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Volumn 11, Issue , 2015, Pages 143-151

Alpha-1 proteinase inhibitors for the treatment of alpha-1 antitrypsin deficiency: Safety, tolerability, and patient outcomes

Author keywords

Alpha 1; Augmentation; Deficiency; Emphysema; Replacement

Indexed keywords

ALPHA 1 ANTITRYPSIN; PLACEBO;

EID: 84922068725     PISSN: 11766336     EISSN: 1178203X     Source Type: Journal    
DOI: 10.2147/TCRM.S51474     Document Type: Article
Times cited : (24)

References (86)
  • 1
    • 84875674428 scopus 로고    scopus 로고
    • The electrophoretic alpha1-globulin pattern of serum in alpha 1-antitrypsin deficiency. 1963
    • Laurell CB, Eriksson S. The electrophoretic alpha1-globulin pattern of serum in alpha 1-antitrypsin deficiency. 1963. COPD. 2013;10(suppl 1):3–8.
    • (2013) COPD , vol.10 , pp. 3-8
    • Laurell, C.B.1    Eriksson, S.2
  • 2
    • 0014530551 scopus 로고
    • Cirrhosis associated with alpha-1-antitrypsin deficiency: A previously unrecognized inherited disorder
    • Sharp HL, Bridges RA, Krivit W, Freier EF. Cirrhosis associated with alpha-1-antitrypsin deficiency: a previously unrecognized inherited disorder. J Lab Clin Med. 1969;73(6):934–939.
    • (1969) J Lab Clin Med , vol.73 , Issue.6 , pp. 934-939
    • Sharp, H.L.1    Bridges, R.A.2    Krivit, W.3    Freier, E.F.4
  • 3
    • 0020510606 scopus 로고
    • Mutation of antitrypsin to antithrombin. Alpha 1-antitrypsin Pittsburgh (358 Met leads to Arg), a fatal bleeding disorder
    • Owen MC, Brennan SO, Lewis JH, Carrell RW. Mutation of antitrypsin to antithrombin. Alpha 1-antitrypsin Pittsburgh (358 Met leads to Arg), a fatal bleeding disorder. N Engl J Med. 1983;309(12):694–698.
    • (1983) N Engl J Med , vol.309 , Issue.12 , pp. 694-698
    • Owen, M.C.1    Brennan, S.O.2    Lewis, J.H.3    Carrell, R.W.4
  • 4
    • 0036433482 scopus 로고    scopus 로고
    • Worldwide racial and ethnic distribution of alpha 1-antitrypsin deficiency: Summary of an analysis of published genetic epidemiologic surveys
    • de Serres FJ. Worldwide racial and ethnic distribution of alpha 1-antitrypsin deficiency: summary of an analysis of published genetic epidemiologic surveys. Chest. 2002;122(5):1818–1829.
    • (2002) Chest , vol.122 , Issue.5 , pp. 1818-1829
    • de Serres, F.J.1
  • 5
    • 0031966749 scopus 로고    scopus 로고
    • Alpha 1-antitrypsin deficiency in Europe: Geographical distribution of Pi types S and Z
    • Hutchison DC. Alpha 1-antitrypsin deficiency in Europe: geographical distribution of Pi types S and Z. Respir Med. 1998;92(3):367–377.
    • (1998) Respir Med , vol.92 , Issue.3 , pp. 367-377
    • Hutchison, D.C.1
  • 6
    • 0024397003 scopus 로고
    • Serum alpha 1-antitrypsin deficiency associated with the common S-type (Glu264----Val) mutation results from intracellular degradation of alpha 1-antitrypsin prior to secretion
    • Curiel DT, Chytil A, Courtney M, Crystal RG. Serum alpha 1-antitrypsin deficiency associated with the common S-type (Glu264----Val) mutation results from intracellular degradation of alpha 1-antitrypsin prior to secretion. J Biol Chem. 1989;264(18):10477–10486.
    • (1989) J Biol Chem , vol.264 , Issue.18 , pp. 10477-10486
    • Curiel, D.T.1    Chytil, A.2    Courtney, M.3    Crystal, R.G.4
  • 7
    • 0026755363 scopus 로고
    • The mechanism of Z alpha 1-antitrypsin accumulation in the liver
    • Lomas DA, Evans DL, Finch JT, Carrell RW. The mechanism of Z alpha 1-antitrypsin accumulation in the liver. Nature. 1992;357(6379):605–607.
    • (1992) Nature , vol.357 , Issue.6379 , pp. 605-607
    • Lomas, D.A.1    Evans, D.L.2    Finch, J.T.3    Carrell, R.W.4
  • 8
    • 0037135563 scopus 로고    scopus 로고
    • Detection of circulating and endothelial cell polymers of Z and wild type alpha 1-antitrypsin by a monoclonal antibody
    • Janciauskiene S, Dominaitiene R, Sternby NH, Piitulainen E, Eriksson S. Detection of circulating and endothelial cell polymers of Z and wild type alpha 1-antitrypsin by a monoclonal antibody. J Biol Chem. 2002; 277(29):26540–26546.
    • (2002) J Biol Chem , vol.277 , Issue.29 , pp. 26540-26546
    • Janciauskiene, S.1    Dominaitiene, R.2    Sternby, N.H.3    Piitulainen, E.4    Eriksson, S.5
  • 9
    • 2442488539 scopus 로고    scopus 로고
    • Z alpha 1-antitrypsin polymerizes in the lung and acts as a neutrophil chemoattractant
    • Mulgrew AT, Taggart CC, Lawless MW, et al. Z alpha 1-antitrypsin polymerizes in the lung and acts as a neutrophil chemoattractant. Chest. 2004;125(5):1952–1957.
    • (2004) Chest , vol.125 , Issue.5 , pp. 1952-1957
    • Mulgrew, A.T.1    Taggart, C.C.2    Lawless, M.W.3
  • 10
    • 0025374923 scopus 로고
    • Neutrophil elastase cleaves C3bi on opsonized pseudomonas as well as CR1 on neutrophils to create a functionally important opsonin receptor mismatch
    • Tosi MF, Zakem H, Berger M. Neutrophil elastase cleaves C3bi on opsonized pseudomonas as well as CR1 on neutrophils to create a functionally important opsonin receptor mismatch. J Clin Invest. 1990;86(1):300–308.
    • (1990) J Clin Invest , vol.86 , Issue.1 , pp. 300-308
    • Tosi, M.F.1    Zakem, H.2    Berger, M.3
  • 11
    • 36849020586 scopus 로고    scopus 로고
    • Cleavage of CXCR1 on neutrophils disables bacterial killing in cystic fibrosis lung disease
    • Hartl D, Latzin P, Hordijk P, et al. Cleavage of CXCR1 on neutrophils disables bacterial killing in cystic fibrosis lung disease. Nat Med. 2007;13(12):1423–1430.
    • (2007) Nat Med , vol.13 , Issue.12 , pp. 1423-1430
    • Hartl, D.1    Latzin, P.2    Hordijk, P.3
  • 12
    • 0021239746 scopus 로고
    • Proteins of the cystic fibrosis respiratory tract. Fragmented immunoglobulin G opsonic antibody causing defective opsonophagocytosis
    • Fick RB Jr, Naegel GP, Squier SU, Wood RE, Gee JB, Reynolds HY. Proteins of the cystic fibrosis respiratory tract. Fragmented immunoglobulin G opsonic antibody causing defective opsonophagocytosis. J Clin Invest. 1984;74(1):236–248.
    • (1984) J Clin Invest , vol.74 , Issue.1 , pp. 236-248
    • Fick, R.B.1    Naegel, G.P.2    Squier, S.U.3    Wood, R.E.4    Gee, J.B.5    Reynolds, H.Y.6
  • 13
    • 0021135892 scopus 로고
    • Reduction of ciliary beat frequency in vitro by sputum from patients with bronchiectasis: A serine proteinase effect
    • Smallman LA, Hill SL, Stockley RA. Reduction of ciliary beat frequency in vitro by sputum from patients with bronchiectasis: a serine proteinase effect. Thorax. 1984;39(9):663–667.
    • (1984) Thorax , vol.39 , Issue.9 , pp. 663-667
    • Smallman, L.A.1    Hill, S.L.2    Stockley, R.A.3
  • 14
    • 57749104104 scopus 로고    scopus 로고
    • Elafin, an elastase-specific inhibitor, is cleaved by its cognate enzyme neutrophil elastase in sputum from individuals with cystic fibrosis
    • Guyot N, Butler MW, McNally P, et al. Elafin, an elastase-specific inhibitor, is cleaved by its cognate enzyme neutrophil elastase in sputum from individuals with cystic fibrosis. J Biol Chem. 2008;283(47):32377–32385.
    • (2008) J Biol Chem , vol.283 , Issue.47 , pp. 32377-32385
    • Guyot, N.1    Butler, M.W.2    McNally, P.3
  • 15
    • 0035823570 scopus 로고    scopus 로고
    • Cathepsin B, L, and S cleave and inactivate secretory leucoprotease inhibitor
    • Taggart CC, Lowe GJ, Greene CM, et al. Cathepsin B, L, and S cleave and inactivate secretory leucoprotease inhibitor. J Biol Chem. 2001;276(36):33345–33352.
    • (2001) J Biol Chem , vol.276 , Issue.36 , pp. 33345-33352
    • Taggart, C.C.1    Lowe, G.J.2    Greene, C.M.3
  • 16
    • 34547921190 scopus 로고    scopus 로고
    • Tauroursodeoxycholic acid inhibits apoptosis induced by Z alpha-1 antitrypsin via inhibition of bad
    • Miller SD, Greene CM, McLean C, et al. Tauroursodeoxycholic acid inhibits apoptosis induced by Z alpha-1 antitrypsin via inhibition of bad. Hepatology. 2007;46(2):496–503.
    • (2007) Hepatology , vol.46 , Issue.2 , pp. 496-503
    • Miller, S.D.1    Greene, C.M.2    McLean, C.3
  • 17
    • 0000461577 scopus 로고
    • Pulmonary emphysema and alpha 1-antitrypsin deficiency
    • Eriksson S. Pulmonary emphysema and alpha 1-antitrypsin deficiency. Acta Med Scand. 1964;175:197–205.
    • (1964) Acta Med Scand , vol.175 , pp. 197-205
    • Eriksson, S.1
  • 18
    • 0019963490 scopus 로고
    • Alpha 1-antitrypsin deficiency: The radiological features of pulmonary emphysema in subjects of Pi type Z and Pi type SZ: A survey by the British Thoracic Association
    • Gishen P, Saunders AJ, Tobin MJ, Hutchison DC. Alpha 1-antitrypsin deficiency: the radiological features of pulmonary emphysema in subjects of Pi type Z and Pi type SZ: a survey by the British Thoracic Association. Clin Radiol. 1982;33(4):371–377.
    • (1982) Clin Radiol , vol.33 , Issue.4 , pp. 371-377
    • Gishen, P.1    Saunders, A.J.2    Tobin, M.J.3    Hutchison, D.C.4
  • 19
    • 0023795389 scopus 로고
    • Clinical features and history of the destructive lung disease associated with alpha-1-antitrypsin deficiency of adults with pulmonary symptoms
    • Brantly ML, Paul LD, Miller BH, Falk RT, Wu M, Crystal RG. Clinical features and history of the destructive lung disease associated with alpha-1-antitrypsin deficiency of adults with pulmonary symptoms. Am Rev Respir Dis. 1988;138(2):327–336.
    • (1988) Am Rev Respir Dis , vol.138 , Issue.2 , pp. 327-336
    • Brantly, M.L.1    Paul, L.D.2    Miller, B.H.3    Falk, R.T.4    Wu, M.5    Crystal, R.G.6
  • 20
    • 0020678269 scopus 로고
    • Alpha 1 antitrypsin deficiency: The clinical and physiological features of pulmonary emphysema in subjects homozygous for Pi type Z. A survey by the British thoracic association
    • Tobin MJ, Cook PJ, Hutchison DC. Alpha 1 antitrypsin deficiency: the clinical and physiological features of pulmonary emphysema in subjects homozygous for Pi type Z. A survey by the British thoracic association. Br J Dis Chest. 1983;77(1):14–27.
    • (1983) Br J Dis Chest , vol.77 , Issue.1 , pp. 14-27
    • Tobin, M.J.1    Cook, P.J.2    Hutchison, D.C.3
  • 21
    • 0021960082 scopus 로고
    • Smoking, lung function, and alpha 1-antitrypsin deficiency
    • Janus ED, Phillips NT, Carrell RW. Smoking, lung function, and alpha 1-antitrypsin deficiency. Lancet. 1985;1(8421):152–154.
    • (1985) Lancet , vol.1 , Issue.8421 , pp. 152-154
    • Janus, E.D.1    Phillips, N.T.2    Carrell, R.W.3
  • 22
    • 0031027927 scopus 로고    scopus 로고
    • Baseline characteristics of enrollees in the national heart, lung and blood institute registry of alpha 1-antitrypsin deficiency. Alpha 1-antitrypsin deficiency registry study group
    • McElvaney NG, Stoller JK, Buist AS, et al. Baseline characteristics of enrollees in the national heart, lung and blood institute registry of alpha 1-antitrypsin deficiency. Alpha 1-antitrypsin deficiency registry study group. Chest. 1997;111(2):394–403.
    • (1997) Chest , vol.111 , Issue.2 , pp. 394-403
    • McElvaney, N.G.1    Stoller, J.K.2    Buist, A.S.3
  • 23
    • 0028812493 scopus 로고
    • Survival in relation to lung function and smoking cessation in patients with severe hereditary alpha 1-antitrypsin deficiency
    • (2 pt 1)
    • Seersholm N, Kok-Jensen A. Survival in relation to lung function and smoking cessation in patients with severe hereditary alpha 1-antitrypsin deficiency. Am J Respir Crit Care Med. 1995;151(2 pt 1):369–373.
    • (1995) Am J Respir Crit Care Med , vol.151 , pp. 369-373
    • Seersholm, N.1    Kok-Jensen, A.2
  • 24
    • 0030873292 scopus 로고    scopus 로고
    • Atopy, asthma, and emphysema in patients with severe alpha-1-antitrypysin deficiency
    • Eden E, Mitchell D, Mehlman B, et al. Atopy, asthma, and emphysema in patients with severe alpha-1-antitrypysin deficiency. Am J Respir Crit Care Med. 1997;156(1):68–74.
    • (1997) Am J Respir Crit Care Med , vol.156 , Issue.1 , pp. 68-74
    • Eden, E.1    Mitchell, D.2    Mehlman, B.3
  • 25
    • 0018128288 scopus 로고
    • Natural history and life expectancy in severe alpha 1-antitrypsin deficiency, Pi Z
    • Larsson C. Natural history and life expectancy in severe alpha 1-antitrypsin deficiency, Pi Z. Acta Med Scand. 1978;204(5):345–351.
    • (1978) Acta Med Scand , vol.204 , Issue.5 , pp. 345-351
    • Larsson, C.1
  • 26
    • 0026551587 scopus 로고
    • High resolution computed tomography (HRCT) in emphysema associated with alpha-1-antitrypsin deficiency
    • Guest PJ, Hansell DM. High resolution computed tomography (HRCT) in emphysema associated with alpha-1-antitrypsin deficiency. Clin Radiol. 1992;45(4):260–266.
    • (1992) Clin Radiol , vol.45 , Issue.4 , pp. 260-266
    • Guest, P.J.1    Hansell, D.M.2
  • 28
    • 0029083915 scopus 로고
    • The liver in adolescents with alpha 1-antitrypsin deficiency
    • Sveger T, Eriksson S. The liver in adolescents with alpha 1-antitrypsin deficiency. Hepatology. 1995;22(2):514–517.
    • (1995) Hepatology , vol.22 , Issue.2 , pp. 514-517
    • Sveger, T.1    Eriksson, S.2
  • 29
    • 0017099344 scopus 로고
    • Liver disease in alpha1-antitrypsin deficiency detected by screening of 200,000 infants
    • Sveger T. Liver disease in alpha1-antitrypsin deficiency detected by screening of 200,000 infants. N Engl J Med. 1976;294(24):1316–1321.
    • (1976) N Engl J Med , vol.294 , Issue.24 , pp. 1316-1321
    • Sveger, T.1
  • 30
    • 0023218940 scopus 로고
    • Alpha 1-antitrypsin deficiency and liver cirrhosis in adults. An analysis of 35 Swedish autopsied cases
    • Eriksson S. Alpha 1-antitrypsin deficiency and liver cirrhosis in adults. An analysis of 35 Swedish autopsied cases. Acta Med Scand. 1987;221(5):461–467.
    • (1987) Acta Med Scand , vol.221 , Issue.5 , pp. 461-467
    • Eriksson, S.1
  • 31
    • 0023613362 scopus 로고
    • Late manifestation of chronic liver disease in adults with alpha-1-antitrypsin deficiency
    • Rakela J, Goldschmiedt M, Ludwig J. Late manifestation of chronic liver disease in adults with alpha-1-antitrypsin deficiency. Dig Dis Sci. 1987;32(12):1358–1362.
    • (1987) Dig Dis Sci , vol.32 , Issue.12 , pp. 1358-1362
    • Rakela, J.1    Goldschmiedt, M.2    Ludwig, J.3
  • 32
    • 0028990371 scopus 로고
    • Alpha 1-antitrypsin deficiency and anti-proteinase 3 antibodies in anti-neutrophil cytoplasmic antibody (ANCA)-associated systemic vasculitis
    • Savige JA, Chang L, Cook L, Burdon J, Daskalakis M, Doery J. Alpha 1-antitrypsin deficiency and anti-proteinase 3 antibodies in anti-neutrophil cytoplasmic antibody (ANCA)-associated systemic vasculitis. Clin Exp Immunol. 1995;100(2):194–197.
    • (1995) Clin Exp Immunol , vol.100 , Issue.2 , pp. 194-197
    • Savige, J.A.1    Chang, L.2    Cook, L.3    Burdon, J.4    Daskalakis, M.5    Doery, J.6
  • 33
    • 0029796480 scopus 로고    scopus 로고
    • Giant intracranial aneurysm and fibromuscular dysplasia in an adolescent with alpha 1-antitrypsin deficiency
    • Schievink WI, Puumala MR, Meyer FB, Raffel C, Katzmann JA, Parisi JE. Giant intracranial aneurysm and fibromuscular dysplasia in an adolescent with alpha 1-antitrypsin deficiency. J Neurosurg. 1996;85(3):503–506.
    • (1996) J Neurosurg , vol.85 , Issue.3 , pp. 503-506
    • Schievink, W.I.1    Puumala, M.R.2    Meyer, F.B.3    Raffel, C.4    Katzmann, J.A.5    Parisi, J.E.6
  • 34
    • 0026499863 scopus 로고
    • The pathologic spectrum of the nephropathy associated with alpha 1-antitrypsin deficiency
    • Davis ID, Burke B, Freese D, Sharp HL, Kim Y. The pathologic spectrum of the nephropathy associated with alpha 1-antitrypsin deficiency. Hum Pathol. 1992;23(1):57–62.
    • (1992) Hum Pathol , vol.23 , Issue.1 , pp. 57-62
    • Davis, I.D.1    Burke, B.2    Freese, D.3    Sharp, H.L.4    Kim, Y.5
  • 35
    • 0026537743 scopus 로고
    • Glomerulonephritis with end-stage liver disease in childhood
    • Noble-Jamieson G, Thiru S, Johnston P, Friend P, Barnes ND. Glomerulonephritis with end-stage liver disease in childhood. Lancet. 1992;339(8795):706–707.
    • (1992) Lancet , vol.339 , Issue.8795 , pp. 706-707
    • Noble-Jamieson, G.1    Thiru, S.2    Johnston, P.3    Friend, P.4    Barnes, N.D.5
  • 36
    • 0019787989 scopus 로고
    • Replacement therapy of alpha 1-antitrypsin deficiency. Reversal of protease-antiprotease imbalance within the alveolar structures of PiZ subjects
    • Gadek JE, Klein HG, Holland PV, Crystal RG. Replacement therapy of alpha 1-antitrypsin deficiency. Reversal of protease-antiprotease imbalance within the alveolar structures of PiZ subjects. J Clin Invest. 1981;68(5):1158–1165.
    • (1981) J Clin Invest , vol.68 , Issue.5 , pp. 1158-1165
    • Gadek, J.E.1    Klein, H.G.2    Holland, P.V.3    Crystal, R.G.4
  • 37
    • 0023148995 scopus 로고
    • Replacement therapy for alpha 1-antitrypsin deficiency associated with emphysema
    • Wewers MD, Casolaro MA, Sellers SE, et al. Replacement therapy for alpha 1-antitrypsin deficiency associated with emphysema. N Engl J Med. 1987;316(17):1055–1062.
    • (1987) N Engl J Med , vol.316 , Issue.17 , pp. 1055-1062
    • Wewers, M.D.1    Casolaro, M.A.2    Sellers, S.E.3
  • 38
    • 0025350658 scopus 로고
    • Alpha 1-antitrypsin deficiency, emphysema, and liver disease. Genetic basis and strategies for therapy
    • Crystal RG. Alpha 1-antitrypsin deficiency, emphysema, and liver disease. Genetic basis and strategies for therapy. J Clin Invest. 1990;85(5):1343–1352.
    • (1990) J Clin Invest , vol.85 , Issue.5 , pp. 1343-1352
    • Crystal, R.G.1
  • 39
    • 84880712208 scopus 로고    scopus 로고
    • IV alpha1 antitrypsin (A1AT) preserves lung density in homozygous alpha1 antitrypsin deficiency (A1ATD); a randomized, placebo-controlled trial
    • Chapman KR, Burdon JGW, Piitulainen E, et al. IV alpha1 antitrypsin (A1AT) preserves lung density in homozygous alpha1 antitrypsin deficiency (A1ATD); a randomized, placebo-controlled trial. Am J Respir Crit Care Med. 2013;187:A6069.
    • (2013) Am J Respir Crit Care Med , vol.187
    • Chapman, K.R.1    Burdon, J.2    Piitulainen, E.3
  • 40
    • 0031242842 scopus 로고    scopus 로고
    • Does alpha1-antitrypsin augmentation therapy slow the annual decline in FEV1 in patients with severe hereditary alpha1-antitrypsin deficiency? Wissenschaftliche Arbeitsgemeinschaft zur Therapie von Lungenerkrankungen (WATL) alpha1-AT study group
    • Seersholm N, Wencker M, Banik N, et al. Does alpha1-antitrypsin augmentation therapy slow the annual decline in FEV1 in patients with severe hereditary alpha1-antitrypsin deficiency? Wissenschaftliche Arbeitsgemeinschaft zur Therapie von Lungenerkrankungen (WATL) alpha1-AT study group. The Eur Respir J. 1997;10(10):2260–2263.
    • (1997) The Eur Respir J , vol.10 , Issue.10 , pp. 2260-2263
    • Seersholm, N.1    Wencker, M.2    Banik, N.3
  • 41
    • 0032110436 scopus 로고    scopus 로고
    • Survival and FEV1 decline in individuals with severe deficiency of alpha1-antitrypsin
    • Survival and FEV1 decline in individuals with severe deficiency of alpha1-antitrypsin. The Alpha-1-Antitrypsin Deficiency Registry Study Group. Am J Respir Crit Care Med. 1998;158(1):49–59.
    • (1998) Am J Respir Crit Care Med , vol.158 , Issue.1 , pp. 49-59
  • 42
    • 0032724157 scopus 로고    scopus 로고
    • A randomized clinical trial of alpha(1)-antitrypsin augmentation therapy
    • Dirksen A, Dijkman JH, Madsen F, et al. A randomized clinical trial of alpha(1)-antitrypsin augmentation therapy. Am J Respir Crit Care Med. 1999;160(5 pt 1):1468–1472.
    • (1999) Am J Respir Crit Care Med , vol.160 , pp. 1468-1472
    • Dirksen, A.1    Dijkman, J.H.2    Madsen, F.3
  • 44
    • 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, et al. Exploring the role of CT densitometry: a randomised study of augmentation therapy in alpha1-antitrypsin deficiency. Eur Respir J. 2009;33(6):1345–1353.
    • (2009) Eur Respir J , vol.33 , Issue.6 , pp. 1345-1353
    • Dirksen, A.1    Piitulainen, E.2    Parr, D.G.3
  • 45
    • 0031943091 scopus 로고    scopus 로고
    • Commercialization of proteins produced in the mammary gland
    • Ziomek CA. Commercialization of proteins produced in the mammary gland. Theriogenology. 1998;49(1):139–144.
    • (1998) Theriogenology , vol.49 , Issue.1 , pp. 139-144
    • Ziomek, C.A.1
  • 46
    • 14744276106 scopus 로고
    • High level expression of active human alpha-1-antitrypsin in the milk of transgenic sheep
    • Wright G, Carver A, Cottom D, et al. High level expression of active human alpha-1-antitrypsin in the milk of transgenic sheep. Biotechnology. 1991;9(9):830–834.
    • (1991) Biotechnology , vol.9 , Issue.9 , pp. 830-834
    • Wright, G.1    Carver, A.2    Cottom, D.3
  • 47
    • 0023588232 scopus 로고
    • Augmentation of lung antineutrophil elastase capacity with recombinant human alpha-1-antitrypsin
    • Casolaro MA, Fells G, Wewers M, et al. Augmentation of lung antineutrophil elastase capacity with recombinant human alpha-1-antitrypsin. J Appl Physiol. 1987;63(5):2015–2023.
    • (1987) J Appl Physiol , vol.63 , Issue.5 , pp. 2015-2023
    • Casolaro, M.A.1    Fells, G.2    Wewers, M.3
  • 48
    • 0024417082 scopus 로고
    • Recombinant DNA-produced alpha 1-antitrypsin administered by aerosol augments lower respiratory tract antineutrophil elastase defenses in individuals with alpha 1-antitrypsin deficiency
    • Hubbard RC, McElvaney NG, Sellers SE, Healy JT, Czerski DB, Crystal RG. Recombinant DNA-produced alpha 1-antitrypsin administered by aerosol augments lower respiratory tract antineutrophil elastase defenses in individuals with alpha 1-antitrypsin deficiency.J Clin Invest. 1989;84(4):1349–1354.
    • (1989) J Clin Invest , vol.84 , Issue.4 , pp. 1349-1354
    • Hubbard, R.C.1    McElvaney, N.G.2    Sellers, S.E.3    Healy, J.T.4    Czerski, D.B.5
  • 49
    • 0027748178 scopus 로고
    • Hepatic gene therapy: Efficient retroviral-mediated gene transfer into rat hepatocytes in vivo
    • Kolodka TM, Finegold M, Kay MA, Woo SL. Hepatic gene therapy: efficient retroviral-mediated gene transfer into rat hepatocytes in vivo. Somat Cell Mol Genet. 1993;19(5):491–497.
    • (1993) Somat Cell Mol Genet , vol.19 , Issue.5 , pp. 491-497
    • Kolodka, T.M.1    Finegold, M.2    Kay, M.A.3    Woo, S.L.4
  • 50
    • 13144305062 scopus 로고    scopus 로고
    • Sustained secretion of human alpha-1-antitrypsin from murine muscle transduced with adeno-associated virus vectors
    • Song S, Morgan M, Ellis T, et al. Sustained secretion of human alpha-1-antitrypsin from murine muscle transduced with adeno-associated virus vectors. Proc Natl Acad Sci U S A. 1998;95(24):14384–14388.
    • (1998) Proc Natl Acad Sci U S A , vol.95 , Issue.24 , pp. 14384-14388
    • Song, S.1    Morgan, M.2    Ellis, T.3
  • 51
    • 79951817601 scopus 로고    scopus 로고
    • Preclinical evaluation of a recombinant adeno-associated virus vector expressing human alpha-1 antitrypsin made using a recombinant herpes simplex virus production method
    • Chulay JD, Ye GJ, Thomas DL, et al. Preclinical evaluation of a recombinant adeno-associated virus vector expressing human alpha-1 antitrypsin made using a recombinant herpes simplex virus production method. Hum Gene Ther. 2011;22(2):155–165.
    • (2011) Hum Gene Ther , vol.22 , Issue.2 , pp. 155-165
    • Chulay, J.D.1    Ye, G.J.2    Thomas, D.L.3
  • 52
    • 84907251315 scopus 로고    scopus 로고
    • Intrapleural administration of an AAVrh.10 vector coding for human alpha1-antitrypsin for the treatment of alpha1-antitrypsin deficiency. Human gene therapy
    • Chiuchiolo MJ, Kaminsky SM, Sondhi D, et al. Intrapleural administration of an AAVrh.10 vector coding for human alpha1-antitrypsin for the treatment of alpha1-antitrypsin deficiency. Human gene therapy. Clin Dev. 2013;24(4):161–173.
    • (2013) Clin Dev , vol.24 , Issue.4 , pp. 161-173
    • Chiuchiolo, M.J.1    Kaminsky, S.M.2    Sondhi, D.3
  • 53
    • 33845807816 scopus 로고    scopus 로고
    • Phase I trial of intramuscular injection of a recombinant adeno-associated virus serotype 2 alphal-antitrypsin (AAT) vector in AAT-deficient adults
    • Brantly ML, Spencer LT, Humphries M, et al. Phase I trial of intramuscular injection of a recombinant adeno-associated virus serotype 2 alphal-antitrypsin (AAT) vector in AAT-deficient adults. Hum Gene Ther. 2006;17(12):1177–1186.
    • (2006) Hum Gene Ther , vol.17 , Issue.12 , pp. 1177-1186
    • Brantly, M.L.1    Spencer, L.T.2    Humphries, M.3
  • 54
    • 70349481529 scopus 로고    scopus 로고
    • Sustained transgene expression despite T lymphocyte responses in a clinical trial of rAAV1-AAT gene therapy
    • Brantly ML, Chulay JD, Wang L, et al. Sustained transgene expression despite T lymphocyte responses in a clinical trial of rAAV1-AAT gene therapy. Proc Natl Acad Sci U S A. 2009;106(38):16363–16368.
    • (2009) Proc Natl Acad Sci U S A , vol.106 , Issue.38 , pp. 16363-16368
    • Brantly, M.L.1    Chulay, J.D.2    Wang, L.3
  • 55
    • 80155145248 scopus 로고    scopus 로고
    • Phase 2 clinical trial of a recombinant adeno-associated viral vector expressing alpha1-antitrypsin: Interim results
    • Flotte TR, Trapnell BC, Humphries M, et al. Phase 2 clinical trial of a recombinant adeno-associated viral vector expressing alpha1-antitrypsin: interim results. Hum Gene Ther. 2011;22(10):1239–1247.
    • (2011) Hum Gene Ther , vol.22 , Issue.10 , pp. 1239-1247
    • Flotte, T.R.1    Trapnell, B.C.2    Humphries, M.3
  • 56
    • 0034193342 scopus 로고    scopus 로고
    • Transfection of nasal mucosa with a normal alpha1-antitrypsin gene in alpha1-antitrypsin-deficient subjects: Comparison with protein therapy
    • Brigham KL, Lane KB, Meyrick B, et al. Transfection of nasal mucosa with a normal alpha1-antitrypsin gene in alpha1-antitrypsin-deficient subjects: comparison with protein therapy. Hum Gene Ther. 2000;11(7):1023–1032.
    • (2000) Hum Gene Ther , vol.11 , Issue.7 , pp. 1023-1032
    • Brigham, K.L.1    Lane, K.B.2    Meyrick, B.3
  • 57
    • 84922069701 scopus 로고    scopus 로고
    • Current status of gene therapy for alpha-1 antitrypsin deficiency
    • Loring HS, Flotte TR. Current status of gene therapy for alpha-1 antitrypsin deficiency. Expert Opin Biol Ther. 2014:1–8.
    • (2014) Expert Opin Biol Ther , pp. 1-8
    • Loring, H.S.1    Flotte, T.R.2
  • 58
    • 0345424863 scopus 로고    scopus 로고
    • E9 Statistical Principles for Clinical Trials. Guidance for Industry; 1998. Available from: http://www.fda.gov/downloads/Drugs/GuidanceComplianceRegulatoryInformation/Guidances/ucm073137.pdf
    • (1998) Guidance for Industry
  • 59
    • 0038216603 scopus 로고    scopus 로고
    • Augmentation therapy with alpha1-antitrypsin: Patterns of use and adverse events
    • Stoller JK, Fallat R, Schluchter MD, et al. Augmentation therapy with alpha1-antitrypsin: patterns of use and adverse events. Chest. 2003;123(5):1425–1434.
    • (2003) Chest , vol.123 , Issue.5 , pp. 1425-1434
    • Stoller, J.K.1    Fallat, R.2    Schluchter, M.D.3
  • 60
    • 0031884479 scopus 로고    scopus 로고
    • Long-term treatment of alpha1-antitrypsin deficiency-related pulmonary emphysema with human alpha1-antitrypsin. Wissenschaftliche Arbeitsgemeinschaft zur Therapie von Lungenerkrankungen (WATL)-alpha1-AT-study group
    • Wencker M, Banik N, Buhl R, Seidel R, Konietzko N. Long-term treatment of alpha1-antitrypsin deficiency-related pulmonary emphysema with human alpha1-antitrypsin. Wissenschaftliche Arbeitsgemeinschaft zur Therapie von Lungenerkrankungen (WATL)-alpha1-AT-study group. Eur Respir J. 1998;11(2):428–433.
    • (1998) Eur Respir J , vol.11 , Issue.2 , pp. 428-433
    • Wencker, M.1    Banik, N.2    Buhl, R.3    Seidel, R.4    Konietzko, N.5
  • 61
    • 0031723072 scopus 로고    scopus 로고
    • Acute allergic reaction and demonstration of specific IgE antibodies against alpha-1-protease inhibitor
    • Meyer FJ, Wencker M, Teschler H, et al. Acute allergic reaction and demonstration of specific IgE antibodies against alpha-1-protease inhibitor. Eur Respir J. 1998;12(4):996–997.
    • (1998) Eur Respir J , vol.12 , Issue.4 , pp. 996-997
    • Meyer, F.J.1    Wencker, M.2    Teschler, H.3
  • 62
    • 77957201481 scopus 로고    scopus 로고
    • Pharmacokinetic comparability of Prolastin(R)-C to Prolastin(R) in alpha(1)-antitrypsin deficiency: A randomized study
    • Stocks JM, Brantly ML, Wang-Smith L, et al. Pharmacokinetic comparability of Prolastin(R)-C to Prolastin(R) in alpha(1)-antitrypsin deficiency: a randomized study. BMC Clin Pharmacol. 2010;10:13.
    • (2010) BMC Clin Pharmacol , vol.10
    • Stocks, J.M.1    Brantly, M.L.2    Wang-Smith, L.3
  • 63
    • 70350450961 scopus 로고    scopus 로고
    • Augmentation therapy for alpha1 antitrypsin deficiency: A meta-analysis
    • Chapman KR, Stockley RA, Dawkins C, Wilkes MM, Navickis RJ. Augmentation therapy for alpha1 antitrypsin deficiency: a meta-analysis. COPD. 2009;6(3):177–184.
    • (2009) COPD , vol.6 , Issue.3 , pp. 177-184
    • Chapman, K.R.1    Stockley, R.A.2    Dawkins, C.3    Wilkes, M.M.4    Navickis, R.J.5
  • 64
    • 77957273246 scopus 로고    scopus 로고
    • Therapeutic efficacy of alpha-1 antitrypsin augmentation therapy on the loss of lung tissue: An integrated analysis of 2 randomised clinical trials using computed tomography densitometry
    • Stockley RA, Parr DG, Piitulainen E, Stolk J, Stoel BC, Dirksen A. Therapeutic efficacy of alpha-1 antitrypsin augmentation therapy on the loss of lung tissue: an integrated analysis of 2 randomised clinical trials using computed tomography densitometry. Respir Res. 2010;11:136.
    • (2010) Respir Res , vol.11
    • Stockley, R.A.1    Parr, D.G.2    Piitulainen, E.3    Stolk, J.4    Stoel, B.C.5    Dirksen, A.6
  • 65
    • 84859787234 scopus 로고    scopus 로고
    • Alpha1-proteinase inhibitor (Human) in the treatment of hereditary emphysema secondary to alpha1-antitrypsin deficiency: Number and costs of years of life gained
    • Sclar DA, Evans MA, Robison LM, Skaer TL. Alpha1-proteinase inhibitor (human) in the treatment of hereditary emphysema secondary to alpha1-antitrypsin deficiency: number and costs of years of life gained. Clin Drug Investig. 2012;32(5):353–360.
    • (2012) Clin Drug Investig , vol.32 , Issue.5 , pp. 353-360
    • Sclar, D.A.1    Evans, M.A.2    Robison, L.M.3    Skaer, T.L.4
  • 66
    • 0037952677 scopus 로고    scopus 로고
    • Cost-effectiveness analysis of augmentation therapy for severe alpha1-antitrypsin deficiency
    • Gildea TR, Shermock KM, Singer ME, Stoller JK. Cost-effectiveness analysis of augmentation therapy for severe alpha1-antitrypsin deficiency. Am J Respir Crit Care Med. 2003;167(10):1387–1392.
    • (2003) Am J Respir Crit Care Med , vol.167 , Issue.10 , pp. 1387-1392
    • Gildea, T.R.1    Shermock, K.M.2    Singer, M.E.3    Stoller, J.K.4
  • 67
    • 0023784904 scopus 로고
    • Biochemical efficacy and safety of monthly augmentation therapy for alpha 1-antitrypsin deficiency
    • Hubbard RC, Sellers S, Czerski D, Stephens L, Crystal RG. Biochemical efficacy and safety of monthly augmentation therapy for alpha 1-antitrypsin deficiency. JAMA. 1988;260(9):1259–1264.
    • (1988) JAMA , vol.260 , Issue.9 , pp. 1259-1264
    • Hubbard, R.C.1    Sellers, S.2    Czerski, D.3    Stephens, L.4    Crystal, R.G.5
  • 68
    • 0030610568 scopus 로고    scopus 로고
    • Pharmacokinetic study of alpha1-antitrypsin infusion in alpha1-antitrypsin deficiency
    • Barker AF, Iwata-Morgan I, Oveson L, Roussel R. Pharmacokinetic study of alpha1-antitrypsin infusion in alpha1-antitrypsin deficiency. Chest. 1997;112(3):607–613.
    • (1997) Chest , vol.112 , Issue.3 , pp. 607-613
    • Barker, A.F.1    Iwata-Morgan, I.2    Oveson, L.3    Roussel, R.4
  • 69
    • 0028070026 scopus 로고
    • Failure to achieve adequate serum levels with monthly replacement therapy in alpha 1-antitrypsin deficiency
    • Cammarata SK, Stone CL, Carey JL, Eichenhorn MS. Failure to achieve adequate serum levels with monthly replacement therapy in alpha 1-antitrypsin deficiency. Chest. 1994;106(2):651–652.
    • (1994) Chest , vol.106 , Issue.2 , pp. 651-652
    • Cammarata, S.K.1    Stone, C.L.2    Carey, J.L.3    Eichenhorn, M.S.4
  • 70
    • 42949085601 scopus 로고    scopus 로고
    • Intravenous human plasma-derived augmentation therapy in alpha 1-antitrypsin deficiency: From pharmacokinetic analysis to individualizing therapy
    • Zamora NP, Pla RV, Del Rio PG, Margaleff RJ, Frias FR, Ronsano JB. Intravenous human plasma-derived augmentation therapy in alpha 1-antitrypsin deficiency: from pharmacokinetic analysis to individualizing therapy. Ann Pharmacother. 2008;42(5):640–646.
    • (2008) Ann Pharmacother , vol.42 , Issue.5 , pp. 640-646
    • Zamora, N.P.1    Pla, R.V.2    Del Rio, P.G.3    Margaleff, R.J.4    Frias, F.R.5    Ronsano, J.B.6
  • 71
    • 33845227581 scopus 로고    scopus 로고
    • Alpha-1-antitrypsin deficiency: Optimal therapeutic regimen based on population pharmacokinetics
    • Soy D, de la Roza C, Lara B, Esquinas C, Torres A, Miravitlles M. Alpha-1-antitrypsin deficiency: optimal therapeutic regimen based on population pharmacokinetics. Thorax. 2006;61(12):1059–1064.
    • (2006) Thorax , vol.61 , Issue.12 , pp. 1059-1064
    • Soy, D.1    De La Roza, C.2    Lara, B.3    Esquinas, C.4    Torres, A.5    Miravitlles, M.6
  • 72
    • 0035096397 scopus 로고    scopus 로고
    • Alpha One Foundation Research Network Registry I. The direct medical costs of alpha(1)-antitrypsin deficiency
    • Mullins CD, Huang X, Merchant S, Stoller JK; Alpha One Foundation Research Network Registry I. The direct medical costs of alpha(1)-antitrypsin deficiency. Chest. 2001;119(3):745–752.
    • (2001) Chest , vol.119 , Issue.3 , pp. 745-752
    • Mullins, C.D.1    Huang, X.2    Merchant, S.3    Stoller, J.K.4
  • 73
    • 0141558799 scopus 로고    scopus 로고
    • Major components of the direct medical costs of alpha1-antitrypsin deficiency
    • Mullins CD, Wang J, Stoller JK. Major components of the direct medical costs of alpha1-antitrypsin deficiency. Chest. 2003;124(3):826–831.
    • (2003) Chest , vol.124 , Issue.3 , pp. 826-831
    • Mullins, C.D.1    Wang, J.2    Stoller, J.K.3
  • 74
    • 0036078175 scopus 로고    scopus 로고
    • Societal implications of the pharmacoeconomics of alpha1-antitrypsin deficiency
    • Mullins CD, Blatt L, Wang J. Societal implications of the pharmacoeconomics of alpha1-antitrypsin deficiency. Expert Rev Pharmacoecon Outcomes Res. 2002;2(3):243–249.
    • (2002) Expert Rev Pharmacoecon Outcomes Res , vol.2 , Issue.3 , pp. 243-249
    • Mullins, C.D.1    Blatt, L.2    Wang, J.3
  • 75
    • 54249121344 scopus 로고    scopus 로고
    • Medical and Scientific Advisory Committee of the Alpha-1 Foundation. Alpha1-antitrypsin augmentation therapy for PI*MZ heterozygotes: A cautionary note
    • Sandhaus RA, Turino G, Stocks J, et al; Medical and Scientific Advisory Committee of the Alpha-1 Foundation. Alpha1-antitrypsin augmentation therapy for PI*MZ heterozygotes: a cautionary note. Chest. 2008;134(4):831–834.
    • (2008) Chest , vol.134 , Issue.4 , pp. 831-834
    • Sandhaus, R.A.1    Turino, G.2    Stocks, J.3
  • 76
    • 84894271236 scopus 로고    scopus 로고
    • Clarification of the risk of chronic obstructive pulmonary disease in alpha1-antitrypsin deficiency PiMZ heterozygotes
    • Molloy K, Hersh CP, Morris VB, et al. Clarification of the risk of chronic obstructive pulmonary disease in alpha1-antitrypsin deficiency PiMZ heterozygotes. Am J Respir Crit Care Med. 2014;189(4):419–427.
    • (2014) Am J Respir Crit Care Med , vol.189 , Issue.4 , pp. 419-427
    • Molloy, K.1    Hersh, C.P.2    Morris, V.B.3
  • 77
    • 0030446288 scopus 로고    scopus 로고
    • Clinical features of individuals with PI*SZ phenotype of alpha 1-antitrypsin deficiency. Alpha 1-Antitrypsin Deficiency Registry Study Group
    • (6 pt 1)
    • Turino GM, Barker AF, Brantly ML, et al. Clinical features of individuals with PI*SZ phenotype of alpha 1-antitrypsin deficiency. Alpha 1-Antitrypsin Deficiency Registry Study Group. Am J Respir Crit Care Med. 1996;154(6 pt 1):1718–1725.
    • (1996) Am J Respir Crit Care Med , vol.154 , pp. 1718-1725
    • Turino, G.M.1    Barker, A.F.2    Brantly, M.L.3
  • 78
    • 0030448774 scopus 로고    scopus 로고
    • Lung transplantation for alpha 1-antitrypsin deficiency emphysema
    • Trulock EP. Lung transplantation for alpha 1-antitrypsin deficiency emphysema. Chest. 1996;110(6 suppl):284S–294S.
    • (1996) Chest , vol.110 , Issue.6 , pp. 284S-294S
    • Trulock, E.P.1
  • 79
    • 0028351634 scopus 로고
    • The proteinase-antiproteinase balance in alpha-1-proteinase inhibitor-deficient lung transplant recipients
    • (4 pt 1)
    • King MB, Campbell EJ, Gray BH, Hertz MI. The proteinase-antiproteinase balance in alpha-1-proteinase inhibitor-deficient lung transplant recipients. Am J Respir Crit Care Med. 1994;149(4 pt 1):966–971.
    • (1994) Am J Respir Crit Care Med , vol.149 , pp. 966-971
    • King, M.B.1    Campbell, E.J.2    Gray, B.H.3    Hertz, M.I.4
  • 80
    • 0028916761 scopus 로고
    • Preliminary evidence that augmentation therapy diminishes degradation of cross-linked elastin in alpha-1-antitrypsin-deficient humans
    • Stone PJ, Morris TA 3rd, Franzblau C, Snider GL. Preliminary evidence that augmentation therapy diminishes degradation of cross-linked elastin in alpha-1-antitrypsin-deficient humans. Respiration. 1995;62(2):76–79.
    • (1995) Respiration , vol.62 , Issue.2 , pp. 76-79
    • Stone, P.J.1    Morris, T.A.2    Franzblau, C.3    Snider, G.L.4
  • 81
    • 0033739182 scopus 로고    scopus 로고
    • Augmentation therapy reduces frequency of lung infections in antitrypsin deficiency: A new hypothesis with supporting data
    • Lieberman J. Augmentation therapy reduces frequency of lung infections in antitrypsin deficiency: a new hypothesis with supporting data. Chest. 2000;118(5):1480–1485.
    • (2000) Chest , vol.118 , Issue.5 , pp. 1480-1485
    • Lieberman, J.1
  • 82
    • 0035095548 scopus 로고    scopus 로고
    • Wissenschaftliche Arbeitsgemeinschaft zur Therapie von Lungenerkrankungen. Longitudinal follow-up of patients with alpha(1)-protease inhibitor deficiency before and during therapy with IV alpha(1)-protease inhibitor
    • Wencker M, Fuhrmann B, Banik N, Konietzko N; Wissenschaftliche Arbeitsgemeinschaft zur Therapie von Lungenerkrankungen. Longitudinal follow-up of patients with alpha(1)-protease inhibitor deficiency before and during therapy with IV alpha(1)-protease inhibitor. Chest. 2001;119(3):737–744.
    • (2001) Chest , vol.119 , Issue.3 , pp. 737-744
    • Wencker, M.1    Fuhrmann, B.2    Banik, N.3    Konietzko, N.4
  • 83
    • 77949329900 scopus 로고    scopus 로고
    • Alpha-1-antitrypsin augmentation therapy in deficient individuals enrolled in the Alpha-1 Foundation DNA and Tissue Bank
    • Tonelli AR, Rouhani F, Li N, Schreck P, Brantly ML. Alpha-1-antitrypsin augmentation therapy in deficient individuals enrolled in the Alpha-1 Foundation DNA and Tissue Bank. Int J Chron Obstruct Pulmon Dis. 2009;4:443–452.
    • (2009) Int J Chron Obstruct Pulmon Dis , vol.4 , pp. 443-452
    • Tonelli, A.R.1    Rouhani, F.2    Li, N.3    Schreck, P.4    Brantly, M.L.5
  • 84
    • 0034523704 scopus 로고    scopus 로고
    • Short-term supplementation therapy does not affect elastin degradation in severe alpha(1)-antitrypsin deficiency. The American–Italian AATD Study Group
    • Gottlieb DJ, Luisetti M, Stone PJ, et al. Short-term supplementation therapy does not affect elastin degradation in severe alpha(1)-antitrypsin deficiency. The American–Italian AATD Study Group. Am J Respir Crit Care Med. 2000;162(6):2069–2072.
    • (2000) Am J Respir Crit Care Med , vol.162 , Issue.6 , pp. 2069-2072
    • Gottlieb, D.J.1    Luisetti, M.2    Stone, P.J.3
  • 85
    • 0036606880 scopus 로고    scopus 로고
    • The effect of augmentation therapy on bronchial inflammation in alpha1-antitrypsin deficiency
    • Stockley RA, Bayley DL, Unsal I, Dowson LJ. The effect of augmentation therapy on bronchial inflammation in alpha1-antitrypsin deficiency. Am J Respir Crit Care Med. 2002;165(11):1494–1498.
    • (2002) Am J Respir Crit Care Med , vol.165 , Issue.11 , pp. 1494-1498
    • Stockley, R.A.1    Bayley, D.L.2    Unsal, I.3    Dowson, L.J.4
  • 86
    • 20744450475 scopus 로고    scopus 로고
    • Alpha1-antitrypsin deficiency
    • Stoller JK, Aboussouan LS. Alpha1-antitrypsin deficiency. Lancet. 2005;365(9478):2225–2236.
    • (2005) Lancet , vol.365 , Issue.9478 , pp. 2225-2236
    • Stoller, J.K.1    Aboussouan, L.S.2


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