The Effect of Alpha-1 Proteinase Inhibitor on Biomarkers of Elastin Degradation in Alpha-1 Antitrypsin Deficiency: An Analysis of the RAPID/RAPID Extension Trials

Ma, Shuren; Lin, Yong Y.; Cantor, Jerome O.; Chapman, Kenneth R.; Sandhaus, Robert A.; Fries, Michael; Edelman, Jonathan M.; McElvaney, N.G.; Turino, Gerard M.

doi:10.15326/jcopdf.4.1.2016.0156

Cited by 45 publications

(38 citation statements)

References 27 publications

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“…A supplementary report to the trial has also detailed reduced circulating desmosine, indicating an effect of augmentation on body elastin breakdown [109].…”

Section: Augmentation Therapy For Aatdmentioning

confidence: 99%

European Respiratory Society statement: diagnosis and treatment of pulmonary disease in α₁-antitrypsin deficiency

et al. 2017

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α-antitrypsin deficiency (AATD) is the most common hereditary disorder in adults. It is associated with an increased risk of developing pulmonary emphysema and liver disease. The pulmonary emphysema in AATD is strongly linked to smoking, but even a proportion of never-smokers develop progressive lung disease. A large proportion of individuals affected remain undiagnosed and therefore without access to appropriate care and treatment.The most recent international statement on AATD was published by the American Thoracic Society and the European Respiratory Society in 2003. Since then there has been a continuous development of novel, more accurate and less expensive genetic diagnostic methods. Furthermore, new outcome parameters have been developed and validated for use in clinical trials and a new series of observational and randomised clinical trials have provided more evidence concerning the efficacy and safety of augmentation therapy, the only specific treatment available for the pulmonary disease associated with AATD.As AATD is a rare disease, it is crucial to organise national and international registries and collect information prospectively about the natural history of the disease. Management of AATD patients must be supervised by national or regional expert centres and inequalities in access to therapies across Europe should be addressed.

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“…A supplementary report to the trial has also detailed reduced circulating desmosine, indicating an effect of augmentation on body elastin breakdown [109].…”

Section: Augmentation Therapy For Aatdmentioning

confidence: 99%

European Respiratory Society statement: diagnosis and treatment of pulmonary disease in α₁-antitrypsin deficiency

et al. 2017

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“…The RAPID trials support the efficacy of augmentation therapy in slowing disease progression during 4 years of treatment and, as lost lung density during placebo treatment never recovered following augmentation therapy in the delayed‐start group, the trials highlight the importance of early initiation of augmentation therapy (Figure ). In a post hoc analysis of the RAPID trials, treatment with AAT was also associated with reduced elastin degradation as evident by biomarkers isodesmosine and desmosine (Figure ) …”

Section: Current Treatmentsmentioning

confidence: 99%

“…A1PI, α 1 proteinase inhibitor; DES/IDES, desmosine/isodesmosine; n, number of patients. Figure used with permission from Chronic Obstructive Pulmonary Diseases: Journal of the COPD Foundation…”

Section: Current Treatmentsmentioning

confidence: 99%

Advances in managing COPD related to α₁‐antitrypsin deficiency: An under‐recognized genetic disorder

Craig

Henao

2018

Allergy

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Abstractα1‐Antitrypsin deficiency (AATD) predisposes individuals to chronic obstructive pulmonary disease (COPD) and liver disease. Despite being commonly described as rare, AATD is under‐recognized, with less than 10% of cases identified. The following is a comprehensive review of AATD, primarily for physicians who treat COPD or asthma, covering the genetics, epidemiology, clinical presentation, screening and diagnosis, and treatments of AATD. For patients presenting with liver and/or lung disease, screening and diagnostic tests are the only methods to determine whether the disease is related to AATD. Screening guidelines have been established by organizations such as the World Health Organization, European Respiratory Society, and American Thoracic Society. High‐risk groups, including individuals with COPD, nonresponsive asthma, bronchiectasis of unknown etiology, or unexplained liver disease, should be tested for AATD. Current treatment options include augmentation therapy with purified AAT for patients with deficient AAT levels and significant lung disease. Recent trial data suggest that lung tissue is preserved by augmentation therapy, and different dosing schedules are currently being investigated. Effective management of AATD and related diseases also includes aggressive avoidance of smoking and biomass burning, vaccinations, antibiotics, exercise, good diet, COPD medications, and serial assessment.

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“…Однако как и в предыдущих исследованиях в этой области, достоверного влияния лечения на другие конечные показатели, такие как легочная функция и КЖ, не выявлено [87]. В дополнительной информации об исследованиях также подробно описано снижение циркулирующего десмосина, что свидетельствует о положительном влиянии ЗТ на процесс разрушения эластина [109].…”

Section: таблица 3 исследования заместительной терапии при дефиците αunclassified

Diagnosis and treatment of pulmonarydisease in α1-antitrypsin deficiency: a statement of European Respiratory Society

Редакционная¹

2018

Pulʹmonologiâ (Mosk.)

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Alfa-1-antitrypsin deficiency (AATD) is the most common hereditary disorder in adults. It is associated with an increased risk of developing pulmonary emphysema and liver disease. The lung injury in AATD is closely associated with smoking, but progressive lung disease could occur even in never-smokers. A number of individuals with AATD remain undiagnosed and therefore do not receive appropriate care and treatment. The most recent international document on AATD was the joint statement of the American Thoracic Society and the European Respiratory Society published in 2003. Thereafter, there has been a continuous development of novel, more accurate and less expensive genetic diagnostic methods. Furthermore, new outcome parameters have been developed and validated for use in clinical trials and a new series of observational and randomized clinical trials have provided more evidence concerning the efficacy and safety of augmentation therapy, the only specific treatment available for the pulmonary disease associated with AATD. As AATD is a rare disease, it is important to createnational and international registries and to collect information prospectively about the natural history of the disease. Management of AATD patients must be supervised by national or regional expert centres and inequalities in access to therapies across Europe should be addressed.

show abstract

The Effect of Alpha-1 Proteinase Inhibitor on Biomarkers of Elastin Degradation in Alpha-1 Antitrypsin Deficiency: An Analysis of the RAPID/RAPID Extension Trials

Cited by 45 publications

References 27 publications

European Respiratory Society statement: diagnosis and treatment of pulmonary disease in α₁-antitrypsin deficiency

European Respiratory Society statement: diagnosis and treatment of pulmonary disease in α₁-antitrypsin deficiency

Advances in managing COPD related to α₁‐antitrypsin deficiency: An under‐recognized genetic disorder

Diagnosis and treatment of pulmonarydisease in α1-antitrypsin deficiency: a statement of European Respiratory Society

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The Effect of Alpha-1 Proteinase Inhibitor on Biomarkers of Elastin Degradation in Alpha-1 Antitrypsin Deficiency: An Analysis of the RAPID/RAPID Extension Trials

Cited by 45 publications

References 27 publications

European Respiratory Society statement: diagnosis and treatment of pulmonary disease in α1-antitrypsin deficiency

European Respiratory Society statement: diagnosis and treatment of pulmonary disease in α1-antitrypsin deficiency

Advances in managing COPD related to α1‐antitrypsin deficiency: An under‐recognized genetic disorder

Diagnosis and treatment of pulmonarydisease in α1-antitrypsin deficiency: a statement of European Respiratory Society

Contact Info

Product

Resources

About

European Respiratory Society statement: diagnosis and treatment of pulmonary disease in α₁-antitrypsin deficiency

European Respiratory Society statement: diagnosis and treatment of pulmonary disease in α₁-antitrypsin deficiency

Advances in managing COPD related to α₁‐antitrypsin deficiency: An under‐recognized genetic disorder