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.

摘要: 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 (range = 1-5)]. Almost (99·8%) without interruption/stopping reduction. These results demonstrate that provides an effective well-tolerated previously on treatment, need dose adjustment.