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

Respiratory Research

Volumn 11, Issue , 2010, Pages

  Stockley, Robert A ^a   Parr, David G ^b   Piitulainen, Eeva ^c   Stolk, Jan ^d   Stoel, Berend C ^d   Dirksen, Asger ^e  

^a UNIVERSITY HOSPITALS BIRMINGHAM NHS FOUNDATION TRUST   (United Kingdom)

^b UNIVERSITY HOSPITALS COVENTRY AND WARWICKSHIRE NHS TRUST   (United Kingdom)

^c LUND UNIVERSITY HOSPITAL   (Sweden)

^d LEIDEN UNIVERSITY MEDICAL CENTER   (Netherlands)

^e UNIVERSITY OF COPENHAGEN   (Denmark)

Author keywords

[No Author keywords available]

Indexed keywords

ALFALASTIN; ALPHA 1 ANTITRYPSIN; PLACEBO; UNCLASSIFIED DRUG;

ADULT; ARTICLE; COMPUTER ASSISTED TOMOGRAPHY; CONTROLLED STUDY; DENSITOMETRY; DISEASE COURSE; DOUBLE BLIND PROCEDURE; DRUG EFFICACY; FEMALE; FOLLOW UP; FORCED EXPIRATORY VOLUME; HUMAN; LUNG EMPHYSEMA; LUNG PARENCHYMA; LUNG VOLUME; MAJOR CLINICAL STUDY; MALE; RANDOMIZED CONTROLLED TRIAL; THERAPY EFFECT; TOTAL LUNG CAPACITY; AGED; COMPARATIVE STUDY; DRUG EFFECT; EMPHYSEMA; LUNG; METHODOLOGY; MIDDLE AGED; RADIOGRAPHY; RANDOMIZED CONTROLLED TRIAL (TOPIC); TREATMENT OUTCOME;

ADULT; AGED; ALPHA 1-ANTITRYPSIN; DENSITOMETRY; EMPHYSEMA; FEMALE; FOLLOW-UP STUDIES; HUMANS; LUNG; MALE; MIDDLE AGED; RANDOMIZED CONTROLLED TRIALS AS TOPIC; TOMOGRAPHY, X-RAY COMPUTED; TREATMENT OUTCOME;

EID: 77957273246     PISSN: 14659921     EISSN: 1465993X     Source Type: Journal    
DOI: 10.1186/1465-9921-11-136     Document Type: Article

References (28)

1. Snider GL, Lucey EC, Christensen TG, Stone PJ, Calore JD, Catanese A, Franzblau C. Emphysema and bronchial secretory cell metaplasia induced in hamsters by human neutrophil products. Am Rev Respir Dis 1984, 129:155-160.
2. Seersholm N, Wencker M, Banik N, Viskum K, Dirksen A, Kok-Jensen A, Konietzko N. Does alpha1-antitrypsin augmentation therapy slow the annual decline in FEV1 in patients with severe hereditary alpha1-antitrypsin deficiency?. Eur Respir J 1997, 10:2260-2263. 10.1183/09031936.97.10102260, 9387950.
3. Wewers MD, Casolaro MA, Sellers SE, Swayze SC, McPhaul KM, Wittes JT, Crystal RG. Replacement therapy for alpha 1-antitrypsin deficiency associated with emphysema. N Engl J Med 1987, 316:1055-1062. 10.1056/NEJM198704233161704, 3494198.
4. Schluchter MD, Stoller JK, Barker AF, Buist AS, Crystal RG, Donohue JF, Fallat RJ, Turino GM, Vreim CE, Wu MC. Feasibility of a clinical trial of augmentation therapy for alpha(1)-antitrypsin deficiency. The Alpha 1-Antitrypsin Deficiency Registry Study Group. Am J Respir Crit Care Med 2000, 161(3 pt 1):796-801.
5. Burrows B. A clinical trial of efficacy of antiproteolytic therapy: can it be done?. Am Rev Respir Dis 1983, 127:S42-S43.
6. 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. 10.1164/rccm.168.7.818, 14522813.
7. Dawkins PA, Dawkins CL, Wood AM, Nightingale PG, Stockley JA, Stockley RA. Rate of progression of lung function impairment in alpha-1-antitrypsin deficiency. Eur Respir J 2009, 33:1338-1344. 10.1183/09031936.00061208, 19164359.
8. Parr DG, Stoel BC, Stolk J, Stockley RA. Pattern of emphysema distribution in alpha1-antitrypsin deficiency influences lung function impairment. Am J Respir Crit Care Med 2004, 170:1172-1178. 10.1164/rccm.200406-761OC, 15306534.
9. Dirksen A, Friis M, Olesen KP, Skovgaard LT, Sørensen K. Progress of emphysema in severe alpha 1-antitrypsin deficiency as assessed by annual CT. Acta Radiol 1997, 38:826-832.
10. Parr DG, Stoel BC, Stolk J, Stockley RA. Validation of computed tomographic lung densitometry for monitoring emphysema in alpha1-antitrypsin deficiency. Thorax 2006, 61:485-490. 10.1136/thx.2005.054890, 2111224, 16537666.
11. Dowson LJ, Newall C, Guest PJ, Hill SL, Stockley RA. Exercise capacity predicts health status in alpha(1)-antitrypsin deficiency. Am J Respir Crit Care Med 2001, 163:936-941.
12. Dowson LJ, Guest PJ, Hill SL, Holder RL, Stockley RA. High-resolution computed tomography scanning in α1-antitrypsin deficiency: relationship to lung function and health status. Eur Respir J 2001, 17:1097-1104. 10.1183/09031936.01.00056501, 11491150.
13. Dawkins PA, Dowson LJ, Guest PJ, Stockley RA. Predictors of mortality in alpha 1-antitrypsin deficiency. Thorax 2003, 58:1020-1026. 10.1136/thorax.58.12.1020, 1746543, 14645964.
14. Dirksen A, Dijkman JH, Madsen F, Stoel B, Hutchison DC, Ulrik CS, Skovgaard LT, Kok-Jensen A, Rudolphus A, Seersholm N, Vrooman HA, Reiber JH, Hansen NC, Heckscher T, Viskum K, Stolk J. A randomized clinical trial of alpha 1-antitrypsin augmentation therapy. Am J Respir Crit Care Med 1999, 160:1468-1472.
15. Dirksen A, Piitulainen E, Parr DG, Deng C, Wencker M, Shaker SB, Stockley RA. Exploring the role of CT densitometry: a randomised study of augmentation therapy in alpha-1 antitrypsin deficiency. Eur Respir J 2009, 33:1345-1353. 10.1183/09031936.00159408, 19196813.
16. The Alpha-1-Antitrypsin Deficiency Registry Study Group Survival and FEV1 decline in individuals with severe deficiency of alpha 1-antitrypsin. Am J Respir Crit Care Med 1998, 158:49-59. The Alpha-1-Antitrypsin Deficiency Registry Study Group.
17. Ketelaars CA, Schlosser MA, Mostert R, Huyer Abu-Saad H, Halfens RJ, Wouters EF. Determinants of health-related quality of life in patients with chronic obstructive pulmonary disease. Thorax 1996, 51:39-43. 10.1136/thx.51.1.39, 472797, 8658367.
18. Hole DJ, Watt GC, Davey-Smith G, Hart CL, Gillis CR, Hawthorne VM. Impaired lung function and mortality risk in men and women: findings from the Renfrew and Paisley prospective population study. BMJ 1996, 313:711-715. 2352103, 8819439.
19. Holmes J, Stockley RA. Radiologic and clinical features of COPD patients with discordant pulmonary physiology: lessons from a-1-antitrypsin deficiency. Chest 2007, 132:909-915. 10.1378/chest.07-0341, 17573491.
20. Klein JS, Gamsu G, Webb WR, Golden JA, Müller NL. High-resolution CT diagnosis of emphysema in symptomatic patients with normal chest radiographs and isolated low diffusing capacity. Radiology 1992, 182:817-821.
21. Drummond MF, Wilson DA, Kanavos P, Ubel P, Rovira J. Assessing the economic challenges posed by orphan drugs. Int J Technol Assess Health Care 2007, 23:36-42. 10.1017/S0266462307051550, 17234015.
22. Saint SK, Bent S, Vittinghoff E, Grady D. Antibiotics in chronic obstructive pulmonary disease exacerbations: a meta-analysis. JAMA 1995, 273:957-960. 10.1001/jama.273.12.957, 7884956.
23. Stewart LA, Parmar MK. Meta-analysis of the literature or of individual patient data: is there a difference?. Lancet 1993, 341:418-422. 10.1016/0140-6736(93)93004-K, 8094183.
24. Riley RD, Lambert PC, bo-Zaid G. Meta-analysis of individual participant data: rationale, conduct, and reporting. BMJ 2010, 340:c221. 10.1136/bmj.c221, 20139215.
25. Liu PY, Swerdloff RS, Christenson PD, Handelsman DJ, Wang C. Rate, extent, and modifiers of spermatogenic recovery after hormonal male contraception: an integrated analysis. Lancet 2006, 367:1412-1420. 10.1016/S0140-6736(06)68614-5, 16650651.
26. Sin DD, Wu L, Anderson JA, Anthonisen NR, Buist AS, Burge PS, Calverley PM, Connett JE, Lindmark B, Pauwels RA, Postma DS, Soriano JB, Szafranski W, Vestbo J. Inhaled corticosteroids and mortality in chronic obstructive pulmonary disease. Thorax 2005, 60:992-997. 10.1136/thx.2005.045385, 1747271, 16227327.
27. Jadad AR, Moore RA, Carroll D, Jenkinson C, Reynolds DJ, Gavaghan DJ, McQuay HJ. Assessing the quality of reports of randomized clinical trials: Is blinding necessary?. Control Clin Trials 1996, 17:1-12. 10.1016/0197-2456(95)00134-4, 8721797.
28. Olivo SA, Macedo LG, Gadotti IC, Fuentes J, Stanton T, Magee DJ. Scales to assess the quality of randomized controlled trials: a systematic review. Phys Ther 2008, 88:156-175. Epub 2007 Dec 11, 10.2522/ptj.20070147, 18073267.