Deterioration of quality of life is associated with the exacerbation frequency in individuals with alpha-1-antitrypsin deficiency &amp;ndash; analysis from the German Registry

Bernhard, Nikolas; Lepper, Philipp M.; Vogelmeier, Claus; Seibert, Martina; Wagenpfeil, Stefan; Bals, Robert; Fähndrich, Sebastian

doi:10.2147/copd.s130925

Cited by 8 publications

(5 citation statements)

References 22 publications

(41 reference statements)

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“…The difference likely reflects a combination of the centralisation of patient care and the degree of monitoring carried out at our centre as well as potential improvements in patient care over the intervening 14 years. More recently a study from Germany [ 26 ] also documented a slow decline in SGRQ in AATD (+ 1.2 units for total score) which weakly reflected annual exacerbation rates [ 26 ]. However, that data was self-reported and included unquantified patients with the SZ phenotype and a significant proportion on augmentation therapy, which may have influenced the results.…”

Section: Discussionmentioning

confidence: 99%

Health status decline in α-1 antitrypsin deficiency: a feasible outcome for disease modifying therapies?

et al. 2018

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BackgroundTrials of disease modifying therapies in Chronic Obstructive Pulmonary Disease (COPD) provide challenges for detecting physiological and patient centred outcomes. The purpose of the current study was to monitor decline in health status in Alpha-1 antitrypsin deficiency (AATD) and determine its’ relationship to conventional physiology.MethodsPatients recruited to the UK-AATD database with a median follow up of 7 years (IQR 5–10) were studied to determine annual change in St George’s Respiratory Questionnaire (SGRQ), FEV1, gas transfer and their feasibility of use in future trials.ResultsAnnual decline in SGRQ had a wide range, was greater for patients with established COPD and correlated with decline in FEV1 (p < 0.0001). Total score decline was greater (p < 0.05) for those with accelerated FEV1 decline (median = 1.07 points/year) compared to those without (median = 0.51). Power calculations indicated effective intervention would not achieve MCID for the SGRQ unless the timeframe was extended for up to 8 years. More than 5000 patients/arm would be required for a statistically significant modest effect over 3 years even in those with rapid FEV1 decline.ConclusionDespite AATD being a rapidly declining form of COPD, deterioration in SGRQ was slow consistent with ageing and the chronic nature of disease progression. Power calculations indicate the numbers needed to detect a difference with disease modifying therapies would be prohibitive especially in this rare cause of COPD. These data have important implications for future study design of disease modifying therapies even in COPD not associated with AATD.Electronic supplementary materialThe online version of this article (10.1186/s12931-018-0844-6) contains supplementary material, which is available to authorized users.

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Section: Discussionmentioning

confidence: 99%

Health status decline in α-1 antitrypsin deficiency: a feasible outcome for disease modifying therapies?

et al. 2018

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“…Our review suggests that patients with AATD who have frequent exacerbations (⩾3 per year) or chronic sputum expectoration have a poorer QoL than patients without [74], and that SGRQ scores may be significantly correlated with both exacerbations and dyspnoea [76]. Exacerbations are often associated with long-term sequelae including significant, permanent loss of lung function [26,129,130]. However, exacerbations are random events that are driven by infections and outcomes can vary substantially.…”

Section: Discussionmentioning

confidence: 99%

Disease burden associated with alpha-1 antitrypsin deficiency: systematic and structured literature reviews

Miravitlles

Herepath²,

Priyendu

et al. 2022

Eur Respir Rev

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Alpha-1 antitrypsin deficiency (AATD) is a rare genetic disorder characterised by reduced levels of circulating alpha-1 antitrypsin and an increased risk of lung and liver disease. Recent reviews of AATD have focused on diagnosis, epidemiology and clinical management; comprehensive reviews examining disease burden are lacking. Therefore, we conducted literature reviews to investigate the AATD disease burden for patients, caregivers and healthcare systems. Embase, PubMed and Cochrane libraries were searched for AATD publications from database inception to June 2021, in accordance with Preferred Reporting Items for Systematic Reviews and Meta-Analyses guidelines. Most published AATD studies were small and short in duration, with variations in populations, designs, measures and outcomes, complicating cross-study comparisons. AATD was associated with significant pulmonary and hepatic morbidity. COPD, emphysema and bronchiectasis were common lung morbidities, where smoking was a key risk factor. Fibrosis and steatosis were the most common liver complications reported in patients with a PiZ allele. Health status analyses suggested a poorer quality of life for AATD patients diagnosed with COPD versus those with non-AATD-associated COPD. The burden for caregivers included loss of personal time due to caring responsibilities, stress and anxiety. AATD was also associated with high direct medical costs and healthcare resource utilisation.

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“…Some studies showed that patients with frequent exacerbations had a significantly lower QOL than patients with less frequent exacerbations 63 . Bernhard et al 64 reported that changes in HRQOL were more dependent on the frequency of exacerbation than on FEV1 and DLCO decline. Hospitalization also increased the financial burden and reduced QOL 65 .…”

Section: Discussionmentioning

confidence: 99%

Influencing factors of good quality of life among chronic obstructive pulmonary disease patients living in Zhejiang Province, China

Ying,

Khunthason,

Apidechkul

et al. 2024

Sci Rep

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Chronic obstructive pulmonary disease (COPD) is a chronic, progressive and debilitating disease that affects quality of life (QOL), especially among patients living in poor environments. This study aimed to determine the influencing factors of good QOL among COPD patients living in Zhejiang, China. A cross-sectional study was conducted to collect data from participants in six tertiary hospitals in Zhejiang Province by a simple random sampling method. A validated questionnaire was used to collect general information, environmental factors, and COPD stage. The standardized St. George's Respiratory Questionnaire (SGRQ) was used to assess QOL. Logistic regression was used to determine influencing factors of good QOL among COPD patients at a significance level of α = 0.05. A total of 420 participants were recruited for analysis. The overall prevalence of patients with good QOL was 25.7%. Six variables were found to be associated with good QOL in the multivariable analysis. Patients who were employed had 2.35 times (95% CI 1.03–5.34) greater odds of having good QOL than those who were unemployed. Those whose family income was higher than 100,000 CNY had 2.49 times (95% CI 1.15–5.39) greater odds of having good QOL than those whose family income was lower than 100,000 CNY. Those who had treatment expenses less than 5,000 CNY had 4.57 (95% CI 1.57–13.30) times greater odds of having good QOL than those who had treatment expenses of 5,000 CNY or higher. Those who had mild or moderate airflow limitation were 5.27 times (95% CI 1.61–17.26) more likely to have good QOL than those who were in a severe or very severe stage of COPD. Those who had a duration of illness less than 60 months had 5.57 times (95% CI 1.40–22.12) greater odds of having good QOL than those who had a duration of illness of 120 months or more. Those who were not hospitalized within the past 3 months had 9.39 times (95% CI 1.62–54.43) greater odds of having good QOL than those who were hospitalized more than twice over the past 3 months. Socioeconomic status, disease stage and accessibility were associated with good QOL among COPD patients in Zhejiang Province, China. Increasing family income and implementing measures to improve the accessibility of medical care, including developing a proper system to decrease the cost of treatment for COPD patients, can improve patients’ QOL.

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Deterioration of quality of life is associated with the exacerbation frequency in individuals with alpha-1-antitrypsin deficiency – analysis from the German Registry

Cited by 8 publications

References 22 publications

Health status decline in α-1 antitrypsin deficiency: a feasible outcome for disease modifying therapies?

Health status decline in α-1 antitrypsin deficiency: a feasible outcome for disease modifying therapies?

Disease burden associated with alpha-1 antitrypsin deficiency: systematic and structured literature reviews

Influencing factors of good quality of life among chronic obstructive pulmonary disease patients living in Zhejiang Province, China

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