Pneumology Department, Health Care Provider of the European Reference Network On Rare Respiratory Diseases (ERN LUNG), Hospital Universitari Vall d'Hebron/Vall d'Hebron Research Institute (VHIR), Vall d'Hebron Barcelona Hospital Campus, Barcelona, Spain.
Institute of Applied Health Research, University of Birmingham, Birmingham, UK.
Respir Res. 2024 Aug 19;25(1):318. doi: 10.1186/s12931-024-02929-5.
Currently, there is conflicting information and guidance on the effective management of Alpha 1 Antitrypsin Deficiency (AATD). Establishing a consensus of assessment and disease management specific to AATD is important for achieving a standardized treatment pathway and for improving patient outcomes. Here, we aim to utilize the Delphi method to establish a European consensus for the assessment and management of patients with severe AATD.
Two rounds of a Delphi survey were completed online by members of the European Alpha-1 Research Collaboration (EARCO). Respondents were asked to indicate their agreement with proposed statements for patients with no respiratory symptoms, stable respiratory disease, and worsening respiratory disease using a Likert scale of 1-7. Levels of agreement between respondents were calculated using a weighted average.
Round 1 of the Delphi survey was sent to 103 members of EARCO and 38/103 (36.9%) pulmonologists from across 15 countries completed all 109 questions. Round 2 was sent to all who completed Round 1 and 36/38 (94.7%) completed all 79 questions. Responses regarding spirometry, body plethysmography, high-resolution computed tomography, and the initiation of augmentation therapy showed little variability among physicians, but there was discordance among other aspects, such as the use of low-dose computed tomography in both a research setting and routine clinical care.
These results provide expert opinions for the assessment and monitoring of patients with severe AATD, which could be used to provide updated recommendations and standardized treatment pathways for patients across Europe.
目前，关于 Alpha 1 抗胰蛋白酶缺乏症（AATD）的有效管理存在相互矛盾的信息和指南。制定针对 AATD 的评估和疾病管理共识对于实现标准化治疗途径和改善患者预后非常重要。在这里，我们旨在利用 Delphi 方法为严重 AATD 患者的评估和管理建立欧洲共识。
通过欧洲 Alpha-1 研究合作组织（EARCO）的成员在线完成了两轮 Delphi 调查。要求受访者使用 1-7 的李克特量表，对无呼吸系统症状、稳定的呼吸系统疾病和呼吸状况恶化的患者的拟议陈述表示同意程度。使用加权平均值计算受访者之间的同意程度。
第一轮 Delphi 调查发送给了 103 名 EARCO 成员，来自 15 个国家的 38/103（36.9%）名肺病专家完成了所有 109 个问题。第二轮调查发送给了所有完成第一轮调查的人，其中 36/38（94.7%）人完成了所有 79 个问题。关于肺量测定、体描法、高分辨率计算机断层扫描和增强治疗开始的回答在医生之间差异很小，但在其他方面存在分歧，例如在研究环境和常规临床护理中使用低剂量计算机断层扫描。
这些结果为严重 AATD 患者的评估和监测提供了专家意见，这可用于为欧洲各地的患者提供更新的建议和标准化的治疗途径。
