1. Efficacy, Safety, and Pharmacokinetics of a Novel Human Immune Globulin Subcutaneous, 20 % in Patients with Primary Immunodeficiency Diseases in North America
- Author
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Suez, Daniel, Stein, Mark, Gupta, Sudhir, Hussain, Iftikhar, Melamed, Isaac, Paris, Kenneth, Darter, Amy, Bourgeois, Christelle, Fritsch, Sandor, Leibl, Heinz, McCoy, Barbara, Gelmont, David, and Yel, Leman
- Subjects
Vaccine Related ,Clinical Research ,6.1 Pharmaceuticals ,Evaluation of treatments and therapeutic interventions ,Infection ,Adolescent ,Adult ,Aged ,Aged ,80 and over ,Bacterial Infections ,Child ,Child ,Preschool ,Female ,Humans ,Immunoglobulins ,Intravenous ,Immunologic Deficiency Syndromes ,Infusions ,Subcutaneous ,Male ,Middle Aged ,Quality of Life ,Treatment Outcome ,Young Adult ,Primary immunodeficiency diseases ,Immunoglobulin replacement therapy ,Subcutaneous administration ,20% immunoglobulin ,Pharmacokinetics ,20 % immunoglobulin ,Immunology - Abstract
Patients with primary immunodeficiency disease (PIDD) typically require life-long intravenous (IV) or subcutaneous (SC) immunoglobulin (Ig) replacement therapy to prevent recurrent infections. The efficacy, safety, and pharmacokinetics of a highly concentrated (20 %) Ig preparation for SC administration (IGSC 20 %) were evaluated in a prospective trial in patients with PIDD. A total of 74 patients (aged 3-83 years) received 4327 IGSC 20 % infusions over a median of 380.5 days. The rate of validated serious bacterial infections was 0.012 event/patient-year (p 14.5 g/l. The median maximum infusion rate was 60 ml/h/site (range 4.4-180), resulting in a median infusion duration of 0.95 h. A volume ≥30 ml was infused per site in 74.8 % of IGSC 20 % infusions. Most (84.9 %) infusions were administered using ≤2 infusion sites; for 99.8 % of infusions, there was no need to interrupt/stop administration or reduce the infusion rate. No related serious adverse event (AE) occurred during IGSC 20 % treatment; related non-serious AEs occurred at a rate of 0.036 event/infusion. The incidence of related local AEs was 0.015 event/infusion and of related systemic AEs was 0.021 event/infusion; most were mild in severity, none severe. Increased infusion rates or volumes were not associated with higher AE rates. The investigated IGSC 20 % treatment was shown to be effective and safe, enabling higher infusion rates and volumes per site compared to conventional SC treatments, resulting in fewer infusion sites and shorter infusion durations.
- Published
- 2016