Khair Kate, Chalmers Elizabeth, Flannery Thuvia, Griffiths Annabel, Rowley Felicity, Tobaruela Guillermo, Chowdary Pratima
Centre for Outcomes and Experience Research in Children's Health Illness and Disability (ORCHID), NIHR Biomedical Research Centre Great Ormond Street Hospital for Children, London, UK.
Paediatric Haemophilia Comprehensive Care Centre, Royal Hospital for Sick Children, Glasgow, UK.
Ther Adv Hematol. 2021 Apr 30;12:20406207211007058. doi: 10.1177/20406207211007058. eCollection 2021.
Despite advances in haemophilia care, inhibitor development remains a significant complication. Although viable treatment options exist, there is some divergence of opinion in the appropriate standard approach to care and goals of treatment. The aim of this study was to assess consensus on United Kingdom (UK) standard of care for child and adult haemophilia patients with inhibitors.
A modified Delphi study was conducted using a two-round online survey. A haemophilia expert steering committee and published literature informed the Round 1 questionnaire. Invited participants included haematologists, haemophilia nurses and physiotherapists who had treated at least one haemophilia patient with inhibitors in the past 5 years. Consensus for 6-point Likert scale questions was pre-defined as ⩾70% participants selecting 1-2 (disagreement) or 5-6 (agreement).
In all, 46.7% and 35.9% questions achieved consensus in Rounds 1 ( = 41) and 2 ( = 34), respectively. Consensus was reached on the importance of improving quality of life (QoL) and reaching clinical goals such as bleed prevention, eradication of inhibitors and pain management. There was agreement on criteria constituting adequate/inadequate responses to immune tolerance induction (ITI) and the appropriate factor VIII dose to address suboptimal ITI response. Opinions varied on treatment aims for adults and children/adolescents, when to offer prophylaxis with bypassing agents and expectations of prophylaxis. Consensus was also lacking on appropriate treatment for mild/moderate patients with inhibitors.
UK healthcare professionals appear to be aligned on the clinical goals and role of ITI when managing haemophilia patients with inhibitors, although novel treatment developments may require reassessment of these goals. Lack of consensus on prophylaxis with bypassing agents and management of mild/moderate cases identifies a need for further research to establish more comprehensive, evidence-based treatment guidance, particularly for those patients who are unable/prefer not to receive non-factor therapies.
尽管血友病治疗取得了进展，但抑制物的形成仍然是一个重大并发症。虽然存在可行的治疗选择，但在适当的标准治疗方法和治疗目标方面存在一些意见分歧。本研究的目的是评估英国针对患有抑制物的儿童和成人血友病患者的护理标准的共识。
采用两轮在线调查进行了一项改良的德尔菲研究。血友病专家指导委员会和已发表的文献为第一轮问卷提供了依据。受邀参与者包括在过去5年中至少治疗过一名患有抑制物的血友病患者的血液科医生、血友病护士和物理治疗师。6点李克特量表问题的共识预先定义为⩾70%的参与者选择1 - 2（不同意）或5 - 6（同意）。
第一轮（n = 41）和第二轮（n = 34）中，分别有46.7%和35.9%的问题达成了共识。在改善生活质量（QoL）以及实现诸如预防出血、消除抑制物和疼痛管理等临床目标的重要性方面达成了共识。对于构成对免疫耐受诱导（ITI）的充分/不充分反应的标准以及应对次优ITI反应的适当因子VIII剂量达成了一致意见。在成人和儿童/青少年的治疗目标、何时提供旁路制剂预防以及预防的期望方面，意见存在差异。对于患有抑制物的轻度/中度患者的适当治疗也缺乏共识。
英国医疗保健专业人员在管理患有抑制物的血友病患者时，在临床目标和ITI的作用方面似乎意见一致，尽管新的治疗进展可能需要重新评估这些目标。在旁路制剂预防和轻度/中度病例管理方面缺乏共识表明需要进一步研究以建立更全面、基于证据的治疗指南，特别是对于那些无法/不愿意接受非因子疗法的患者。
