Awareness of individual goals, preferences, and priorities of persons with severe congenital haemophilia A for a tailored shared decision-making approach to liver-directed gene therapy. A practical guideline

IF 6.9 2区 医学 Q1 HEMATOLOGY
Giovanni Di Minno , Gaia Spadarella , Nelson Mauro Maldonato , Natascia De Lucia , Giancarlo Castaman , Raimondo De Cristofaro , Cristina Santoro , Flora Peyvandi , Anna Borrelli , Angelo Lupi , Marco Follino , Gerardo Guerrino , Filomena Morisco , Matteo Di Minno
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引用次数: 0

Abstract

In clinical medicine, shared decision making (SDM) is a well-recognized strategy to enhance engagement of both patients and clinicians in medical decisions. The success of liver-directed gene therapy (GT) to transform severe congenital haemophilia A (HA) from an incurable to a curable disease has launched a shift beyond current standards of treatment. However, GT acceptance remains low in the community of HA persons. We argue for both persons with haemophilia (PWH) and specialists in HA care including clinicians, as needing SDM-oriented educational programs devoted to GT. Here, we provide an ad hoc outline to implement education to SDM and tailor clinician information on GT to individual PWHs. Based on routine key components of SDM: patient priorities; recommendations based on individual risk reduction; adverse effects; drug-drug interactions; alternatives to GT; and ongoing re-assessment of the objectives as risk factors (and individual priorities) change, this approach is finalized to exploit efficacious communication.

了解严重先天性血友病A患者的个人目标、偏好和优先事项,为肝脏定向基因治疗提供量身定制的共同决策方法。实用指南
在临床医学中,共同决策(SDM)是一种公认的策略,可以提高患者和临床医生在医疗决策中的参与度。肝脏导向基因治疗(GT)成功地将严重先天性血友病A (HA)从无法治愈的疾病转变为可治愈的疾病,这已经启动了超越当前治疗标准的转变。然而,医管局人士对GT的接受程度仍然较低。我们认为,无论是血友病患者(PWH)还是医院护理专家(包括临床医生),都需要以SDM为导向的、专门针对GT的教育计划。在这里,我们提供了一个特别的大纲,以实施对SDM的教育,并为个体PWHs量身定制关于GT的临床医生信息。基于SDM的常规关键组成部分:患者优先级;基于减少个人风险的建议;不利影响;药物之间的相互作用;GT的替代方案;随着风险因素(和个人优先级)的变化,对目标进行持续的重新评估,这种方法最终确定为利用有效的沟通。
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来源期刊
Blood Reviews
Blood Reviews 医学-血液学
CiteScore
13.80
自引率
1.40%
发文量
78
期刊介绍: Blood Reviews, a highly regarded international journal, serves as a vital information hub, offering comprehensive evaluations of clinical practices and research insights from esteemed experts. Specially commissioned, peer-reviewed articles authored by leading researchers and practitioners ensure extensive global coverage across all sub-specialties of hematology.
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