Elisabeth Clodi, Austria
Octapharma Pharmazeutika Produktionsges.m.b.H. Global Medical & Scientific AffairsPresenter of 1 Presentation
CLINICAL EFFICACY, SAFETY AND TOLERABILITY OF A 16.5% SUBCUTANEOUS IMMUNOGLOBULIN (SCIG) PREPARATION IN PEDIATRIC PID PATIENTS
Abstract
Background and Aims
In this study (NCT 01888484) the efficacy, safety and tolerability of a 16.5% IgG preparation for subcutaneous administration (cutaquig®, Octapharma/Switzerland) were evaluated in adult and pediatric PID patients. This sub-analysis focused specifically on pediatric patients. Primary outcome was to assess efficacy in preventing serious bacterial infections. Secondary endpoints included tolerability, safety and rate of other infections.
Methods
Prospective, open-label, single-arm phase 3 study conducted in North America and Europe. PID patients stable on IVIG treatment for at least six infusions (with IgG trough levels ≥5.0 g/L) underwent a 12-week wash-in/wash-out period, followed by a 52-week efficacy period. In both periods patients received weekly SCIG doses.
Results
Twenty-four children (age: 2-16 years; 25% female) received a total of 1,350 SCIG infusions. No serious bacterial infections were recorded. Among the 77 other infections during the efficacy period only one infection (RSV bronchiolitis) was graded as severe. All other infections were either mild (86%) or moderate (13%) in intensity. Infection rate per person-year was 3.6. Of the 115 reported adverse events, 5 were assessed as being related to the study drug (all non-serious). Rate of related adverse events per infusion: 0.0037. The majority of infusions (82%) did not trigger an infusion site reaction. Serum IgG trough levels were relatively constant and higher at the end of the study than after IVIG treatment.
Conclusions
Results demonstrate that treatment with the subcutaneous 16.5% human normal immunoglobulin preparation (cutaquig®) in pediatric patients with PID is effective, safe, and well tolerated.