Habetswallner Francesco, Iorio Raffaele, Annicchiarico Giuseppina, Limongelli Giuseppe, Uccheddu Maria Bonaria, Galeone Carlotta, Mariani Paolo, Mantegazza Renato
Clinical Neurophysiology Unit, Cardarelli Hospital, Naples, Italy.
Dipartimento di Neuroscienze, Università Cattolica del Sacro Cuore, Rome, Italy.
BMJ Open. 2024 Dec 22;14(12):e086225. doi: 10.1136/bmjopen-2024-086225.
To provide comprehensive information on the burden of myasthenia gravis (MG) in Italy, including the unmet needs of patients and several other aspects related to the disease, based on skilled viewpoints of MG experts.
Iterative analysis conducted in accordance with the best practices of the Delphi method, including anonymity, controlled feedback, and statistical stability of consensus.
24 clinicians, 18 public health experts and 4 patient associations experts completed all the Delphi iterations between 18 April and 3 July 2023, for a total of 46 participants from several Italian Regions.
Five areas of investigation related to MG were examined: epidemiology in Italy and characteristics of disease; diagnostic issues and Italian patient journey; unmet needs during the acute and chronic phases of MG; quality of life; public health management of MG. Consensus in the Delphi iterations was defined by both the percentage level of agreement between panellists or the median value of the responses.
We reported a high level of agreement (ie, >66.7% of panellists) on the prevalence and incidence of disease in Italy and on several management issues. A strong impact of MG on the quality of life of patients also emerged. Cross-agreement was achieved among different subgroups of panellists (ie, clinicians, public health experts and patient associations representatives) for most items proposed.
This study provided guidance for educational and practical aspects of MG in Italy, highlighted disease severity and its role on patients' quality of life. A few gaps related to the handling of MG in Italy also emerged.
基于重症肌无力（MG）专家的专业观点，提供有关意大利重症肌无力负担的全面信息，包括患者未满足的需求以及与该疾病相关的其他几个方面。
按照德尔菲法的最佳实践进行迭代分析，包括匿名性、可控反馈和共识的统计稳定性。
24名临床医生、18名公共卫生专家和4名患者协会专家在2023年4月18日至7月3日期间完成了所有德尔菲迭代，共有来自意大利多个地区的46名参与者。
研究了与重症肌无力相关的五个调查领域：意大利的流行病学和疾病特征；诊断问题和意大利患者就医过程；重症肌无力急性和慢性阶段未满足的需求；生活质量；重症肌无力的公共卫生管理。德尔菲迭代中的共识由专家小组成员之间的一致百分比水平或回答的中位数定义。
我们报告了在意大利疾病的患病率和发病率以及几个管理问题上达成了高度一致（即超过66.7%的专家小组成员）。重症肌无力对患者生活质量的强烈影响也显现出来。对于大多数提出的项目，在不同的专家小组成员亚组（即临床医生、公共卫生专家和患者协会代表）之间达成了交叉共识。
本研究为意大利重症肌无力的教育和实践方面提供了指导，强调了疾病的严重性及其对患者生活质量的影响。在意大利重症肌无力的处理方面也出现了一些差距。
