title: Efficacy and safety of IgPro20, a subcutaneous immunoglobulin, in Japanese patients with primary immunodeficiency diseases.
abstract: OBJECTIVE
  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 3-4-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: 1.06-1.13). 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.
  
  
  CONCLUSIONS
  IgPro20 was effective and well tolerated in Japanese patients with PID.
authors: Department of Pediatrics, Graduate School of Medicine and Pharmaceutical Sciences, University of Toyama, Toyama, Japan.
authors: Hirokazu
authors: H
authors: Kanegane
authors: Kohsuke
authors: K
authors: Imai
authors: Masafumi
authors: M
authors: Yamada
authors: Hidetoshi
authors: H
authors: Takada
authors: Tadashi
authors: T
authors: Ariga
authors: Martin
authors: M
authors: Bexon
authors: Mikhail
authors: M
authors: Rojavin
authors: Wilson
authors: W
authors: Hu
authors: Midori
authors: M
authors: Kobayashi
authors: John-Philip
authors: JP
authors: Lawo
authors: Shigeaki
authors: S
authors: Nonoyama
authors: Toshiro
authors: T
authors: Hara
authors: Toshio
authors: T
authors: Miyawaki
mesh_terms: D000293:Adolescent; D000328:Adult; D044466:Asian People; D002648:Child; D002675:Child, Preschool; D004334:Drug Administration Schedule; D005260:Female; D006801:Humans; D007074:Immunoglobulin G / Q000008:administration & dosage / Q000009:adverse effects / Q000627:therapeutic use*; D016756:Immunoglobulins, Intravenous / Q000009:adverse effects / Q000627:therapeutic use*; D007153:Immunologic Deficiency Syndromes / Q000188:drug therapy*; D007279:Injections, Subcutaneous; D007564:Japan; D008297:Male; D016896:Treatment Outcome; D055815:Young Adult
chemical_list: C558471:Hizentra; D007074:Immunoglobulin G; D016756:Immunoglobulins, Intravenous