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Safety and Efficacy of PrivigenA (R), a Novel 10% Liquid Immunoglobulin Preparation for Intravenous Use, in Patients with Primary Immunodeficiencies

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Stein, Mark R. Vermylen, Christiane [UCL] Nelson, Robert P. Church, Joseph A. Wasserman, Richard L. Bichler, Johann

The present study was designed to evaluate the efficacy and safety of a novel, 10% liquid formulation of intravenous immunoglobulin, stabilized with 250 mmol/L l-proline (PrivigenA (R)), in patients with primary immunodeficiency disease. Eighty adults and children diagnosed with common variable immunodeficiency or X-linked agammaglobulinemia received intravenous PrivigenA (R) infusions (200-888 mg/kg) at 3- or 4-week intervals over a 12-month period, according to their previously established maintenance dose. The primary endpoint was the annual rate of acute serious bacterial infections. There were six episodes of acute serious bacterial infections, corresponding to an annual rate of 0.08; the annual rate for all infections was 3.55. Mean serum IgG trough levels were between 8.84 and 10.27 g/L. A total of 1,038 infusions were administered, most of them at the maximum rate permitted (8.0 mg kg(-1) min(-1)). Temporally associated adverse events, possibly or probably related to study drug, occurred in 9% of infusions, either during or within 72 h after infusion end. PrivigenA (R) is well tolerated and effective for the treatment of primary immunodeficiency.

Document type Article de périodique (Journal article) – Clinical Trial, Phase III, Journal Article, Multicenter Study, Research Support, Non-U.S. Gov't
Access type Accès restreint
Publication date 2009
Language Anglais
Journal information "Journal of Clinical Immunology" - Vol. 29, no. 1, p. 137-44 (2009)
Peer reviewed yes
Publisher Springer/plenum Publishers (New York)
issn 0271-9142
e-issn 1573-2592
Publication status Publié
Affiliations UCL - Cliniques universitaires Saint-Luc
UCL - MD/GYPE - Département de gynécologie, d'obstétrique et de pédiatrie
UCL - (SLuc) Service d'hématologie et d'oncologie pédiatrique
MESH Subject Young Adult ; Middle Aged ; Male ; Immunologic Deficiency Syndromes - drug therapy ; Immunoglobulins, Intravenous - administration & dosage - adverse effects ; Immunoglobulin G - blood ; Humans ; Female ; Child, Preschool ; Child ; Aged ; Adult ; Adolescent
Keywords Primary immunodeficiency (PID) ; intravenous immunoglobulin (IVIG) ; X-linked agammaglobulinemia (XLA) ; common variable immunodeficiency (CVID)
Links
  1. Bruton OC. Agammaglobulinemia. Pediatrics 1952;9(6):722–8.
  2. Stiehm ER, Vaerman JP, Fudenberg HH. Plasma infusions in immunologic deficiency states: metabolic and therapeutic studies. Blood 1966;28(6):918–37.
  3. Ammann AJ, Ashman RF, Buckley RH, Hardie WR, Krantmann HJ, Nelson J, et al. Use of intravenous gamma-globulin in antibody immunodeficiency: results of a multicenter controlled trial. Clin Immunol Immunopathol. 1982;22(1):60–7. doi: 10.1016/0090-1229(82)90022-8 .
  4. CUNNINGHAM-RUNDLES CHARLOTTE, Efficacy of Intravenous Immunoglobulin in Primary Humoral Immunodeficiency Disease, 10.7326/0003-4819-101-4-435
  5. Buckley RH, Schiff RI. The use of intravenous immune globulin in immunodeficiency diseases. N Engl J Med. 1991;325(2):110–7.
  6. Bolli R, Spycher MO, Brügger R, Wüst B, Gennari K. IgG-dimer formation in liquid immunoglobulin preparations is inhibited by nicotinamide and other amphiphilic compounds. J Autoimmun. 1996;96(Suppl 1):96.
  7. Kumar TK, Samuel D, Jayaraman G, Srimathi T, Yu C. The role of proline in the prevention of aggregation during protein folding in vitro. Biochem Mol Biol Int. 1998;46(3):509–17.
  8. Schnorf J, Arnet B, Burek-Kozlowska A, Gennari K, Rohner R, Späth PJ, et al. Laboratory parameters measured during infusion of immunoglobulin preparations for intravenous use and related to tolerability. In: Kazatchkine MD, Morell A, editors. Intravenous immunoglobulin research and therapy. New York: Parthenon; 1996. p. 312–3.
  9. Kazatchkine MD, Kaveri SV. Immunomodulation of autoimmune and inflammatory diseases with intravenous immune globulin. N Engl J Med. 2001;345(10):747–55. doi: 10.1056/NEJMra993360 .
  10. Druml W, Heinzel G, Kleinberger G. Amino acid kinetics in patients with sepsis. Am J Clin Nutr. 2001;73(5):908–13.
  11. Spycher MO, Bolli R, Hodler G, Gennari K, Hubsch A, Späth P, et al. Well-tolerated liquid intravenous immunoglobulin G preparations (IVIG) have a low immunoglobulin G dimer (IgG-dimer) content. J Autoimmun. 1996;96(Suppl 1):96.
  12. Kempf C, Stucki M, Boschetti N. Pathogen inactivation and removal procedures used in the production of intravenous immunoglobulins. Biologicals 2007;35(1):35–42. doi: 10.1016/j.biologicals.2006.01.002 .
  13. Stucki M, Boschetti N, Schaefer W, Hostettler T, Kaesermann F, Nowak T, et al. Investigations of prion and virus safety of a new liquid IVIG product. Biologicals 2008;36:239–47. doi: 10.1016/j.biologicals.2008.01.004 .
  14. Conley ME, Notarangelo LD, Etzioni A. Diagnostic criteria for primary immunodeficiencies. Representing PAGID (Pan-American Group for Immunodeficiency) and ESID (European Society for Immunodeficiencies). Clin Immunol. 1999;93(3):190–7. doi: 10.1006/clim.1999.4799 .
  15. FDA. Safety, efficacy, and pharmacokinetic studies to support marketing of immune globulin intravenous (human) as replacement therapy for primary humoral immunodeficiency. Draft guidance for Industry. 2005.
  16. Johnson NL, Kotz S. Discrete distributions. New York: Wiley; 1969.
  17. Treatment experiences and preferences of patients with primary immune deficiency diseases. Survey results presented at the Immune Deficiency Foundation Meeting, 20 June 2003. 2003.
  18. Golding B. IGIV clinical endpoints. Committee Update. In Proceedings of the 65th Meeting; 16 March 2000; Silver Spring, MD. 2000.
  19. Berger M, Pinciaro PJ. Safety, efficacy, and pharmacokinetics of Flebogamma 5% [immune globulin intravenous (human)] for replacement therapy in primary immunodeficiency diseases. J Clin Immunol. 2004;24(4):389–96. doi: 10.1023/B:JOCI.0000029108.18995.61 .
  20. Church JA, Leibl H, Stein MR, Melamed IR, Rubinstein A, Schneider LC, et al. Efficacy, safety and tolerability of a new 10% liquid intravenous immune globulin [IGIV 10%] in patients with primary immunodeficiency. J Clin Immunol. 2006;26(4):388–95. doi: 10.1007/s10875-006-9025-3 .
  21. Ochs HD, Pinciaro PJ. Octagam 5%, an intravenous IgG product, is efficacious and well tolerated in subjects with primary immunodeficiency diseases. J Clin Immunol. 2004;24(3):309–14. doi: 10.1023/B:JOCI.0000025453.23817.3f .
  22. Roifman CM, Schroeder H, Berger M, Sorensen R, Ballow M, Buckley RH, et al. Comparison of the efficacy of IGIV-C, 10% (caprylate/chromatography) and IGIV-SD, 10% as replacement therapy in primary immune deficiency. A randomized double-blind trial. Int Immunopharmacol. 2003;3(9):1325–33. doi: 10.1016/S1567-5769(03)00134-6 .
  23. Work Loss Data Institute. Official disability guidelines. 2002;6/17(02). http://www.disabilitydurations.com/pr_repmdc.htm .
  24. Stiehm ER. Human intravenous immunoglobulin in primary and secondary antibody deficiencies. Pediatr Infect Dis J. 1997;16(7):696–707. doi: 10.1097/00006454-199707000-00012 .
  25. Roifman CM, Levison H, Gelfand EW. High-dose versus low-dose intravenous immunoglobulin in hypogammaglobulinaemia and chronic lung disease. Lancet 1987;1(8541):1075–7. doi: 10.1016/S0140-6736(87)90494-6 .
  26. Ochs HD, Fischer SH, Wedgwood RJ, Wara DW, Cowan MJ, Ammann AJ, et al. Comparison of high-dose and low-dose intravenous immunoglobulin therapy in patients with primary immunodeficiency diseases. Am J Med. 1984;76(3A):78–82. doi: 10.1016/0002-9343(84)90324-3 .
  27. Eijkhout Heleen W., van der Meer Jos W.M., Kallenberg Cees G.M., Weening Ron S., van Dissel Jaap T., Sanders Lieke A.M., Strengers Paul F.W., Nienhuis Henriët, Schellekens Peter Th.A., , The Effect of Two Different Dosages of Intravenous Immunoglobulin on the Incidence of Recurrent Infections in Patients with Primary Hypogammaglobulinemia : A Randomized, Double-Blind, Multicenter Crossover Trial, 10.7326/0003-4819-135-3-200108070-00008
  28. Baxter Healthcare. Gammagard liquid summary basis of approval. Baxter Healthcare; 2004.
  29. Flebogamma 5%, Prescribing Information. Grifols, December 2003.
  30. Brennan VM, Salome-Bentley NJ, Chapel HM. Prospective audit of adverse reactions occurring in 459 primary antibody-deficient patients receiving intravenous immunoglobulin. Clin Exp Immunol. 2003;133(2):247–51. doi: 10.1046/j.1365-2249.2003.02199.x .
  31. Pierce LR, Jain N. Risks associated with the use of intravenous immunoglobulin. Transfus Med Rev. 2003;17(4):241–51. doi: 10.1016/S0887-7963(03)00038-5 .
Bibliographic reference Stein, Mark R. ; Vermylen, Christiane ; Nelson, Robert P. ; Church, Joseph A. ; Wasserman, Richard L. ; et. al. Safety and Efficacy of PrivigenA (R), a Novel 10% Liquid Immunoglobulin Preparation for Intravenous Use, in Patients with Primary Immunodeficiencies. In: Journal of Clinical Immunology, Vol. 29, no. 1, p. 137-44 (2009)
Permanent URL http://hdl.handle.net/2078.1/35824