Efficacy and Safety of IgPro20, a Subcutaneous Immunoglobulin, in Japanese Patients with Primary Immunodeficiency Diseases

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• 作者：Hirokazu Kanegane (1)
  Kohsuke Imai (2)
  Masafumi Yamada (3)
  Hidetoshi Takada (4)
  Tadashi Ariga (3)
  Martin Bexon (5)
  Mikhail Rojavin (6)
  Wilson Hu (7)
  Midori Kobayashi (8)
  John-Philip Lawo (9)
  Shigeaki Nonoyama (10)
  Toshiro Hara (4)
  Toshio Miyawaki (11)
  
• 关键词：Primary immunodeficiency ; PID ; primary antibody deficiency ; SCIG ; Hizentra? ; IgPro20 ; Japan
• 刊名：Journal of Clinical Immunology
• 出版年：2014
• 出版时间：February 2014
• 年：2014
• 卷：34
• 期：2
• 页码：204-211
• 全文大小：211 KB
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  6. Hagan JB, Fasano MB, Spector S, Wasserman RL, Melamed I, Rojavin MA, et al. Efficacy and safety of a new 20?% immunoglobulin preparation for subcutaneous administration, IgPro20, in patients with primary immunodeficiency. J Clin Immunol. 2010;30:734-5. CrossRef
  7. Jolles S, Bernatowska E, de Gracia J, Borte M, Cristea V, Peter HH, et al. Efficacy and safety of Hizentra? in patients with primary immunodeficiency after a dose-equivalent switch from intravenous or subcutaneous replacement therapy. Clin Immunol. 2011;141:90-02. CrossRef
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  17. Takada H, Kanegane H, Nomura A, Yamamoto K, Ihara K, Takahashi Y, et al. Female agammaglobulinemia due to the Bruton tyrosine kinase deficiency caused by extremely skewed X-chromosome inactivation. Blood. 2004;103:185-. CrossRef
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• 作者单位：Hirokazu Kanegane (1)
  Kohsuke Imai (2)
  Masafumi Yamada (3)
  Hidetoshi Takada (4)
  Tadashi Ariga (3)
  Martin Bexon (5)
  Mikhail Rojavin (6)
  Wilson Hu (7)
  Midori Kobayashi (8)
  John-Philip Lawo (9)
  Shigeaki Nonoyama (10)
  Toshiro Hara (4)
  Toshio Miyawaki (11)
  
  1. Department of Pediatrics, Graduate School of Medicine and Pharmaceutical Sciences, University of Toyama, Toyama, Japan
  2. Department of Community Pediatrics, Perinatal and Maternal Medicine, Tokyo Medical and Dental University, Tokyo, Japan
  3. Department of Pediatrics, Hokkaido University Graduate School of Medicine, Sapporo, Japan
  4. Department of Pediatrics, Graduate School of Medical Sciences, Kyushu University, Fukuoka, Japan
  5. CSL Behring AG, Berne, Switzerland
  6. CSL Behring LLC, King of Prussia, PA, USA
  7. CSL Limited, Research and Development, Melbourne, Victoria, Australia
  8. CSL Behring KK, Tokyo, Japan
  9. CSL Behring GmbH, Marburg, Germany
  10. Department of Pediatrics, National Defense Medical College, Saitama, Japan
  11. Toyama City Hospital, Toyama, Japan
  
• ISSN：1573-2592

文摘

Purpose Intravenous (IVIG) and subcutaneous (SCIG) immunoglobulin infusions are widely used for the treatment of patients with primary immunodeficiency (PID) worldwide. This prospective, multicenter, open-label, single-arm Phase III study evaluated the efficacy, tolerability, and safety of IgPro20 (Hizentra?; L-proline–stabilized 20?% human SCIG) in adult and pediatric Japanese patients with PID. Methods Patients received three IVIG infusions at 3--week intervals followed by a dose-equivalent switch to weekly SCIG infusions. A 12-week wash-in/wash-out period was followed by a 12-week SCIG efficacy period. The primary efficacy endpoint was the comparison of total serum IgG trough levels during the IVIG and SCIG efficacy periods by calculating the geometric mean ratio (GMR). Results The GMR of IgG trough levels on SCIG versus IVIG was 1.09 (2-sided 90?% confidence interval: 1.06-.13). No serious bacterial infections were reported. Eleven patients (52.4?%) had infectious episodes with an overall rate of 2.98 infections/patient/year; 7 patients (33.3?%) missed school/work/daycare due to infection (3.48?days/patient/year). Sixteen patients (76.2?%) were treated with antibiotics for an adverse event (AE; 47.6?%) or prophylaxis (23.8?%), resulting in 167.42?days/patient/year of antibiotic use. During SCIG treatment, 24 patients (96.0?%) had 269 AEs (0.461 AEs per/infusion) including local reactions as the most common AE (20 patients, 80.0?%). Local tolerability of IgPro20 was assessed as “very good-or “good-after 85.4?% of SCIG infusions. One patient (4.0?%) experienced a serious AE of moderate severity (bacterial infection) that was considered unrelated to study medication. Conclusion IgPro20 was effective and well tolerated in Japanese patients with PID.