每日剂量预防血友病:一项评估可行性和疗效的随机交叉先导研究。

Daily dosing prophylaxis for haemophilia: a randomized crossover pilot study evaluating feasibility and efficacy.

机构信息

Malmö Centre for Thrombosis and Haemostasis, Lund University, Malmö, Sweden.

出版信息

Haemophilia. 2012 Nov;18(6):855-9. doi: 10.1111/j.1365-2516.2012.02879.x. Epub 2012 Jun 11.

Abstract

Regular replacement therapy (prophylaxis) for haemophilia has been shown to prevent development of disabling arthropathy and to provide a better quality of life compared to treatment on demand; however, at a substantially higher cost. Calculations based on pharmacokinetic principles have shown that shortening dose intervals may reduce cost. The aim of this prospective, randomized, crossover pilot study was to address whether daily dosing is feasible, if it reduces concentrate consumption and is as effective in preventing bleeding as the standard prophylactic dosing regimen. In a 12+12 month crossover study, 13 patients were randomized to start either their own previously prescribed standard dose, or daily dosing adjusted to maintain at least the same trough levels as obtained with the standard dose. Ten patients completed the study. A 30% reduction in cost of factor concentrates was achieved with daily prophylaxis. However, the number of bleeding events increased in some patients in the daily dosing arm and patients reported decreased quality of life during daily prophylaxis. Daily treatment had a greater impact on daily life, and the patients found it more stressful.Prophylaxis with daily dosing may be feasible and efficacious in some patients. A substantial reduction of factor consumption and costs can be realized, but larger studies are needed before the introduction of daily prophylaxis into clinical routine can be recommended.

摘要

常规替代疗法(预防治疗)已被证明可预防致残性关节炎的发展,并提供更好的生活质量,与按需治疗相比;然而,成本也大大增加。基于药代动力学原理的计算表明,缩短给药间隔可能会降低成本。这项前瞻性、随机、交叉先导研究的目的是探讨每日给药是否可行,如果可行,是否能减少浓缩物的消耗,并像标准预防治疗方案一样有效地预防出血。在一项 12+12 个月的交叉研究中,13 名患者被随机分配开始接受他们自己之前规定的标准剂量,或根据每日剂量调整,以维持至少与标准剂量相同的谷浓度。10 名患者完成了这项研究。每日预防治疗可使因子浓缩物的成本降低 30%。然而,在每日给药组的一些患者中,出血事件的数量增加,患者报告在每日预防治疗期间生活质量下降。每日治疗对日常生活的影响更大,患者发现它更具压力。每日预防治疗在某些患者中可能是可行和有效的。可以实现因子消耗和成本的大量减少,但在推荐将每日预防治疗引入临床常规之前,还需要进行更大规模的研究。

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