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Volumn 19, Issue 1, 2018, Pages

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

Author keywords

Alpha 1 antitrypsin deficiency; Disease progression; Health related quality of life

Indexed keywords

ADULT; ALPHA 1 ANTITRYPSIN DEFICIENCY; ARTICLE; CHRONIC OBSTRUCTIVE LUNG DISEASE; COHORT ANALYSIS; CONTROLLED STUDY; DETERIORATION; DISEASE ASSOCIATION; DISEASE COURSE; EFFECT SIZE; FEASIBILITY STUDY; FEMALE; FOLLOW UP; FORCED EXPIRATORY VOLUME; FORCED VITAL CAPACITY; HEALTH STATUS; HUMAN; MALE; ST. GEORGE RESPIRATORY QUESTIONNAIRE; AGED; CROSS-SECTIONAL STUDY; DISEASE EXACERBATION; MIDDLE AGED; REGISTER; TREATMENT OUTCOME; VERY ELDERLY;

EID: 85050284592     PISSN: 14659921     EISSN: 1465993X     Source Type: Journal    
DOI: 10.1186/s12931-018-0844-6     Document Type: Article
Times cited : (14)

References (35)
  • 1
    • 80053350305 scopus 로고    scopus 로고
    • Changes in forced expiratory volume in 1 second over time in COPD
    • Vestbo J, Edwards LD, Scanlon PD, Yates JC, Agusti A, et al. Changes in forced expiratory volume in 1 second over time in COPD. N Engl J Med. 2011;365(13):1184-92.
    • (2011) N Engl J Med , vol.365 , Issue.13 , pp. 1184-1192
    • Vestbo, J.1    Edwards, L.D.2    Scanlon, P.D.3    Yates, J.C.4    Agusti, A.5
  • 2
    • 84855280191 scopus 로고    scopus 로고
    • Annual change in pulmonary function and clinical phenotype in chronic obstructive pulmonary disease
    • Nishimura M, Makita H, Nagai K, Konno S, Nasuhara Y, et al. Annual change in pulmonary function and clinical phenotype in chronic obstructive pulmonary disease. Am J Respir Crit Care Med. 2012;185(1):44-52.
    • (2012) Am J Respir Crit Care Med , vol.185 , Issue.1 , pp. 44-52
    • Nishimura, M.1    Makita, H.2    Nagai, K.3    Konno, S.4    Nasuhara, Y.5
  • 3
    • 84890541399 scopus 로고    scopus 로고
    • Club cell protein 16 and disease progression in chronic obstructive pulmonary disease
    • Park HY, Churg A, Wright JL, Li Y, Tam S, et al. Club cell protein 16 and disease progression in chronic obstructive pulmonary disease. Am J Respir Crit Care Med. 2013;188(12):1413-9.
    • (2013) Am J Respir Crit Care Med , vol.188 , Issue.12 , pp. 1413-1419
    • Park, H.Y.1    Churg, A.2    Wright, J.L.3    Li, Y.4    Tam, S.5
  • 5
    • 0034889702 scopus 로고    scopus 로고
    • High-resolution computed tomography scanning in alpha1-antitrypsin deficiency: relationship to lung function and health status
    • Dowson LJ, Guest PJ, Hill SL, Holder RL, Stockley RA. High-resolution computed tomography scanning in alpha1-antitrypsin deficiency: relationship to lung function and health status. Eur Respir J. 2001;17(6):1097-104.
    • (2001) Eur Respir J , vol.17 , Issue.6 , pp. 1097-1104
    • Dowson, L.J.1    Guest, P.J.2    Hill, S.L.3    Holder, R.L.4    Stockley, R.A.5
  • 6
    • 0031746535 scopus 로고    scopus 로고
    • Effect of exacerbation on quality of life in patients with chronic obstructive pulmonary disease
    • Seemungal TA, Donaldson GC, Paul EA, Bestall JC, Jeffries DJ, et al. Effect of exacerbation on quality of life in patients with chronic obstructive pulmonary disease. Am J Respir Crit Care Med. 1998;157(5):1418-22.
    • (1998) Am J Respir Crit Care Med , vol.157 , Issue.5 , pp. 1418-1422
    • Seemungal, T.A.1    Donaldson, G.C.2    Paul, E.A.3    Bestall, J.C.4    Jeffries, D.J.5
  • 7
    • 85007475300 scopus 로고    scopus 로고
    • Chronic Obstructive Pulmonary Disease: Developing Drugs for Treatment Guidance for Industry (Draft Guidance)
    • U.S. Food and Drug Administration. Chronic Obstructive Pulmonary Disease: Developing Drugs for Treatment Guidance for Industry (Draft Guidance). 2016 [Available from: http://www.fda.gov/downloads/Drugs/GuidanceComplianceRegulatoryInformation/Guidances/UCM071575.pdf.
    • (2016)
  • 8
    • 85050279408 scopus 로고    scopus 로고
    • Department of Health and Human Services FaDA, Center for Drug Evaluation and Research (CDER)
    • U.S, Chronic Obstructive Pulmonary Disease: Use of the St. George's Respiratory Questionnaire as a PRO Assessment Tool Guidance for Industry
    • U.S. Food and Drug Administration. In: U.S. Department of Health and Human Services FaDA, Center for Drug Evaluation and Research (CDER), editor. Chronic Obstructive Pulmonary Disease: Use of the St. George's Respiratory Questionnaire as a PRO Assessment Tool Guidance for Industry; 2018.
    • (2018)
  • 9
    • 48949103222 scopus 로고    scopus 로고
    • Effect of pharmacotherapy on rate of decline of lung function in chronic obstructive pulmonary disease: results from the TORCH study
    • Celli BR, Thomas NE, Anderson JA, Ferguson GT, Jenkins CR, et al. Effect of pharmacotherapy on rate of decline of lung function in chronic obstructive pulmonary disease: results from the TORCH study. Am J Respir Crit Care Med. 2008;178(4):332-8.
    • (2008) Am J Respir Crit Care Med , vol.178 , Issue.4 , pp. 332-338
    • Celli, B.R.1    Thomas, N.E.2    Anderson, J.A.3    Ferguson, G.T.4    Jenkins, C.R.5
  • 10
    • 84968779698 scopus 로고    scopus 로고
    • Fluticasone furoate and vilanterol and survival in chronic obstructive pulmonary disease with heightened cardiovascular risk (SUMMIT): a double-blind randomised controlled trial
    • 10030.
    • Vestbo J, Anderson JA, Brook RD, Calverley PM, Celli BR, et al. Fluticasone furoate and vilanterol and survival in chronic obstructive pulmonary disease with heightened cardiovascular risk (SUMMIT): a double-blind randomised controlled trial. Lancet. 2016;387(10030):1817-26.
    • (2016) Lancet , vol.387 , pp. 1817-1826
    • Vestbo, J.1    Anderson, J.A.2    Brook, R.D.3    Calverley, P.M.4    Celli, B.R.5
  • 12
    • 85050305632 scopus 로고    scopus 로고
    • Clinical and Surrogate Endpoints for Evaluating Efficacy of Alpha 1-Proteinase Inhibitor (Human) Augmentation Therapy: FDA Blood Products Advisory Committee; 2009
    • Plasma Protein Therapeutics Association. Clinical and Surrogate Endpoints for Evaluating Efficacy of Alpha 1-Proteinase Inhibitor (Human) Augmentation Therapy: FDA Blood Products Advisory Committee; 2009. [Available from: https://www.pptaglobal.org/./954-ppta-statement-on-clinical-and-surrogate-endpoints.
  • 13
    • 84937969384 scopus 로고    scopus 로고
    • Intravenous augmentation treatment and lung density in severe alpha1 antitrypsin deficiency (RAPID): a randomised, double-blind, placebo-controlled trial
    • Chapman KR, Burdon JG, Piitulainen E, Sandhaus RA, Seersholm N, et al. Intravenous augmentation treatment and lung density in severe alpha1 antitrypsin deficiency (RAPID): a randomised, double-blind, placebo-controlled trial. Lancet. 2015;386(9991):360-8.
    • (2015) Lancet , vol.386 , Issue.9991 , pp. 360-368
    • Chapman, K.R.1    Burdon, J.G.2    Piitulainen, E.3    Sandhaus, R.A.4    Seersholm, N.5
  • 15
    • 67649407825 scopus 로고    scopus 로고
    • Exploring the role of CT densitometry: a randomised study of augmentation therapy in alpha1-antitrypsin deficiency
    • Dirksen A, Piitulainen E, Parr DG, Deng C, Wencker M, et al. Exploring the role of CT densitometry: a randomised study of augmentation therapy in alpha1-antitrypsin deficiency. Eur Respir J. 2009;33(6):1345-53.
    • (2009) Eur Respir J , vol.33 , Issue.6 , pp. 1345-1353
    • Dirksen, A.1    Piitulainen, E.2    Parr, D.G.3    Deng, C.4    Wencker, M.5
  • 16
    • 0032110436 scopus 로고    scopus 로고
    • Survival and FEV1 decline in individuals with severe deficiency of alpha1-antitrypsin
    • The Alpha-1-Antitrypsin Deficiency Registry Study Group. Survival and FEV1 decline in individuals with severe deficiency of alpha1-antitrypsin. A J Respir Crit Care Med. 1998;158(1):49-59.
    • (1998) A J Respir Crit Care Med , vol.158 , Issue.1 , pp. 49-59
  • 18
    • 85014597265 scopus 로고    scopus 로고
    • Global strategy for the diagnosis, management and prevention of chronic obstructive lung disease 2017 report
    • Vogelmeier CF, Criner GJ, Martinez FJ, Anzueto A, Barnes PJ, et al. Global strategy for the diagnosis, management and prevention of chronic obstructive lung disease 2017 report. Respirology. 2017;22(3):575-601.
    • (2017) Respirology , vol.22 , Issue.3 , pp. 575-601
    • Vogelmeier, C.F.1    Criner, G.J.2    Martinez, F.J.3    Anzueto, A.4    Barnes, P.J.5
  • 19
    • 84983353692 scopus 로고    scopus 로고
    • Individualized lung function trends in alpha-1-antitrypsin deficiency: a need for patience in order to provide patient centered management?
    • Stockley RA, Edgar RG, Pillai A, Turner AM. Individualized lung function trends in alpha-1-antitrypsin deficiency: a need for patience in order to provide patient centered management? Int J Chron Obstruct Pulmon Dis. 2016;11:1745.
    • (2016) Int J Chron Obstruct Pulmon Dis. , vol.11 , pp. 1745
    • Stockley, R.A.1    Edgar, R.G.2    Pillai, A.3    Turner, A.M.4
  • 21
    • 84901854418 scopus 로고    scopus 로고
    • Spirometric and gas transfer discordance in a alpha1 -antitrypsin deficiency. Patient characteristics and progression
    • Ward H, Turner AM, Stockley RA. Spirometric and gas transfer discordance in a alpha1 -antitrypsin deficiency. Patient characteristics and progression. Chest. 2014;145(6):1316-24.
    • (2014) Chest , vol.145 , Issue.6 , pp. 1316-1324
    • Ward, H.1    Turner, A.M.2    Stockley, R.A.3
  • 22
    • 18344386781 scopus 로고    scopus 로고
    • Interpretation of quality of life scores from the St George's respiratory questionnaire
    • Ferrer M, Villasante C, Alonso J, Sobradillo V, Gabriel R, et al. Interpretation of quality of life scores from the St George's respiratory questionnaire. Eur Respir J. 2002;19(3):405-13.
    • (2002) Eur Respir J , vol.19 , Issue.3 , pp. 405-413
    • Ferrer, M.1    Villasante, C.2    Alonso, J.3    Sobradillo, V.4    Gabriel, R.5
  • 23
    • 85012012686 scopus 로고    scopus 로고
    • Health status and lung function in the swedish alpha 1-antitrypsin deficient cohort, identified by neonatal screening, at the age of 37-40 years
    • Piitulainen E, Mostafavi B, Tanash HA. Health status and lung function in the swedish alpha 1-antitrypsin deficient cohort, identified by neonatal screening, at the age of 37-40 years. Int J Chron Obstruct Pulmon Dis. 2017;12:495.
    • (2017) Int J Chron Obstruct Pulmon Dis , vol.12 , pp. 495
    • Piitulainen, E.1    Mostafavi, B.2    Tanash, H.A.3
  • 24
    • 20444455824 scopus 로고    scopus 로고
    • Exacerbations in alpha1-antitrypsin deficiency
    • Needham M, Stockley RA. Exacerbations in alpha1-antitrypsin deficiency. Eur Respir J. 2005;25(6):992-1000.
    • (2005) Eur Respir J , vol.25 , Issue.6 , pp. 992-1000
    • Needham, M.1    Stockley, R.A.2
  • 25
    • 0347291903 scopus 로고    scopus 로고
    • Correlation between annual change in health status and computer tomography derived lung density in subjects with alpha1-antitrypsin deficiency
    • Stolk J, Ng WH, Bakker ME, Reiber JHC, Rabe KF, et al. Correlation between annual change in health status and computer tomography derived lung density in subjects with alpha1-antitrypsin deficiency. Thorax. 2003;58(12):1027-30.
    • (2003) Thorax , vol.58 , Issue.12 , pp. 1027-1030
    • Stolk, J.1    Ng, W.H.2    Bakker, M.E.3    Reiber, J.H.C.4    Rabe, K.F.5
  • 26
    • 85019674829 scopus 로고    scopus 로고
    • Deterioration of quality of life is associated with the exacerbation frequency in individuals with alpha-1-antitrypsin deficiency-analysis from the german registry
    • Bernhard N, Lepper PM, Vogelmeier C, Seibert M, Wagenpfeil S, et al. Deterioration of quality of life is associated with the exacerbation frequency in individuals with alpha-1-antitrypsin deficiency-analysis from the german registry. Int J Chron Obstruct Pulmon Dis. 2017;12:1427.
    • (2017) Int J Chron Obstruct Pulmon Dis , vol.12 , pp. 1427
    • Bernhard, N.1    Lepper, P.M.2    Vogelmeier, C.3    Seibert, M.4    Wagenpfeil, S.5
  • 27
    • 18244368740 scopus 로고    scopus 로고
    • Improved health outcomes in patients with COPD during 1 yr's treatment with tiotropium
    • Vincken W, van Noord JA, Greefhorst AP, Bantje TA, Kesten S, et al. Improved health outcomes in patients with COPD during 1 yr's treatment with tiotropium. Eur Respir J. 2002;19(2):209-16.
    • (2002) Eur Respir J , vol.19 , Issue.2 , pp. 209-216
    • Vincken, W.1    van Noord, J.A.2    Greefhorst, A.P.3    Bantje, T.A.4    Kesten, S.5
  • 28
    • 72549116822 scopus 로고    scopus 로고
    • Adherence to inhaled therapy, mortality and hospital admission in COPD
    • Vestbo J, Anderson JA, Calverley PM, Celli B, Ferguson GT, et al. Adherence to inhaled therapy, mortality and hospital admission in COPD. Thorax. 2009;64(11):939-43.
    • (2009) Thorax , vol.64 , Issue.11 , pp. 939-943
    • Vestbo, J.1    Anderson, J.A.2    Calverley, P.M.3    Celli, B.4    Ferguson, G.T.5
  • 29
    • 0037425745 scopus 로고    scopus 로고
    • Combined salmeterol and fluticasone in the treatment of chronic obstructive pulmonary disease: a randomised controlled trial
    • Calverley P, Pauwels R, Vestbo J, Jones P, Pride N, et al. Combined salmeterol and fluticasone in the treatment of chronic obstructive pulmonary disease: a randomised controlled trial. Lancet. 2003;361(9356):449-56.
    • (2003) Lancet , vol.361 , Issue.9356 , pp. 449-456
    • Calverley, P.1    Pauwels, R.2    Vestbo, J.3    Jones, P.4    Pride, N.5
  • 31
    • 70349135597 scopus 로고    scopus 로고
    • Intravenous alpha-1 antitrypsin augmentation therapy for treating patients with alpha-1 antitrypsin deficiency and lung disease
    • Gøtzsche PC, Johansen HK. Intravenous alpha-1 antitrypsin augmentation therapy for treating patients with alpha-1 antitrypsin deficiency and lung disease. Cochrane Libr. 2016; https://doi.org/10.1002/14651858.CD007851.pub3.
    • (2016) Cochrane Libr.
    • Gøtzsche, P.C.1    Johansen, H.K.2
  • 32
    • 84884656212 scopus 로고    scopus 로고
    • Assessing regression to the mean effects in health care initiatives
    • Linden A. Assessing regression to the mean effects in health care initiatives. BMC Med Res Methodol. 2013;13(1):119.
    • (2013) BMC Med Res Methodol , vol.13 , Issue.1 , pp. 119
    • Linden, A.1
  • 33
    • 78649949010 scopus 로고    scopus 로고
    • Retinoid treatment of emphysema in patients on the Alpha-1 international registry. The REPAIR study: study design, methodology and quality control of study assessments
    • Stolk J, Cooper BG, Stoel B, Rames A, Rutman O, et al. Retinoid treatment of emphysema in patients on the Alpha-1 international registry. The REPAIR study: study design, methodology and quality control of study assessments. Ther Adv Respir Dis. 2010;4(6):319-32.
    • (2010) Ther Adv Respir Dis , vol.4 , Issue.6 , pp. 319-332
    • Stolk, J.1    Cooper, B.G.2    Stoel, B.3    Rames, A.4    Rutman, O.5
  • 34
    • 69249228595 scopus 로고    scopus 로고
    • Mortality in alpha-1-antitrypsin deficiency in the United Kingdom
    • Dawkins P, Wood A, Nightingale P, Stockley R. Mortality in alpha-1-antitrypsin deficiency in the United Kingdom. Respir Med. 2009;103(10):1540-7.
    • (2009) Respir Med , vol.103 , Issue.10 , pp. 1540-1547
    • Dawkins, P.1    Wood, A.2    Nightingale, P.3    Stockley, R.4
  • 35
    • 33745135090 scopus 로고    scopus 로고
    • Validation of computed tomographic lung densitometry for monitoring emphysema in alpha1-antitrypsin deficiency
    • Parr DG, Stoel BC, Stolk J, Stockley RA. Validation of computed tomographic lung densitometry for monitoring emphysema in alpha1-antitrypsin deficiency. Thorax. 2006;61(6):485-90.
    • (2006) Thorax , vol.61 , Issue.6 , pp. 485-490
    • Parr, D.G.1    Stoel, B.C.2    Stolk, J.3    Stockley, R.A.4


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