Abstract：
BACKGROUND: Regular and consistent disease assessment could provide a clearer picture of burden in generalised myasthenia gravis (gMG) and improve patient care; however, the use of assessment tools in practice lacks standardisation. This modified Delphi approach was taken to review current evidence on assessment tool use in gMG and develop expert-derived consensus recommendations for good practice.
METHODS: A European expert panel of 15 experienced gMG neurologists contributed to development of this consensus, four of whom formed a lead Sub-committee. The PICO (Population, Intervention, Control, Outcomes) framework was used to define six clinical questions on gMG assessment tools, a systematic literature review was conducted, and evidence-based statements were developed. According to a modified Delphi voting process, consensus was reached when ≥70% of the experts rated agreement with a statement as ≥8 on a scale of 1-10.
RESULTS: Eighteen expert- and evidence-based consensus statements based on six themes were developed. Key recommendations include: consistent use of the Myasthenia Gravis Activities of Daily Living score (MG-ADL) across clinical settings, followed by a simple question (e.g., Patient Acceptable Symptom State [PASS]) or scale to determine patient satisfaction in clinical practice; use of a Quantitative Myasthenia Gravis [QMG] or quality of life [QoL] assessment when the MG-ADL indicates disease worsening; and consideration of symptom state to determine the timing and frequency of recommended assessments. Expert panel consensus was reached on all 18 statements after two voting rounds.
CONCLUSIONS: This process provided evidence- and expert consensus-based recommendations for the use of objective and subjective assessment tools across gMG research and care to improve management and outcomes for patients.
METHODS: A European expert panel of 15 experienced gMG neurologists contributed to development of this consensus, four of whom formed a lead Sub-committee. The PICO (Population, Intervention, Control, Outcomes) framework was used to define six clinical questions on gMG assessment tools, a systematic literature review was conducted, and evidence-based statements were developed. According to a modified Delphi voting process, consensus was reached when ≥70% of the experts rated agreement with a statement as ≥8 on a scale of 1-10.
RESULTS: Eighteen expert- and evidence-based consensus statements based on six themes were developed. Key recommendations include: consistent use of the Myasthenia Gravis Activities of Daily Living score (MG-ADL) across clinical settings, followed by a simple question (e.g., Patient Acceptable Symptom State [PASS]) or scale to determine patient satisfaction in clinical practice; use of a Quantitative Myasthenia Gravis [QMG] or quality of life [QoL] assessment when the MG-ADL indicates disease worsening; and consideration of symptom state to determine the timing and frequency of recommended assessments. Expert panel consensus was reached on all 18 statements after two voting rounds.
CONCLUSIONS: This process provided evidence- and expert consensus-based recommendations for the use of objective and subjective assessment tools across gMG research and care to improve management and outcomes for patients.
摘要：
背景：定期和一致的疾病评估可以更清楚地了解广泛性重症肌无力（gMG）的负担，并改善患者护理；然而，评估工具在实践中的使用缺乏标准化。采用这种改进的Delphi方法来审查gMG中评估工具使用的当前证据，并为良好实践制定专家得出的共识建议。
方法：由15名经验丰富的gMG神经科医师组成的欧洲专家小组为这一共识的发展做出了贡献。其中四人组成了一个牵头小组委员会。PICO（人口，干预,Control,结果)框架用于定义gMG评估工具上的六个临床问题，进行了系统的文献综述，并制定了基于证据的陈述。根据修改后的德尔福投票程序，当≥70%的专家在1-10的评分范围内对陈述的一致性评分为≥8时,达成共识.
结果：根据六个主题制定了18项基于专家和证据的共识声明。主要建议包括：在临床环境中一致使用重症肌无力日常生活活动评分（MG-ADL），后跟一个简单的问题(例如，患者可接受的症状状态[PASS]）或量表以确定患者在临床实践中的满意度；当MG-ADL表明疾病恶化时，使用定量重症肌无力[QMG]或生活质量[QoL]评估；并考虑症状状态以确定推荐评估的时间和频率。经过两轮投票，专家小组就所有18项声明达成了共识。
结论：该过程为gMG研究和护理中使用客观和主观评估工具提供了基于证据和专家共识的建议，以改善患者的管理和预后。
方法：由15名经验丰富的gMG神经科医师组成的欧洲专家小组为这一共识的发展做出了贡献。其中四人组成了一个牵头小组委员会。PICO（人口，干预,Control,结果)框架用于定义gMG评估工具上的六个临床问题，进行了系统的文献综述，并制定了基于证据的陈述。根据修改后的德尔福投票程序，当≥70%的专家在1-10的评分范围内对陈述的一致性评分为≥8时,达成共识.
结果：根据六个主题制定了18项基于专家和证据的共识声明。主要建议包括：在临床环境中一致使用重症肌无力日常生活活动评分（MG-ADL），后跟一个简单的问题(例如，患者可接受的症状状态[PASS]）或量表以确定患者在临床实践中的满意度；当MG-ADL表明疾病恶化时，使用定量重症肌无力[QMG]或生活质量[QoL]评估；并考虑症状状态以确定推荐评估的时间和频率。经过两轮投票，专家小组就所有18项声明达成了共识。
结论：该过程为gMG研究和护理中使用客观和主观评估工具提供了基于证据和专家共识的建议，以改善患者的管理和预后。
