title: Efficacy and safety of hizentra®, a new 20% immunoglobulin preparation for subcutaneous administration, in pediatric patients with primary immunodeficiency.
abstract: Subcutaneous IgG treatment for primary immunodeficiencies (PI) is particularly well suited for children because it does not require venous access and is mostly free of systemic adverse events (AEs). In a prospective, open-label, multicenter, single-arm, Phase III study, 18 children and five adolescents with PI were switched from previous intravenous (IVIG) or subcutaneous (SCIG) IgG treatment to receive dose-equivalent, weekly subcutaneous infusions of Hizentra(®) for 40 weeks. Mean IgG trough levels were maintained in patients previously on SCIG, or increased in those previously on IVIG, regardless of age. No serious bacterial infections were reported during the efficacy period of the study. The rates of non-serious infections were 4.77 (children) and 5.18 (adolescents) infections per patient per year. Related AEs were observed in seven children (38.9%) and two adolescents (40%). Three serious AEs and two AEs leading to discontinuation (all unrelated) were reported in children. Hizentra(®) is an effective and well-tolerated treatment for pediatric patients.
authors.affiliation: Hospital St. Georg GmbH Leipzig, Academic Teaching Hospital of the University of Leipzig, Delitzscher Strasse 141, 04129, Leipzig, Germany. Michael.Borte@sanktgeorg.de
authors.forename: Michael
authors.initials: M
authors.lastname: Borte
authors.forename: Malgorzata
authors.initials: M
authors.lastname: Pac
authors.forename: Margit
authors.initials: M
authors.lastname: Serban
authors.forename: Teresa
authors.initials: T
authors.lastname: Gonzalez-Quevedo
authors.forename: Bodo
authors.initials: B
authors.lastname: Grimbacher
authors.forename: Stephen
authors.initials: S
authors.lastname: Jolles
authors.forename: Othmar
authors.initials: O
authors.lastname: Zenker
authors.forename: Jutta
authors.initials: J
authors.lastname: Neufang-Hueber
authors.forename: Bernd
authors.initials: B
authors.lastname: Belohradsky
mesh_terms: D000293:Adolescent; D002648:Child; D002675:Child, Preschool; D057915:Drug Substitution; D005260:Female; D006801:Humans; D007074:Immunoglobulin G / Q000008:administration & dosage* / Q000009:adverse effects; D016756:Immunoglobulins, Intravenous / Q000008:administration & dosage* / Q000009:adverse effects; D007153:Immunologic Deficiency Syndromes / Q000276:immunology / Q000473:pathology / Q000503:physiopathology / Q000628:therapy*; D007279:Injections, Subcutaneous; D008297:Male; D011446:Prospective Studies; D016896:Treatment Outcome; D028761:Withholding Treatment
chemical_list: C558471:Hizentra; D007074:Immunoglobulin G; D016756:Immunoglobulins, Intravenous