Background: Health-related quality of life (HRQoL) assessments such as St. George’s Respiratory Questionnaire (SGRQ) are often used as outcome measures to evaluate patient-perceived changes in health status among individuals with lung disease. Several factors have been linked to deterioration in SGRQ, including symptoms (dyspnea, wheezing) and exercise intolerance. Whether these findings apply to individuals with alpha-1 antitrypsin deficiency (AATD) remains incompletely studied. This longitudinal study examines the trajectory of SGRQ scores in a cohort of United States individuals with AATD-associated lung disease and defines factors associated with longitudinal change.
Methods: Individuals with AATD-associated lung disease enrolled in AlphaNet, a disease management program, who had ≥ 3 SGRQ measurements collected between 2009 and 2019, and baseline data for clinically important variables were included in these analyses. Data collected after lung transplants were excluded. Mixed-effects model analyses were used to evaluate the changes in SGRQ total and subscale scores over time and by modified Medical Research Council (mMRC) Scale, use of oxygen, age, sex, productive cough, and exacerbation frequency at baseline. Sensitivity analyses were conducted to examine the potential effect of survivor bias.
Results: Participants (n=2456, mean age 57.1± 9.9 years, 47% female) had a mean SGRQ total score of 44.7± 18.9 at baseline, 48% used oxygen regularly, and 55% had ≥ 2 exacerbations per year. The median length of follow-up was 6 (IQR 3– 9) years. The SGRQ total score and subscales remained stable throughout the observation period. Age, mMRC categories, presence or absence of productive cough, frequency of exacerbations, and use of oxygen at baseline were significantly associated with the rate of change of SGRQ total (p< 0.0001).
Conclusion: We observed long-term stability in HRQoL and an association between the rate of change in SGRQ and baseline mMRC, exacerbation frequency, productive cough, and use of oxygen in this cohort of individuals with AATD-associated lung disease.



中文翻译：

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患有 Alpha-1 抗胰蛋白酶缺乏相关肺病的个体群体中 SGRQ 的长期稳定性

背景：与健康相关的生活质量（HRQoL）评估，例如圣乔治呼吸问卷（SGRQ），通常被用作评估肺病患者感知的健康状况变化的结果指标。有几个因素与 SGRQ 恶化有关，包括症状（呼吸困难、喘息）和运动不耐受。这些发现是否适用于 α-1 抗胰蛋白酶缺乏症 (AATD) 患者仍有待完全研究。这项纵向研究调查了一组患有 AATD 相关肺病的美国个体的 SGRQ 评分轨迹，并定义了与纵向变化相关的因素。
方法：参加疾病管理项目 AlphaNet 的患有 AATD 相关肺部疾病的个体，在 2009 年至 2019 年间收集了 ≥ 3 次 SGRQ 测量值，并且临床重要变量的基线数据纳入这些分析中。肺移植后收集的数据被排除在外。使用混合效应模型分析来评估 SGRQ 总分和子量表分数随时间的变化，并通过修改后的医学研究委员会 (mMRC) 量表、氧气的使用、年龄、性别、咳嗽咳痰和基线恶化频率来评估。进行敏感性分析以检验幸存者偏差的潜在影响。
结果：参与者（n=2456，平均年龄 57.1±9.9 岁，47% 为女性）基线时的平均 SGRQ 总分为 44.7±18.9，48% 定期使用氧气，55% 每年有 ≥ 2 次病情加重。中位随访时间为 6 (IQR 3-9) 年。 SGRQ 总分和分量表在整个观察期间保持稳定。年龄、mMRC 类别、是否存在咳痰、病情加重频率以及基线时氧气的使用与 SGRQ 总量的变化率显着相关（p < 0.0001）。
结论：我们在这组患有 AATD 相关肺部疾病的个体中观察到 HRQoL 的长期稳定性以及 SGRQ 变化率和基线 mMRC、恶化频率、咳嗽和吸氧之间的关联。