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)

References

• American Thoracic Society, European Respiratory Society. American Thoracic Society/European Respiratory Society Statement. Am J Respir Crit Care Med 2003; 168(7):818–900.
   PubMed Web of Science ®Google Scholar
• 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.
   PubMed Web of Science ®Google Scholar
• Wewers MD, Casolaro MA, Sellers SE, Swayze SC, McPhaul KM, Wittes JT, Crystal RG. Replacement therapy for alpha1-antitrypsin deficiency associated with emphysema. N Engl J Med 1987; 316(17):1055–1062.
   PubMed Web of Science ®Google Scholar
• 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.
   PubMed Web of Science ®Google Scholar
• Prolastin-C [prescribing information]. Research Triangle Park, NC: Grifols Therapeutics, Inc.; 2012.
   Google Scholar
• Mohanka M, Khemasuwan D, Stoller JK. A review of augmentation therapy for alpha-1 antitrypsin deficiency. Expert Opin Biol Ther 2012; 12(6):685–700.
   PubMed Web of Science ®Google Scholar
• Gotzsche PC, Johansen HK. Intravenous alpha-1 antitrypsin augmentation therapy for treating patients with alpha-1 antitrypsin deficiency and lung disease. Cochrane Database Syst Rev 2010(7):CD007851. doi: 10.1002/14651858.CD007851.pub2
   PubMed Web of Science ®Google Scholar
• 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? Wissenschaftliche Arbeitsgemeinschaft zur Therapie von Lungenerkrankungen (WATL) alpha1-AT study group. Eur Respir J 1997; 10(10):2260–2263.
   PubMed Web of Science ®Google Scholar
• The Alpha-1-Antitrypsin Deficiency Registry Study Group. Survival and FEV1 decline in individuals with severe deficiency of α1-antitrypsin. Am J Respir Crit Care Med 1998; 158(1):49–59.
   PubMed Web of Science ®Google Scholar
• Wencker M, Fuhrmann B, Banik N, Konietzko N. 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.
   PubMed Web of Science ®Google Scholar
• 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 deficiency. Eur Respir J 2009; 33(6):1345-1353.
   PubMed Web of Science ®Google Scholar
• 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 deficiency. Alpha 1-Antitrypsin Deficiency Registry Study Group. Chest 1997; 111(2):394–403.
   PubMed Web of Science ®Google Scholar
• Gottlieb DJ, Luisetti M, Stone PJ, Allegra L, Cantey-Kiser JM, Grassi C, Snider GL. 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.
   PubMed Web of Science ®Google Scholar
• 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.
   PubMed Web of Science ®Google Scholar
• Campos MA, Alazemi S, Zhang G, Wanner A, Salathe M, Baier H, Sandhaus RA. Exacerbations in subjects with alpha-1 antitrypsin deficiency receiving augmentation therapy. Respir Med 2009; 103(10):1532–1539.
   PubMed Web of Science ®Google Scholar
• Kohansal R, Martinez-Camblor P, Agusti A, Buist AS, Mannino DM, Soriano JB. The natural history of chronic airflow obstruction revisited: an analysis of the Framingham offspring cohort. Am J Respir Crit Care Med 2009; 180(1):3–10.
   PubMed Web of Science ®Google Scholar
• Vestbo J, Hurd SS, Agustí 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.
   PubMed Web of Science ®Google Scholar
• Barker AF, Siemsen F, Pasley D, D'Silva R, Buist AS. Replacement therapy for hereditary alpha1-antitrypsin deficiency. A program for long-term administration. Chest 1994; 105(5):1406–1410.
   PubMed Web of Science ®Google Scholar
• 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.
   PubMed Web of Science ®Google Scholar
• Grifols Therapeutics Inc. The comparison of the pharmacokinetic, safety and tolerability of alpha-1 MP and prolastin in adult alpha1-antitrypsin deficient 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>. Identifier: NCT00295061
   Google Scholar
• 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 deficiency: a randomized study. BMC Clin Pharmacol 2010; 10:13.
   PubMedGoogle Scholar
• 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.
   PubMed Web of Science ®Google Scholar
• Hubbard RC, Sellers SS, Czerski DD, Stephens LL, Crystal RG. Biochemical efficacy and safety of monthly augmentation therapy for α1-antitrypsin deficiency. JAMA 1988; 260(9): 1259–1264.
   PubMed Web of Science ®Google Scholar