背景:目前的文献对α-甘露糖苷酶(AM)患者的初始评估和常规随访治疗缺乏共识。进行了Delphi小组,以生成和验证有关初始评估最佳实践的建议,常规随访护理,和AM患者的综合护理协调。
方法:使用了涉及3轮在线调查的改良Delphi方法。一名独立管理员和两名无表决权的医生共同主席管理调查开发,匿名数据收集,和分析。由来自12个国家的20名医生组成的多学科小组在第一次调查中回答了57个开放式问题。第二轮包括11个排名问题和44个投票声明。在第3轮中,小组成员投票通过了60项共识声明。在所有3个回合中,小组反应率≥95%。小组成员使用5分Likert量表来表示重要性(评分≥3)或一致性(评分≥4)。共识先验定义为≥75%的同意与≥75%的小组成员投票。
结果:就60项声明达成共识,涵盖3个关键领域:初步评估,常规随访护理,和治疗相关的随访。小组就基因检测评估的类型和频率达成一致,基线评估,生活质量,生化措施,受影响的身体系统,接受治疗,以及AM患者的综合护理协调。49份声明达成了90%到100%的共识,8份声明达成了80%到85%的共识,一份声明达成了75%的共识。两项声明各自就15项基线评估达成共识,将在儿科和成人患者诊断后的初次随访时进行。
结论:这是第一个Delphi研究,监测AM患者的最佳实践建议,可以改善他们的护理和健康。
BACKGROUND: Current literature lacks consensus on initial assessments and routine follow-up care of patients with alpha-mannosidosis (AM). A Delphi panel was conducted to generate and validate recommendations on best practices for initial assessment, routine follow-up care, and integrated care coordination of patients with AM.
METHODS: A modified Delphi method involving 3 rounds of online surveys was used. An independent administrator and 2 nonvoting physician co-chairs managed survey development, anonymous data collection, and analysis. A multidisciplinary panel comprising 20 physicians from 12 countries responded to 57 open-ended questions in the first survey. Round 2 consisted of 11 ranking questions and 44 voting statements. In round 3, panelists voted to validate 60 consensus statements. The panel response rate was ≥95% in all 3 rounds. Panelists used 5-point Likert scales to indicate importance (score of ≥3) or agreement (score of ≥4). Consensus was defined a priori as ≥75% agreement with ≥75% of panelists voting.
RESULTS: Consensus was reached on 60 statements, encompassing 3 key areas: initial assessments, routine follow-up care, and treatment-related follow-up. The panel agreed on the type and frequency of assessments related to genetic testing, baseline evaluations, quality of life, biochemical measures, affected body systems, treatment received, and integrated care coordination in patients with AM. Forty-nine statements reached 90% to 100% consensus, 8 statements reached 80% to 85% consensus, and 1 statement reached 75% consensus. Two statements each reached consensus on 15 baseline assessments to be conducted at the initial follow-up visit after diagnosis in pediatric and adult patients.
CONCLUSIONS: This is the first Delphi study providing internationally applicable, best-practice recommendations for monitoring patients with AM that may improve their care and well-being.