title: Efficacy, Safety, and Pharmacokinetics of a Novel Human Immune Globulin Subcutaneous, 20 % in Patients with Primary Immunodeficiency Diseases in North America.
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 < 0.0001 compared with the historical control), and the annualized rate of infection was 2.41 events/patient. Median IgG trough levels were >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.
authors: Allergy, Asthma and Immunology Clinic PA, Irving, TX, USA.
authors: Daniel
authors: D
authors: Suez
authors: Allergy Associates of the Palm Beaches, North Palm Beach, FL, USA.
authors: Mark
authors: M
authors: Stein
authors: Division of Basic and Clinical Immunology, University of California at Irvine, Irvine, CA, USA.
authors: Sudhir
authors: S
authors: Gupta
authors: Vital Prospects Clinical Research Institute, Tulsa, OK, USA.
authors: Iftikhar
authors: I
authors: Hussain
authors: IMMUNOe International Research Centers, Centennial, CO, USA.
authors: Isaac
authors: I
authors: Melamed
authors: LSU Health Sciences Center, Children's Hospital, New Orleans, LA, USA.
authors: Kenneth
authors: K
authors: Paris
authors: Oklahoma Institute of Allergy and Asthma Clinical Research, LLC, Oklahoma City, OK, USA.
authors: Amy
authors: A
authors: Darter
authors: Baxalta Innovations GmbH, now part of Shire, Vienna, Austria.
authors: Christelle
authors: C
authors: Bourgeois
authors: Baxalta Innovations GmbH, now part of Shire, Vienna, Austria.
authors: Sandor
authors: S
authors: Fritsch
authors: Baxalta Innovations GmbH, now part of Shire, Vienna, Austria.
authors: Heinz
authors: H
authors: Leibl
authors: Baxalta US Inc., now part of Shire, 650 East Kendall Street, Cambridge, MA, USA.
authors: Barbara
authors: B
authors: McCoy
authors: Baxalta US Inc., now part of Shire, Westlake Village, CA, USA.
authors: David
authors: D
authors: Gelmont
authors: Division of Basic and Clinical Immunology, University of California at Irvine, Irvine, CA, USA. Leman.Yel@shire.com.|Baxalta US Inc., now part of Shire, 650 East Kendall Street, Cambridge, MA, USA. Leman.Yel@shire.com.
authors: Leman
authors: L
authors: Yel
mesh_terms: D000293:Adolescent; D000328:Adult; D000368:Aged; D000369:Aged, 80 and over; D001424:Bacterial Infections / Q000209:etiology / Q000517:prevention & control; D002648:Child; D002675:Child, Preschool; D005260:Female; D006801:Humans; D016756:Immunoglobulins, Intravenous / Q000008:administration & dosage* / Q000009:adverse effects / Q000493:pharmacokinetics*; D007153:Immunologic Deficiency Syndromes / Q000150:complications / Q000175:diagnosis / Q000188:drug therapy*; D055104:Infusions, Subcutaneous; D008297:Male; D008875:Middle Aged; D011788:Quality of Life; D016896:Treatment Outcome; D055815:Young Adult
chemical_list: D016756:Immunoglobulins, Intravenous
keywords: 20 % immunoglobulin; Immunoglobulin replacement therapy; Pharmacokinetics; Primary immunodeficiency diseases; Subcutaneous administration