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.

ERS statement on the multidisciplinary respiratory management of ataxia telangiectasia

“…These findings suggest that the recognition of groups who may be at higher risk of lower pulmonary function may help to direct care and improve clinical outcomes in A-T. If Ig therapy is considered, most centres administer subcutaneous Ig, as is currently recommended for severe immundeficiencies [29][30][31].…”

Pharmacokinetics, Safety, and Tolerability of Subcutaneous Immune Globulin Injection (Human), 10 % Caprylate/Chromatography Purified (GAMUNEX®-C) in Pediatric Patients with Primary Immunodeficiency Disease

“…Historically, lifelong treatment of these types of PID with immunoglobulin (Ig) through various routes (e.g., intravenous (IV), subcutaneous (SC), intramuscular) has been wellestablished and has been shown to decrease the incidence of infections in these patients [1][2][3][4][5][6][7][8][9][10]. Moreover, SCIg therapy has been shown through several studies to be effective, safe, and well-tolerated in patients with PID [11][12][13][14][15][16][17][18][19][20][21][22][23][24][25]. SCIg therapy may be beneficial for patients who are poor candidates for IVIg, have poor venous access, or have recurrent systemic infusion-related reactions (i.e., headache, fever, chills, or myalgia).…”

“…Particularly with children, difficult venous access can be a barrier to IVIg therapy. The results from several studies have shown that SCIg in children is feasible and safe [14,16,19,[21][22][23][24][25]. It is now generally accepted that repeated SCIg infusions result in lower systemic adverse effects such as headaches and joint pain occurring within 72 h of the SC infusion [9].…”

Rapid push: new opportunities in subcutaneous immunoglobulin replacement therapy

“…In Poland, SCIG therapy was introduced in 2001 at the Children's Memorial Health Institute in Warsaw. Comparison of two methods in children revealed a slightly lower number of infections and days with antibiotics due to infections on subcutaneous infusions, indicating SCIG as a better choice of treatment in some group of patients [4,[13][14][15][16][17].…”