背景:Pegvaliase,酶替代疗法,是苯丙酮尿症(PKU)的治疗选择。由于PKU的神经病理生理和疾病负担,个体可经历与PEGvaliase治疗无关的基线焦虑.此外,对于正在考虑或接受治疗的患者,聚乙二醇酶治疗的某些方面可能会引起焦虑.本手稿的目的是提出最佳实践建议,以识别和管理可能发生在PEGvaliase旅程中的焦虑症状。
方法:采用改良的Delphi方法在多学科专家小组中寻求共识。为此,我们举行了一次面对面的会议,随后进行了一项针对医学专家和患者的调查,就如何解决pegvaliase旅程中的焦虑提出初步建议.会后,进行了另一项调查,对拟议的解决方案和缓解策略进行排名,并从中制定了一套建议。对所有建议进行了表决,目的是达成共识,定义为专家达到≥75%的一致性。
结果:专家组就治疗前焦虑管理的28项最佳实践建议达成共识,感应和滴定,早期维护(前功效),和后期维持(疗效后)阶段。这些建议提供了识别和解决与pegvaliase相关的焦虑的最常见原因的策略。包括自我注射,副作用,滴定时间表,规定的饮食变化,和可变的见效时间。总的来说,在考虑或接受pegvaliase的人中管理焦虑涉及以患者为中心的沟通,共同决策,个性化的治疗方案。
结论:本文描述的最佳实践建议可以指导医疗保健提供者在pegvaliase治疗的不同阶段积极解决焦虑,并支持提供者在可能经历基线和治疗相关焦虑的个体中启动和管理pegvaliase。
BACKGROUND: Pegvaliase, an enzyme substitution therapy, is a treatment option for phenylketonuria (PKU). Due to the neuropathophysiology and disease burden of PKU, individuals can experience baseline anxiety unrelated to pegvaliase therapy. In addition, there are aspects of pegvaliase therapy that may be anxiety-inducing for those considering or receiving treatment. The aim of this manuscript is to present best practice recommendations for the identification and management of anxiety symptoms that can occur along the pegvaliase journey.
METHODS: A modified Delphi approach was used to seek consensus among a multidisciplinary panel of experts. To this end, an in-person meeting was held that was preceded by a medical specialist- and patient-specific survey to develop preliminary recommendations on ways to address anxiety along the pegvaliase journey. After the meeting, an additional survey was conducted to rank the proposed solutions and mitigation strategies from which a set of recommendations was developed. All recommendations were voted on with the aim of consensus generation, defined as achieving ≥75% agreement among experts.
RESULTS: The panel reached consensus on a total of 28 best practice recommendations for the management of anxiety during the pre-treatment, induction and titration, early maintenance (pre-efficacy), and late maintenance (post-efficacy) stages. The recommendations offer strategies to identify and address the most common causes of pegvaliase-related anxiety, including self-injection, side effects, the titration schedule, prescribed dietary changes, and variable time to efficacy. Overall, managing anxiety in those considering or receiving pegvaliase involves patient-centered communication, shared decision-making, and personalized treatment plans.
CONCLUSIONS: The best practice recommendations described herein can guide healthcare providers in proactively addressing anxiety during the different stages of pegvaliase treatment, and support providers with initiating and managing pegvaliase in individuals who may experience baseline and treatment-related anxiety.