title: Long-term efficacy, safety, and tolerability of Hizentra® for treatment of primary immunodeficiency disease.
abstract: Hizentra(®) (20% subcutaneous immunoglobulin [SCIG]) was administered to subjects with primary immunodeficiency disease in two extension studies in the EU and US to assess long-term efficacy and tolerability. Subjects (aged 4-69 years) were treated for 148 weeks in the EU (N = 40; 5405 infusions) and 87 weeks in the US (N = 21; 1735 infusions). Weekly doses were 116.0 mg/kg (EU) and 193.2 mg/kg (US); IgG levels were 7.97 g/L (EU) and 11.98 g/L (US). Annualized rates of serious bacterial infections were 0.05 infections/subject/year (EU) and 0.06 infections/subject/year (US). Rates of any infection were 3.33 infections/subject/year (EU) and 2.38 infections/subject/year (US). The rate of bronchopulmonary infections was higher in the EU study. No treatment-related serious AEs occurred; no subject discontinued because of treatment-related AEs. Self-administered Hizentra afforded sustained effective protection from infections and favorable tolerability during an extended treatment period of up to 3 years.
authors.affiliation: University Hospital of Wales, Cardiff, UK. Electronic address: jollessr@cardiff.ac.uk.
authors.forename: Stephen
authors.initials: S
authors.lastname: Jolles
authors.affiliation: Hospital St. Georg GmbH Leipzig, Academic Teaching Hospital of the University of Leipzig, Leipzig, Germany. Electronic address: Michael.Borte@sanktgeorg.de.
authors.forename: Michael
authors.initials: M
authors.lastname: Borte
authors.affiliation: Indiana University School of Medicine and the IU-Simon Cancer Center, Indianapolis, IN, USA. Electronic address: ronelson@iu.edu.
authors.forename: Robert P
authors.initials: RP
authors.lastname: Nelson
authors.affiliation: CSL Behring, LLC, King of Prussia, PA, USA. Electronic address: Mikhail.Rojavin@cslbehring.com.
authors.forename: Mikhail
authors.initials: M
authors.lastname: Rojavin
authors.affiliation: CSL Behring AG, Berne, Switzerland. Electronic address: Martin.Bexon@cslbehring.com.
authors.forename: Martin
authors.initials: M
authors.lastname: Bexon
authors.affiliation: CSL Behring, Marburg, Germany. Electronic address: John-Philip.Lawo@cslbehring.com.
authors.forename: John-Philip
authors.initials: JP
authors.lastname: Lawo
authors.affiliation: Medical City Children's Hospital, Dallas, TX, USA. Electronic address: drrichwasserman@gmail.com.
authors.forename: Richard L
authors.initials: RL
authors.lastname: Wasserman
mesh_terms: D000293:Adolescent; D000328:Adult; D000368:Aged; D002648:Child; D002675:Child, Preschool; D005260:Female; D006801:Humans; D007074:Immunoglobulin G / Q000008:administration & dosage / Q000009:adverse effects / Q000097:blood / Q000627:therapeutic use*; D007153:Immunologic Deficiency Syndromes / Q000188:drug therapy*; D007155:Immunologic Factors / Q000008:administration & dosage / Q000009:adverse effects / Q000627:therapeutic use*; D008297:Male; D008875:Middle Aged; D016896:Treatment Outcome; D055815:Young Adult
chemical_list: C558471:Hizentra; D007074:Immunoglobulin G; D007155:Immunologic Factors
keywords: 20% SCIG; Hizentra; Primary immunodeficiency; Subcutaneous immunoglobulin