References
- JanciauskieneSNitaISubramaniyamDLiQLanacasterJRMatalonSAlpha-1 antitrypsin inhibits the activity of the matriptasa catalytic domain in vitroAm J Respir Cell Mol Biol200839 631 63718723439
- PetracheIFijalkowskaIMedlerTRAlpha-1 antitrypsin inhibits caspase-3 activity preventing lung endothelial cell apoptosisAm J Pathol2006169 1155 116617003475
- CarrollTO’ConnorCRevesEMcElvaneyNAlpha-1 antitrypsin deficiency: a genetic risk factor for COPDCOPD Current Concepts and PracticeOngKian-ChungDr978-953-51-0163-5
- GooptuBLomasDAConformational Pathology of the Serpins: themes, variations and therapeutic strategiesAnnu Rev Biochem200978 147 17619245336
- PerlmutterDHBrodskyJLBalisteriWFTrapnellBCMolecular pathogenesis of alpha-1-antitrypsin deficiency associated liver disease: a meeting reviewHepatology200745 1313 132317464974
- JanciuskieneSMBalsRKoczullaRVolgelmeierCKöhnleinTWelteTThe discovery of alpha-1 antitrypsin and its role in health and diseaseRespir Med20111058 1129 113921367592
- ShowsTBMcAlpinePJThe 1981 catalogue of assigned human genetic markers and report of the nomenclature committee. Oslo Conference: Sixth International Workshop on human Gene MppingCytogenet Cell Genet198232 221 2457140365
- VidalRBlancoICasasFJardíRMiravitllesMDiagnoses and treatment of alpha-1 antitrypsin deficiencyArch Bronconeumol20064212 645 65917178069
- Alpha-1 antitrypsin deficiency Task forceAmerican Thoracic Society/European Respiratory Society Statement: Standards for the diagnosis and management of individuals with alpha-1 antitrypsin deficiencyAm J Respir Crit Care Med2003168 818 90014522813
- De SerresFBlancoIFernández-BustilloEPI*S and PI*Z Alpha-1 antitrypsin deficiency worldwide. A review of existing genetic epidemiological dataMonaldi Arch Chest Dis2007674 184 20818309698
- TurinoGMBarkerAFBrantlyMLCohenABConellyRPCrystalRGClinical features of individuals with PI*SZ phenotype of alpha-1 antitrypsin deficiency. Alpha-1 antitrypsin deficiency Registry Study GroupAm J Respir Crit Care Med1996154 1718 17258970361
- LeePGildeaTRStollerJKEmphysema in non smokers: alpha-1- antitrypsin deficiency and other causesCleve Cli J Med200269 928 929
- CoxDLevisonHEmphysema of early onset associated with a complete deficiency of alpha-1 antitrypsin (null homozygotes)Am Rev Respir Dis1988137 371 3753257661
- DeMeoDLSilvermanEKAlpha-1 antitrypsin defieicny. Genetic aspects of alpha-1 antitrypsin deficiency: phenotypes and genetic modifiers of emphysema riskThorax200459 259 26414985567
- SeersholmNKok-JensenADirksenADecline in FEV1 among patients with severe hereditary alpha-1 antitrypsin deficiency type PiZAm J Respir Crit Care Med1995152 1922 19258520756
- PiituilainenETornlingGErikssonSEnviromental correlates of impaired lung function in non-smokers with severe alpha-1 antitrypsin deficiency (PiZZ)Thorax199853 939 94310193391
- DeMeoDLSandhausRABarkerAFDeterminants of airflow obstruction in severe alpha-1 antitrypsin deficiencyThorax200762 806 81317389752
- SeersholmNKok-JensenADirksenASurvival of patients with severe alpha-1 antitrypsin deficiency with special reference to non-index casesThorax199494 695 698
- TanashHANilssonPMNilssonJAPiituilainenEClinical course and prognosis of never smokers with severe AATD (PiZ)Thorax200863 1091 109518682522
- GadekJEKlinHGHhollandPVCrystalRGReplacement therapy of alpha-1 antitrypsin deficiency. Reversal of protease-antiprotease imbalance within the alveolar structures of PiZ subjectsJ Clin Invest198168 1158 11657028785
- From NIH: Intravenous replacement therapy for patients with severe alpha-1 antitrypsin deficiencyJAMA1982248 16936981713
- WewersMDCasolaroMASellersSESwayzeSCMc PhaulKMWittesJTReplacement therapy for alpha-1 antitrypsin deficiency associated with emphysemaN Engl J Med198731617 1055 10623494198
- KueppersFThe role of augmentation therapy in alpha-1 antitrypsin deficiencyCurr Med Res Opin201127 579 58821226542
- MohankaMKhemasuwanDStollerJKA review of augmentation therapy for alpha-1 antitrypsin deficiencyExpert Opin Biol Ther201212 685 70022500781
- GauvainCMornexJFPisonCHealth related quality of life in patients with alpha-1 antitrypsin deficiency: the French ExperienceCOPD20151 46 51
- MordwinkinNMLouieSGAralast: an alpha 1-protease inhibitor for the treatment of alpha-1 antitrypsin deficiencyExpert Opin Pharmacother2007815 2609 261417931094
- StollerJKRouhaniFBrantlyMBiochemical efficacy and safety of a new pooled human alpha (1)-antitrypsin, RespitinChest20021221 66 7412114340
- Respreeza EMA/554260/2016EPAR Summary for the publicEuropean Medicines AgencyLondon2016
- StocksJMBrantlyMPollockDMulticenter study: the biochemical efficacy, safety and tolerability of a new alpha-1 proteinase inhibitor, ZemairaCOPD200631 17 2317175661
- BlancoILaraBDe SerresFEfficacy of alpha-1 antitrypsin augmentation therapy in conditions other than pulmonary emphysemaOrphanet Journal of Rare Diseases2011 6 14
- TeschlerHLong-term experience in the treatment of alpha-1 antitrypsin deficiency: 25 years of augmentation therapyEur Respir Rev201524 46 5125726554
- CoanMNBrockwayWJEguizabalHPreparation and properties of alpha-1 proteinase inhibitor concentrate from human plasmaVox Sang1985486 333 3423874474
- BurnoutTConstansJClercADescampsJMartinacheLGoudemandMBiochemical and biological properties of an alpha-1 antitrypsin concentrateVox Sang1986524 291 297
- StocksJMBrantlyMLWang-SmithLPharmacokinetic comparability of Prolastin®-C to Prolastin® in alpha-1 antitrypsin deficiency: a raondmized studyBMC Clin Pharmacol201010 1310.1186/1472-6904-10-1320920295
- SoyDde la RozaCLaraBEsquinasCTorresAMiravitllesMAlpha-1 antitrypsin deficiency: optimal therapeutic regimen based on population pharmacokineticsThorax200661 1059 106416928711
- WrightGCarverACottomDHigh level expression of active human alpha-1 antitrypsin in the milk of transgenic sheepBiotechnology19919 830 8341367357
- SpencerLTHumphriesJEBrantlyMLTransgenic Human Alpha-1 antitrypsin Study GroupAntibody response to aerosolized transgenic human alpha-1 antitrypsinN engl J Med2005352 2030 203115888711
- RossDBrownTHarperRPamarthiMNixonJBromirskiJProduction and characterization of a novel human recombinant alpha-1 antitrypsin in PERC. C6 cellsJ Biotechnol2012162 262 27323036927
- HubbardRCBrantlyMLSellerSEAntineutrophilic elastase defenses of the lower respiratory tract in alpha-1 antitryspin deficiency directly augmented with aerosol of alpha-1 antitrypsinAnn Intern Med1989111 206 2122787611
- SiekmeierRLung deposition of inhaled alpha-1-proteinase inhibitor (alpha1-PI) problems and experience of alpha1-PI inhalation therapy in patients with hereditary alpha1-PI deficiency and cystic fibrosisEur J Med Res201015 164 17421147646
- HubbardRCSellersSCzerskiDStephensLCristalRGBiochemical efficacy and safety of monthly augmentation therapy for alpha-1 antitrypsin deficiencyJAMA19882609 1259 12643261353
- StockleyRABayleyDLUnsalIDowsonWThe effect of augmentation therapy on bronchial inflammation in alpha-1 antitrypsin deficiencyAm J Respir Crit Care Med2002165 1494 149812045122
- MaSLinYYHeJRouhaniFNBrantlyMTurinoGMAlpha-1 antitrypsin augmentation therapy and biomarkers of elastin degradationCOPD201310 473 48123560990
- SeersholmNWenckerMBanikNDoes alpha-1 augmentation therapy slow the annual decline in FEV1 in patients with severe hereditary alpha-1 antitrypsin deficiency? Wissenschaftliche Arbeitsgemeinschaft Zup-Therapie Von Lunge-Nerkrankungen (WATL) Alpha1-AT study groupEur Respir J199710 2260 22639387950
- The alpha-1-antitrypsin Deficiency Registry Study GroupSurvival and FEV1 decline in individuals with severe deficiency of alpha-1 antitrypsinAm J Respir Crit Care Med1998158 49 599655706
- DirksenADijkmanJHMadsenFA randomized clinical trial of alpha-1 antitrypsin augmentation therapyAm J Respir Crit Care Med1999160 1468 147210556107
- LiebermanJAugmentation therapy reduces frequency og lung infections in antitrypsin deficiency: a new hipothesis with supporting dataChest2000118 1480 148511083705
- WenckerMFurhmannBBanikNKonietzkoNLongitudinal follow-up of patients with alpha-1 antiportease inhibitor deficiency before and during therapy with IV alpha (1)-protease inhibitorChest2001119 737 74411243951
- TonelliARRouhaniFLiNSchreckPBrantlyMLAlpha-1 antitrypsin augmentation therapy in deficient individuals enrolled in the Alpha-1 Foundation DNA and tissue bankInt J COPD20094 443 452
- DirksenAPiitulainenEParrDGExploring the role of CT densitometry: a randomised study of augmentation therapy in alpha1-antitrypsin deficiencyEur Respir J2009336 1345 135319196813
- ChapmanKRStockleyRADawkinsCWilkesMMNavickisRJAugmentation therapy for alpha-1 antitrypsin deficiency: a meta-analysisCOPD20096 177 18419811373
- GøtzschePCJohansenHKIntravenous alpha-1 antitrypsin augmentation therapy for treating patients with alpha-1 antitrypsin deficiency and lung disease (Review)Cochrane Database Syst Rev20107 CD007851
- StockleyRAParrDGPiituilainenEStolkJStoelBBCDirksenATherapeutic efficacy of alpha-1 antitrypsin augmentation therapy on the loss of lung tissue: an integrated analysis of 2 randomised clinical trials using computed tomography densitometryRespir Res201011 13620920370
- Barros-TizónJCTorresMLBlancoIMartínezMTInvestigators of the rEXA study groupReduction of severe exacerbations and hospitalization-derived costs in alpha-1-antitrypsin-deficient patients treated with alpha-1-antitrypsin augmentation therapyTher Adv Respir Dis20126 67 78.922354900
- ChapmanKRBurdonJGPiituilainenERAPID Trial Study GroupIntravenous augmentation treatment and lung density in severe alpha-1 antitrypsin deficiency (RAPID): a randomized, double-blind, placebo-controlled trialLancet2015386 360 36826026936
- GøtzschePCJohansenHKIntravenous alpha-1 antitrypsin augmentation therapy for treating patients with alpha-1 antitrypsin deficiency and lung disease (review)Cochrane Database of Syst Rev20169 CD00785127644166
- McElvaneyBurdonJHolmesMGlanvilleAWarkPThompsonPLong-term efficacy and safety of alpha-1 proteinase inhibitor treatment for emphysema cause by severe alpha-1 antitrypsin: an open label extension trial (RAPID-OLE)The Lancet Respiratory2016 101016/52213-2006(16)30430-1
- CasasFHernandezJMGeneral treatment and intravenous treatment with alpha-1 antitrypsin. Chapter 12. Deficit de alfa-1 antitripsina: fisiopatologia, enfermedades relacionadas, diagnostico y tratamientoEditorial Respira978-84-944876-8-2
- SchluchterMDStollerJKBarkerAFFeasibility of a clinical trial of augmentation therapy for alpha(1)-antitrypsin deficiency. The Alpha 1-Antitrypsin Deficiency Registry Study GroupAm J Respir Crit Care Med20001613 Pt 1 796 80110712324
- DowsonLJGuestPJStockleyRALongitudinal changes in physiological, radiological, and health status measurements in alpha (1)-antitrypsin deficiency and factors associated with declineAm J Respir Crit Care Med200116410 Pt 1 1805 180911734427
- StolkJNgWHBakkerMECorrelation between annual change in health status and computer tomography derived lung density in subjects with alpha1-antitrypsin deficiencyThorax20035812 1027 103014645966
- ParrDGStoelBCStolkJValidation of computed tomographic lung densitometry for monitoring emphysema in alpha1-antitrypsin deficiencyThorax2006616 485 49016537666
- HrubanRHMezianeMAZerhouniEAHigh resolution computed tomography of inflation-fixed lungs. Pathologic-radiologic correlation of centrilobular emphysemaAm Rev Respir Dis19871364 935 9403310774
- HayhurstMDMacNeeWFlenleyDCDiagnosis of pulmonary emphysema by computerised tomographyLancet198428398 320 3226146866
- GouldGAMacNeeWMcLeanACT measurements of lung density in life can quantitate distal airspace enlargement – an essential defining feature of human emphysemaAm Rev Respir Dis19881372 380 3923341629
- GevenoisPAde MaertelaerVDe VuystPComparison of computed density and macroscopic morphometry in pulmonary emphysemaAm J Respir Crit Care Med19951522 653 6577633722
- GevenoisPADe VuystPde MaertelaerVComparison of computed density and microscopic morphometry in pulmonary emphysemaAm J Respir Crit Care Med19961541 187 1928680679
- MullerNLStaplesCAMillerRR“Density mask”. An objective method to quantitate emphysema using computed tomographyChest1988944 782 7873168574
- GouldGARedpathATRyanMLung CT density correlates with measurements of airflow limitation and the diffusing capacityEur Respir J199142 141 1462044729
- KinsellaMMullerNLAbboudRTQuantitation of emphysema by computed tomography using a “density mask” program and correlation with pulmonary function testsChest1990972 315 3212298057
- DowsonLJNewallCGuestPJExercise capacity predicts health status in alpha (1)-antitrypsin deficiencyAm J Respir Crit Care Med20011634 936 94111282769
- JohannessenASkorgeTDBottaiMMortality by level of emphysema and airway wall thicknessAm J Respir Crit Care Med20131876 602 60823328525
- DawkinsPADowsonLJGuestPJPredictors of mortality in alpha1-antitrypsin deficiencyThorax20035812 1020 102614645964
- DowsonLJGuestPJHillSLHigh-resolution computed tomography scanning in alpha1-antitrypsin deficiency: relationship to lung function and health statusEur Respir J2001176 1097 110411491150
- KalenderWARienmullerRSeisslerWMeasurement of pulmonary parenchymal attenuation: use of spirometric gating with quantitative CTRadiology19901751 265 2682315492
- MoroniCMascalchiMCamiciottoliGComparison of spirometric-gated and -ungated HRCT in COPDJ Comput Assist Tomogr2003273 375 37912794602
- HoffmanEAReinhardtJMSonkaMCharacterization of the interstitial lung diseases via density-based and texture-based analysis of computed tomography images of lung structure and functionAcad Radiol20031010 1104 111814587629
- StolkJDirksenAvan der LugtAARepeatability of lung density measurements with low-dose computed tomography in subjects with alpha-1-antitrypsin deficiency-associated emphysemaInvest Radiol20013611 648 65111606842
- DirksenAMonitoring the progress of emphysema by repeat computed tomography scans with focus on noise reductionProc Am Thorac Soc200859 925 92819056718
- StoelBCPutterHBakkerMEVolume correction in computed tomography densitometry for follow-up studies on pulmonary emphysemaProc Am Thorac Soc200859 919 92419056717
- ShakerSBDirksenALaursenLCVolume adjustment of lung density by computed tomography scans in patients with emphysemaActa Radiol2004454 417 42315323394
- ParrDGDirksenAPiitulainenEExploring the optimum approach to the use of CT densitometry in a randomised placebo-controlled study of augmentation therapy in alpha 1-antitrypsin deficiencyRespir Res200910 7519678952
- Food and Drug AdministrationBlood Products Advisory Committee. Clinical and Surrogate Endpoints for Evaluating Efficacy of Alpha1-Proteinase Inhibitor (Human) Augmentation Therapy95th Meeting Issue SummaryGaithersburg, Maryland, USAJuly 20–21; 2009
- FischerAStegemannJScheuchGSiekmeierRNovel devices for individualized controlled inhalation can optimized aerosol therapy in efficacy, patient care and power of clinical trailsEur J Med Res200914 71 77
- GellerDEKesserKCThe I-neb adaptative aerosol delivery system enhances delivery of alpha-1 antitrypsin with controlled inhalationJ Aerosol Med Pulm Drug Deliv201023 S55 S5920373910
- SorrellsSCamprubiSGriffinRChenJAyguasanosaJSPARTA clinical trial design: exploring the efficacy and safety of two dose regimens of alpha-1 antitrypsin proteinase inhibitor augmentation therapy in alpha-1 antitrypsin deficiencyRespir Med20151094 490 49925727857
- HernandezJMFumeroSAlvarezASuccessful alpha-1 antitrypsin replacement therapy in a patient with alpha-1 antitrypsin deficiency and granulomatosis with polyangiitisRheumatology201352 755 75722923761
- GøtzschePCJohansenHKIntravenous alpha-1 antitrypsin augmentation therapy for treating patients with alpha-1 antitrypsin deficiency and lung disease (Review)Cochrane Database Syst Rev20107 CD007851 Available from: http://www.alpha1.org/Portals/0/Response-Cochrane.pdf
- StollerJKFallatRSchluchterMDAugmentation Therapy with alpha-1 anitrypsin: patterns of use and adverse eventsChest20031235 1425 143412740257
- WenckerMBbanikNJulRSeidelRKonietzkoNLong term treatment of alpha-1 antitrypsin deficiency related pulmonary emphysema with human alpha-1 antitrypsin. Wissenshaftliche Arbeitsgemeinschaft zur Therapie von Lungenerkrankungen (WALT) alpha-1-AT-study groupEur Respir J1998112 428 4339551749
- MeyerFJWenckerMTeschlerHAcute allergic reaction and demonstration of specific IgE antibodies against alpha-1 proteiase inhibitorEur Respir J1998124 996 9979817182
- ChotirmallSAl-AlawiMMcEneryTMcElvaneyNAlpha-1 proteinase inhibitors for the treatment of alpha-1 antitrypsin deficiency: safety, tolerability and patients outcomesTher Clin Risk Manag201511 143 15125673994
- MarciniukDDHernandezPBalterMBourbeauJChapmanKRFordGTCanadian Thoracic Society COPD clinical assembly Alpha-1 Antitrypsin Deficiency Expert Working GroupAlpha-1 antitrypsin deficiency targeted testing and augmentation therapy: a Canadian Thoracic Society clnical practice guidelineCan Respir J201219 109 16
- MengaGMiravitllesMBlancoIEchazarretaSSorrochePBFernandezMENormativas de diagnostic y tratamiento del deficit de alfa-1 antitripsinaRAMR20141 28 46
- CasasFBlancoIMartinezMTBustamanteAMiravitllesMCadenasSIndications for active cases searches and intravenous alpha-1 antitrypsin treatment for patients wirh alpha-1 antitrypsin deficiency chronic pulmonary obstructive disease: an updateArch Bronchoneumol2015514 185 192
- LaraBMiravitllesMCasasFReview of national and international guidelines about diagnoses and treatment of alpha-1 antitrypsin deficiencyDeficit de alfa-1 antitripsina: fisiopatologia, enfermedades relacionadas, diagnostico y tratamiento. Editorial Respira978-84-944876-8-2