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

• DOI: 10.1111/cei.12866
• Publication Date: 2016-09-10T08:46:50Z
• Authors: M Borte; G Kriván; B Derfalvi; L Maródi; T Harrer; S Jolles; C Bourgeois; W Engl; H Leibl; B McCoy; D Gelmont; L Yel
• Keywords: Tolerability

Summary A highly concentrated (20%) immunoglobulin (Ig)G preparation for subcutaneous administration (IGSC 20%), would offer a new option antibody replacement therapy in patients with primary immunodeficiency diseases (PIDD). The efficacy, safety, tolerability and pharmacokinetics of IGSC 20% were evaluated prospective trial Europe 49 PIDD aged 2–67 years. Over median 358 days, received 2349 infusions at monthly doses equivalent to those administered previous intravenous or IgG treatment. rate validated acute bacterial infections (VASBIs) was significantly lower than 1 per year (0·022/patient-year, P < 0·0001); the all 4·38/patient-year. Median trough concentrations ≥ 8 g/l. There no serious adverse event (AE) deemed related treatment; non-serious AEs occurred 0·101 event/infusion. incidence local 0·069 event/infusion (0·036 event/infusion, when excluding 13-year-old patient who reported 79 162 total AEs). systemic 0·032 Most mild, none severe. For 64·6% 94·8% infusions, AE occurred. infusion duration 0·95 (range = 0·3-4·1) h using mainly one two sites [median 2 1–5)]. Almost (99·8%) without interruption/stopping reduction. These results demonstrate that provides an effective well-tolerated previously on treatment, need dose adjustment.