Abstract：
BACKGROUND: Exacerbations are common in individuals with alpha-1 antitrypsin deficiency (AATD)-related lung disease. This study intended to identify independent predictive factors for exacerbations in AATD using the Portuguese European Alpha-1 Research Collaboration (EARCO) registry.
METHODS: This study includes patients from the Portuguese EARCO registry, a prospective multicenter cohort (NCT04180319). From October 2020 to April 2023, this registry enrolled 137 patients, 14 of whom were excluded for analysis for either missing 12 months of follow-up or baseline pulmonary function.
RESULTS: Among the 123 AATD patients, 27 (22.0%) had at least one exacerbation in the last 12 months of follow-up. Patients with Pi*ZZ phenotype were three times more likely than the rest of the population to experience any exacerbation (32.7 vs. 14.1%, p = 0.014; OR 3.0). BODE index was significantly higher in exacerbators than in non-exacerbators (3.9 ± 2.4 vs. 1.3 ± 1.2; p < 0.001), including on multivariate analysis (p = 0.002). Similar results were found for BODEx (multivariate p < 0.001). DLCO was the only functional parameter independently associated with exacerbations (p = 0.024).
CONCLUSIONS: DLCO, BODE, and BODEx were independent predictors of exacerbations at 12 months in AATD patients. Understanding these risk factors can aid decision-making on AATD-related lung disease management and improve patient outcomes.
METHODS: This study includes patients from the Portuguese EARCO registry, a prospective multicenter cohort (NCT04180319). From October 2020 to April 2023, this registry enrolled 137 patients, 14 of whom were excluded for analysis for either missing 12 months of follow-up or baseline pulmonary function.
RESULTS: Among the 123 AATD patients, 27 (22.0%) had at least one exacerbation in the last 12 months of follow-up. Patients with Pi*ZZ phenotype were three times more likely than the rest of the population to experience any exacerbation (32.7 vs. 14.1%, p = 0.014; OR 3.0). BODE index was significantly higher in exacerbators than in non-exacerbators (3.9 ± 2.4 vs. 1.3 ± 1.2; p < 0.001), including on multivariate analysis (p = 0.002). Similar results were found for BODEx (multivariate p < 0.001). DLCO was the only functional parameter independently associated with exacerbations (p = 0.024).
CONCLUSIONS: DLCO, BODE, and BODEx were independent predictors of exacerbations at 12 months in AATD patients. Understanding these risk factors can aid decision-making on AATD-related lung disease management and improve patient outcomes.
摘要：
背景：急性加重在α-1抗胰蛋白酶缺乏症(AATD)相关肺病患者中很常见。这项研究旨在使用葡萄牙欧洲Alpha-1研究合作组织（EARCO）注册表确定AATD恶化的独立预测因素。
方法：本研究包括来自葡萄牙EARCO注册的患者，前瞻性多中心队列（NCT04180319）。从2020年10月到2023年4月，该注册登记了137名患者，其中14人因缺少12个月的随访或基线肺功能而被排除在分析之外。
结果：在123名AATD患者中，27人（22.0%）在最后12个月的随访中至少有一次加重。Pi*ZZ表型患者经历任何恶化的可能性是其他人群的三倍(32.7vs.14.1%,p=0.014；或3.0）。比较者的BODE指数明显高于非比较者（3.9±2.4vs.1.3±1.2；p<0.001)，包括多变量分析(p=0.002)。对于BODEx也发现了类似的结果(多变量p<0.001)。DLCO是唯一与急性加重独立相关的功能参数(p=0.024)。
结论：DLCO，BODE,和BODEx是AATD患者12个月时急性加重的独立预测因子。了解这些风险因素可以帮助AATD相关肺部疾病管理的决策并改善患者预后。
方法：本研究包括来自葡萄牙EARCO注册的患者，前瞻性多中心队列（NCT04180319）。从2020年10月到2023年4月，该注册登记了137名患者，其中14人因缺少12个月的随访或基线肺功能而被排除在分析之外。
结果：在123名AATD患者中，27人（22.0%）在最后12个月的随访中至少有一次加重。Pi*ZZ表型患者经历任何恶化的可能性是其他人群的三倍(32.7vs.14.1%,p=0.014；或3.0）。比较者的BODE指数明显高于非比较者（3.9±2.4vs.1.3±1.2；p<0.001)，包括多变量分析(p=0.002)。对于BODEx也发现了类似的结果(多变量p<0.001)。DLCO是唯一与急性加重独立相关的功能参数(p=0.024)。
结论：DLCO，BODE,和BODEx是AATD患者12个月时急性加重的独立预测因子。了解这些风险因素可以帮助AATD相关肺部疾病管理的决策并改善患者预后。
