402
Views
23
CrossRef citations to date
0
Altmetric
Brief Reviews

The role of augmentation therapy in alpha-1 antitrypsin deficiency

Pages 579-588 | Accepted 14 Dec 2010, Published online: 12 Jan 2011

References

  • Laurell CB, Eriksson S. The electrophoretic α1-globulin pattern of serum in α1-antitrypsin deficiency. Scand J Clin Lab Invest 1963;15:132-40
  • Eriksson S. Pulmonary emphysema and alpha1-antitrypsin deficiency. Acta Med Scand 1964;175:197-205
  • Eriksson S. Studies in alpha 1-antitrypsin deficiency. Acta Med Scand Suppl 1965;432:1-85
  • Kueppers F. Chronic obstructive pulmonary disease. In: King R, Rotter J, Motulsky A, eds. The Genetic Basis of Common Diseases. New York: Oxford University Press, 1992:222-39
  • de Serres FJ, Blanco I, Fernandez-Bustillo E. PI S and PI Z alpha-1 antitrypsin deficiency worldwide. A review of existing genetic epidemiological data. Monaldi Arch Chest Dis 2007;67:184-208
  • Fregonese L, Stolk J. Hereditary alpha-1-antitrypsin deficiency and its clinical consequences. Orphanet J Rare Dis 2008;3:16
  • Hogarth DK, Rachelefsky G. Screening and familial testing of patients for alpha 1-antitrypsin deficiency. Chest 2008;133:981-8
  • Kohnlein T, Welte T. Alpha-1 antitrypsin deficiency: pathogenesis, clinical presentation, diagnosis, and treatment. Am J Med 2008;121:3-9
  • Rachelefsky G, Hogarth DK. Issues in the diagnosis of alpha 1-antitrypsin deficiency. J Allergy Clin Immunol 2008;121:833-8
  • Wood AM, Stockley RA. Alpha one antitrypsin deficiency: from gene to treatment. Respiration 2007;74:481-92
  • Aboussouan LS, Stoller JK. Detection of alpha-1 antitrypsin deficiency: a review. Respir Med 2009;103:335-41
  • Petrache I, Hajjar J, Campos M. Safety and efficacy of alpha-1-antitrypsin augmentation therapy in the treatment of patients with alpha-1-antitrypsin deficiency. Biologics 2009;3:193-204
  • Heresi GA, Stoller JK. Augmentation therapy in alpha-1 antitrypsin deficiency. Expert Opin Biol Ther 2008;8:515-26
  • Wewers MD, Casolaro MA, Sellers SE, et al. Replacement therapy for alpha 1-antitrypsin deficiency associated with emphysema. N Engl J Med 1987;316:1055-62
  • 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. Eur Respir J 1997;10:2260-3
  • The Alpha-1-Antitrypsin Deficiency Registry Study Group. Survival and FEV1 decline in individuals with severe deficiency of alpha1-antitrypsin. Am J Respir Crit Care Med 1998;158:49-59
  • Wencker M, Fuhrmann B, Banik N, et al. Longitudinal follow-up of patients with alpha(1)-protease inhibitor deficiency before and during therapy with IV alpha(1)-protease inhibitor. Chest 2001;119:737-44
  • 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;160:1468-72
  • 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;33:1345-53
  • Barker AF, Iwata-Morgan I, Oveson L et al. Pharmacokinetic study of alpha1-antitrypsin infusion in alpha1-antitrypsin deficiency. Chest 1997;112:607-13
  • Hubbard RC, Sellers S, Czerski D, et al. Biochemical efficacy and safety of monthly augmentation therapy for alpha 1-antitrypsin deficiency. JAMA 1988;260:1259-64
  • American Thoracic Society-European Respiratory Society. American Thoracic Society/European Respiratory Society statement: standards for the diagnosis and management of individuals with alpha-1 antitrypsin deficiency. Am J Respir Crit Care Med 2003;168:818-900
  • Crystal RG. Alpha 1-antitrypsin deficiency, emphysema, and liver disease. Genetic basis and strategies for therapy. J Clin Invest 1990;85:1343-52
  • Soy D, de la Roza C, Lara B, et al. Population pharmacokinetics: simulations and individual dosage optimization of alpha-1-antitrypsin replacement therapy. Pharmacotherapy 2005;25:1500-1
  • Zamora NP, Pla RV, Del Rio PG, et al. Intravenous human plasma-derived augmentation therapy in alpha(1)-antitrypsin deficiency: from pharmacokinetic analysis to individualizing therapy. Ann Pharmacother 2008;42:640-6
  • Chapman KR, Stockley RA, Dawkins C, et al. Augmentation therapy for alpha1-antitrypsin deficiency: a meta-analysis. COPD 2009;6:177-84
  • Chapman KR, Bradi AC, Paterson D, et al. Slower lung function decline during augmentation therapy in patients with alpha 1-antitrypsin deficiency (A1ATD): results from the Canadian AIR registry. Am J Respir Crit Care Med 2005;A808
  • Gøtzsche PC, Johansen HK. Intravenous alpha-1 antitrypsin augmentation therapy for treating patients with alpha-1 antitrypsin deficiency and lung disease (Review). Cochrane Database Syst Rev 2010;7:CD007851
  • Ratner M. Cochrane meta-analysis on alpha-1 antitrypsin prompts furor. Nat Biotechnol 2010;28:885-6
  • Stockley RA, Parr DG, Piitulainen E, et al. 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
  • Koczulla A, Andress J, Bittkowski N, et al. Quality of life in persons with alpha-1-antitrypsin deficiency - data from the German Registry [abstract]. International Conference of the American Thoracic Society, Toronto, Ontario, Canada, May 16-21, 2008;#D36
  • Lieberman J. Augmentation therapy reduces frequency of lung infections in antitrypsin deficiency: a new hypothesis with supporting data. Chest 2000;118:1480-5
  • Stoller JK, Fallat R, Schluchter MD, et al. Augmentation therapy with alpha1-antitrypsin: patterns of use and adverse events. Chest 2003;123:1425-34
  • Wencker M, Banik N, Buhl R, et al. 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;11:428-33
  • Bernhardt T, Cortes R. Post license safety of Prolastin [abstract]. Annual Congress of the European Respiratory Society, Munich, Germany, Sep 02-06, 2006;#E2987
  • Stoller JK, Rouhani F, Brantly M, et al. Biochemical efficacy and safety of a new pooled human plasma alpha(1)-antitrypsin, Respitin. Chest 2002;122:66-74
  • Stocks JM, Brantly M, Pollock D, et al. Multi-center study: the biochemical efficacy, safety and tolerability of a new α1-proteinase inhibitor, Zemaira. COPD 2006;3:17-23
  • Bergman G, Romberg V, Hubsch A. Safety and tolerability of an alpha1-proteinase inhibitor: results of four studies [abstract]. Chest 2007;132:528b-9b
  • Cohn E, Strong L, Hughes W, et al. Preparation and properties of serum and plasma proteins. IV. A system for the separation into fractions of the protein and lipoprotein components of biological tissues and fluids. J Am Chem Soc 1946;68:459-75
  • Coan MH, Brockway WJ, Eguizabal H, et al. Preparation and properties of alpha 1-proteinase inhibitor concentrate from human plasma. Vox Sang 1985;48:333-42
  • Baxter Healthcare Corporation. Aralast [alpha1-proteinase inhibitor (human)]. Prescribing information, 2007
  • Baxter Healthcare Corporation. Aralast NP [alpha1-proteinase inhibitor (human)]. Prescribing information, 2010
  • Taniguchi T, Rolf J, Bhattacharya P, et al. Process for separating alpha1-proteinase inhibitor from cohn fraction IV1 and IV4 paste. In: Alpha Therapeutic Corporation, ed. Los Angeles, CA, 2001; U.S. patent number 6,284,874
  • Louie SG, Sclar DA, Gill MA. Aralast: a new alpha1-protease inhibitor for treatment of alpha-antitrypsin deficiency. Ann Pharmacother 2005;39:1861-9
  • Kolarich D, Turecek PL, Weber A, et al. Biochemical, molecular characterization, and glycoproteomic analyses of alpha(1)-proteinase inhibitor products used for replacement therapy. Transfusion 2006;46:1959-77
  • Cowden DI, Fisher GE, Weeks RL. A pilot study comparing the purity, functionality and isoform composition of alpha-1-proteinase inhibitor (human) products. Curr Med Res Opin 2005;21:877-83
  • Kueppers F, Patel U. Differences in microheterogeneity and non-therapeutic protein content of the three commercial preparations of purified human alpha1-proteinase inhibitor [abstract]. Chest 2005;128:262S
  • Heide K, Haupt H. Darstellung noch nicht therapeutisch angewandter Plasmaproteine. Behringwerk Mitteilungen 1964;43:161-93
  • Bao JJ, Sifers RN, Kidd VJ, et al. Molecular evolution of serpins: homologous structure of the human alpha 1-antichymotrypsin and alpha 1-antitrypsin genes. Biochemistry 1987;26:7755-9
  • Fagerhol MK. Quantitative studies on the inherited variants of serum alpha-1-antitrypsin. Scand J Clin Lab Invest 1969;23:97-103
  • Stocks J, Brantly M, Barker A. Comparable pharmacokinetic properties of two alpha1-proteinase inhibitors after single therapeutic doses [abstract]. Chest 2005;128:261S-b
  • Spencer L, Pollock D, Stocks J, et al. Lower respiratory tract biochemical efficacy of intravenous administration of a new form of highly purified alpha1-antitrypsin [abstract]. Chest 2005;128:261S-a
  • Stocks J, Brantly ML, Campos MA, et al. Pharmacokinetic (PK) Comparability of TAL6004 to Prolastin in alpha-1 antitrypsin (AAT) deficiency: results of a multicenter, randomized, double-blind, crossover study. Am J Respir Crit Care Med 2009;179:A3522
  • Brantly M. Alpha 1-antitrypsin genotypes and phenotypes. In: Crystal RG, ed. Alpha 1-antitrypsin Deficiency (Lung Biology in Health and Disease). New York, NY: Marcel Dekker, 1995;88:45-59
  • United Network for Organ Sharing (UNOS). www.unos.org. Accessed 03/28/2008
  • Zhang B, Lu Y, Campbell-Thompson M, et al. Alpha1-antitrypsin protects beta-cells from apoptosis. Diabetes 2007;56:1316-23
  • Koulmanda M, Budo E, Bonner-Weir S, et al. Modification of adverse inflammation is required to cure new-onset type 1 diabetic hosts. Proc Natl Acad Sci U S A 2007;104:13074-9
  • Dawkins P, Bayley D, Stockley R. Correlations of plasma C-reactive protein with outcome measures in alpha-1-antitrypsin deficiency [abstract]. International Conference of the American Thoracic Society, Toronto, Ontario, Canada, May 16-21, 2008;#D35
  • Senn O, Russi EW, Schindler C, et al. Circulating alpha1-antitrypsin in the general population: determinants and association with lung function. Respir Res 2008;9:35
  • Wilson A, Kwok L, Little F, et al. Lentivirus-based expression of human alpha-1 antitrypsin abrogates elastase-induced emphysema in mice [abstract]. International Conference of the American Thoracic Society, Toronto, Ontario, Canada, May 16-21, 2008;#D50
  • Wencker M, Schulte M, Seitz J, et al. High efficiency of Prolastin inhalation in patients with alpha-1 antitrypsin (AAT) deficiency and cystic fibrosis (CF) compared to healthy volunteers [abstract]. International Conference of the American Thoracic Society, Toronto, Ontario, Canada, May 16-21, 2008;#A60
  • Stockley RA, Bayley DL, Unsal I, et al. The effect of augmentation therapy on bronchial inflammation in 1-antitrypsin deficiency. Am J Respir Crit Care Med 2002;165:1494-8
  • Vaughan L, Lorier MA, Carrell RW. Alpha 1-Antitrypsin microheterogeneity. Isolation and physiological significance of isoforms. Biochim Biophys Acta 1982;701:339-45

Reprints and Corporate Permissions

Please note: Selecting permissions does not provide access to the full text of the article, please see our help page How do I view content?

To request a reprint or corporate permissions for this article, please click on the relevant link below:

Academic Permissions

Please note: Selecting permissions does not provide access to the full text of the article, please see our help page How do I view content?

Obtain permissions instantly via Rightslink by clicking on the button below:

If you are unable to obtain permissions via Rightslink, please complete and submit this Permissions form. For more information, please visit our Permissions help page.