Safety and Pharmacokinetics of 120 mg/kg versus 60 mg/kg Weekly Intravenous Infusions of Alpha-1 Proteinase Inhibitor in Alpha-1 Antitrypsin Deficiency: A Multicenter, Randomized, Double-Blind, Crossover Study (SPARK)

COPD: Journal of Chronic Obstructive Pulmonary Disease

Volumn 10, Issue 6, 2013, Pages 687-695

  Campos, Michael A ^a   Kueppers, Friedrich ^b   Stocks, James M ^c   Strange, Charlie ^d   Chen, Junliang ^e   Griffin, Rhonda ^e   Wang Smith, Laurene ^f   Brantly, Mark L ^g  

^a UNIVERSITY OF MIAMI MILLER SCHOOL OF MEDICINE   (United States)

^b TEMPLE UNIVERSITY HOSPITAL   (United States)

^c UNIVERSITY OF TEXAS HEALTH CENTER   (United States)

^d MEDICAL UNIVERSITY OF SOUTH CAROLINA   (United States)

^e Grifols Therapeutics Inc   (United States)

^f INDAPharma LLC   (United States)

^g UNIVERSITY OF FLORIDA COLLEGE OF MEDICINE   (United States)

Author keywords

Alpha 1 antitrypsin deficiency; Alpha 1 proteinase Inhibitor; Chronic obstructive pulmonary disease

Indexed keywords

ALPHA 1 ANTITRYPSIN;

ADULT; ALPHA 1 ANTITRYPSIN DEFICIENCY; AREA UNDER THE CURVE; ARTICLE; CHRONIC OBSTRUCTIVE LUNG DISEASE; CLINICAL ARTICLE; CONTROLLED STUDY; CONTUSION; CREATININE BLOOD LEVEL; CROSSOVER PROCEDURE; DOUBLE BLIND PROCEDURE; DRUG BLOOD LEVEL; DRUG DOSE COMPARISON; DRUG FATALITY; DRUG SAFETY; DRUG TOLERABILITY; FEMALE; GLUCOSE BLOOD LEVEL; HUMAN; IMMUNOGENICITY; MALE; MAXIMUM PLASMA CONCENTRATION; MULTICENTER STUDY; PRIORITY JOURNAL; PROTEINURIA; RANDOMIZED CONTROLLED TRIAL; ROSACEA; SIDE EFFECT; SUBCUTANEOUS NODULE; THROMBOCYTOPENIA; URINARY TRACT INFECTION;

ADOLESCENT; ADULT; AGED; ALPHA 1-ANTITRYPSIN; ALPHA 1-ANTITRYPSIN DEFICIENCY; AREA UNDER CURVE; COHORT STUDIES; CROSS-OVER STUDIES; DOSE-RESPONSE RELATIONSHIP, DRUG; DOUBLE-BLIND METHOD; DRUG ADMINISTRATION SCHEDULE; FEMALE; HUMANS; INFUSIONS, INTRAVENOUS; MALE; MIDDLE AGED; SERINE PROTEINASE INHIBITORS; TREATMENT OUTCOME; YOUNG ADULT;

EID: 84887955432     PISSN: 15412555     EISSN: 15412563     Source Type: Journal    
DOI: 10.3109/15412555.2013.800852     Document Type: Article

References (23)

1. American Th oracic Society, European Respiratory Society. American Th oracic Society/European Respiratory Society Statement. Am J Respir Crit Care Med 2003; 168(7):818-900.
2. Gadek JE, Fells GA, Zimmerman RL, Rennard SI, Crystal RG. Antielastases of the human alveolar structures. Implications for the protease-Antiprotease theory of emphysema. J Clin Invest 1981; 68(4):889-898.
3. Wewers MD, Casolaro MA, Sellers SE, Swayze SC, McPhaul KM, Wittes JT, Crystal RG. Replacement therapy for alpha1- antitrypsin defi ciency associated wiThemphysema. N Engl J Med 1987; 316(17):1055-1062.
4. Gadek JE, Klein HG, Holland PV, Crystal RG. Replacement therapy of alpha 1-Antitrypsin defi ciency. Reversal of proteaseantiprotease imbalance within the alveolar structures of PiZ subjects. J Clin Invest 1981; 68(5):1158-1165.
5. Prolastin-C [prescribing information]. Research Triangle Park, NC: Grifols Therapeutics, Inc.; 2012.
6. Mohanka M, Khemasuwan D, Stoller JK. A review of augmentation therapy for alpha-1 antitrypsin defi ciency. Expert Opin Biol Ther 2012; 12(6):685-700.
7. Gotzsche PC, Johansen HK. Intravenous alpha-1 antitrypsin augmentation therapy for treating patients with alpha-1 antitrypsin defi ciency and lung disease. Cochrane Database Syst Rev 2010(7):CD007851. doi: 10.1002/14651858. CD007851.pub2
8. 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 defi ciency? Wissenschaftliche Arbeitsgemeinschaft zur Therapie von Lungenerkrankungen (WATL) alpha1-AT study group. Eur Respir J 1997; 10(10):2260-2263.
9. The Alpha-1-Antitrypsin Defi ciency Registry Study Group. Survival and FEV1 decline in individuals with severe defi ciency of ?1-Antitrypsin. Am J Respir Crit Care Med 1998; 158(1):49-59.
10. Wencker M, Fuhrmann B, Banik N, Konietzko N. Longitudinal follow-up of patients with alpha(1)-protease inhibitor defi ciency before and during therapy with IV alpha(1)-protease inhibitor. Chest 2001; 119(3):737-744.
11. 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 alpha1- antitrypsin defi ciency. Eur Respir J 2009; 33(6):1345-1353.
12. McElvaney NG, Stoller JK, Buist AS, Prakash UB, Brantly ML, Schluchter MD, Crystal RD. Baseline characteristics of enrollees in the National Heart, Lung and Blood Institute Registry of alpha 1-Antitrypsin defi ciency. Alpha 1-Antitrypsin Defi ciency Registry Study Group. Chest 1997; 111(2):394-403.
13. Gottlieb DJ, Luisetti M, Stone PJ, Allegra L, Cantey-Kiser JM, Grassi C, Snider GL. Short-term supplementation therapy does not aff ect elastin degradation in severe alpha(1)-Antitrypsin defi ciency. The American-Italian AATD Study Group. Am J Respir Crit Care Med 2000; 162(6):2069-2072.
14. Stoller JK, Rouhani F, Brantly M, Shahin S, Dweik RA, Stocks JM, Clausen J, Campbell E, Norton F. Biochemical efficacy and safety of a new pooled human plasma alpha(1)-Antitrypsin, Respitin. Chest 2002;122(1):66-74.
15. Campos MA, Alazemi S, Zhang G, Wanner A, Salathe M, Baier H, Sandhaus RA. Exacerbations in subjects with alpha-1 antitrypsin defi ciency receiving augmentation therapy. Respir Med 2009; 103(10):1532-1539.
16. Kohansal R, Martinez-Camblor P, Agusti A, Buist AS, Mannino DM, Soriano JB. The natural history of chronic airfl ow obstruction revisited: an analysis of the Framingham off spring cohort. Am J Respir Crit Care Med 2009; 180(1):3-10.
17. Vestbo J, Hurd SS, Agust AG, Jones PW, Vogelmeier C, Anzueto A, Barnes PJ, Fabbri LM, Martinez FJ, Nishimura M, Stockley RA, Sin DD, Rodriguez-Roisin R. Global strategy for the diagnosis, management, and prevention of chronic obstructive pulmonary disease: GOLD executive summary. Am J Respir Crit Care Med. 2013;187(4):347-365.
18. Barker AF, Siemsen F, Pasley D, DSilva R, Buist AS. Replacement therapy for hereditary alpha1-Antitrypsin defi ciency. A program for long-term administration. Chest 1994; 105(5):1406-1410.
19. Barker AF, Iwata-Morgan I, Oveson L, Roussel R. Pharmacokinetic study of alpha1-Antitrypsin infusion in alpha1-Antitrypsin defi ciency. Chest 1997; 112(3):607-613.
20. Grifols Therapeutics Inc. The comparison of the pharmacokinetic, safety and tolerability of alpha-1 MP and prolastin in adult alpha1-Antitrypsin defi cient patients (ChAMP). In: ClinicalTrials.gov. Bethesda (MD): National Library of Medicine (US). 2000-2013. Available from: http://clinicaltrials.gov/ct2/show/ NCT00295061 http://clinicaltrials. gov/ct2/show/NCT00295061. Identifi er: NCT00295061
21. Stocks JM, Brantly ML, Wang-Smith L, Campos MA, Chapman KR, Kueppers F, Sandhaus RA, Strange C, Turino G. Pharmacokinetic comparability of Prolastin?-C and Prolastin? in alpha(1)-Antitrypsin defi ciency: a randomized study. BMC Clin Pharmacol 2010; 10:13.
22. 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(5 Pt 1):1468-1472.
23. Hubbard RC, Sellers SS, Czerski DD, Stephens LL, Crystal RG. Biochemical efficacy and safety of monthly augmentation therapy for ?1-Antitrypsin defi ciency. JAMA 1988; 260(9): 1259-1264.