Hémostase, Université de Lyon, Saint Etienne, France; Laboratoire de Pharmacologie - Toxicologie, CHU de Saint-Etienne, Saint-Etienne, France.
Unité d'Hémostase Clinique, Hôpital Cardiologique Louis Pradel, Université Lyon 1, Lyon, France.
Thromb Res. 2020 Aug;192:52-60. doi: 10.1016/j.thromres.2020.05.005. Epub 2020 May 11.
Replacement therapy with concentrates of factor VIII or IX remains the gold standard for severe haemophilia management. The recent development of clotting factor products with extended half-life, widely available on the market since 2 years, facilitates adherence, improves considerably the patients' quality of life, and simplifies the management of breakthrough bleedings or surgery. These molecules have also brought to the limelight the concepts of optimization and personalization of anti-haemophilic prophylaxis. Pharmacokinetics (PK) is one of the tools that can help haematologists to adapt in a more objective and precise manner the prophylaxis regimen to each individual patient's specific needs. For many years, clinicians at haemophilia centres have been using some simple PK parameters, such as recovery and residual level. However, recently, they have been confronted with an important number of new PK parameters they were not familiar with, but that can be used to improve patient management. Due to the accumulation of PK data and their relative complexity, it is now necessary to analyse the relevance of the different PK parameters relative to haemophilia specificities, and also to know their limits to better use them.
VIII 因子或 IX 因子浓缩物的替代疗法仍然是严重血友病管理的金标准。近年来，具有延长半衰期的凝血因子产品得到了广泛的发展，并于 2 年前在市场上广泛应用，这有利于提高患者的依从性，极大地改善了患者的生活质量，并简化了突破性出血或手术的管理。这些分子还凸显了优化和个性化抗血友病预防治疗的概念。药代动力学（PK）是一种可以帮助血液学家更客观、更精确地根据每个患者的具体需求调整预防治疗方案的工具之一。多年来，血友病中心的临床医生一直在使用一些简单的 PK 参数，如恢复和残留水平。然而，最近，他们遇到了许多他们不熟悉的新 PK 参数，但这些参数可以用于改善患者的管理。由于 PK 数据的积累及其相对复杂性，现在有必要分析不同 PK 参数与血友病特异性的相关性，以及了解其局限性，以便更好地利用它们。
