目标:对于A型血友病(PwHA)患者,关节出血导致关节损伤和血友病相关的关节病,影响运动范围和预期寿命。现有的A型血友病管理指南支持医疗保健专业人员(HCP)和PwHA努力维护关节健康。然而,应该审查这种指导,考虑本手稿中提出的新证据和共识。
方法:在英国PwHA管理方面有经验的15个HCP参加了三轮Delphi小组。在≥70%的小组成员中定义了共识,同意或不同意李克特规模的问题,对于多项或单项选择题,≥70%选择相同的选项。未达成共识的问题已在下一轮进行了修订。
结果:26.8%(11/41),在第1、2和3轮中,分别有44.8%(13/29)和93.3%(14/15)的声明达成共识。HCP同意,应向基线因子VIII(FVIII)水平≤5IU/dL的患者提供预防,在没有治疗负担的地方,预防的目的应该是达到FVIII的谷值水平≥15IU/dL,并在FVIII水平≥20-30IU/dL的情况下维持较长时间,以提供更好的出血保护.PwHA的理想目标是防止所有关节出血,这可以通过维持正常化(50-150IU/dL)FVIII水平来实现。
结论:专家小组在PwHA中保持关节健康的方法上基本一致,这一共识可能有助于指导HCPs。
OBJECTIVE: For people with haemophilia A (PwHA), bleeding in the
joints leads to joint damage and haemophilia-related arthropathy, impacting range of motion and life expectancy. Existing
guidelines for managing haemophilia A support healthcare professionals (HCPs) and PwHA in their efforts to preserve joint health. However, such guidance should be reviewed, considering emerging evidence and
consensus as presented in this manuscript.
METHODS: Fifteen HCPs experienced in the management of PwHA in the UK participated in a three-round Delphi panel.
Consensus was defined at ≥70% of panellists agreeing or disagreeing for Likert-scale questions, and ≥70% selecting the same option for multiple- or single-choice questions. Questions not reaching
consensus were revised for the next round.
RESULTS: 26.8% (11/41), 44.8% (13/29) and 93.3% (14/15) of statements reached consensus in Rounds 1, 2 and 3, respectively. HCPs agreed that prophylaxis should be offered to patients with a baseline factor VIII (FVIII) level of ≤5 IU/dL and that, where there is no treatment burden, the aim of prophylaxis should be to achieve a trough FVIII level ≥15 IU/dL and maintain a longer period with FVIII levels of ≥20-30 IU/dL to provide better bleed protection. The aspirational goal for PwHA is to prevent all joint bleeds, which may be achieved by maintaining normalised (50-150 IU/dL) FVIII levels.
CONCLUSIONS: The panel of experts were largely aligned on approaches to preserving joint health in PwHA, and this consensus may help guide HCPs.