Efficacy, safety, tolerability and pharmacokinetics of a novel human immune globulin subcutaneous, 20%: a phase 2/3 study in Europe in patients with primary immunodeficiencies
OPEN Clinical and experimental immunology | 11 Sep 2016
M Borte, G Kriván, B Derfalvi, L Maródi, T Harrer, S Jolles, C Bourgeois, W Engl, H Leibl, B McCoy, D Gelmont and L Yel
A highly-concentrated (20%) immunoglobulin G (IgG) preparation for subcutaneous administration (IGSC 20%), would offer a new option for antibody replacement therapy in patients with primary immunodeficiency diseases (PIDD). The efficacy, safety, tolerability and pharmacokinetics of IGSC 20% were evaluated in a prospective trial in Europe in 49 patients with PIDD aged 2 to 67 years. Over a median of 358 days, patients received 2349 IGSC 20% infusions at monthly doses equivalent to those administered for previous intravenous or subcutaneous IgG treatment. The rate of validated acute bacterial infections (VASBIs) was significantly lower than 1 per year (0.022/patient-year, p<0.0001); the rate of all infections was 4.38/patient-year. Median trough IgG concentrations were ≥8g/l. There was no serious adverse event (AE) deemed related to IGSC 20% treatment; related non-serious AEs occurred at a rate of 0.101 event/infusion. The incidence of local related AEs was 0.069 event/infusion (0.036 event/infusion, when excluding a 13 year-old patient who reported 79/162 total related local AEs). The incidence of related systemic AEs was 0.032 event/infusion. Most related AEs were mild, none severe. For 64.6% of patients and in 94.8% of IGSC 20% infusions, no local related AE occurred. The median infusion duration was 0.95 (range: 0.3-4.1) h using mostly 1 to 2 administration sites (median = 2 sites [range: 1-5]). Almost all infusions (99.8%) were administered without interruption/stopping or rate reduction. These results demonstrate that IGSC 20% provides an effective and well-tolerated therapy for patients previously on intravenous or subcutaneous treatment, without the need for dose adjustment. This article is protected by copyright. All rights reserved.
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