Safety and efficacy of subcutaneous human immunoglobulin in children with primary immunodeficiency

Abstract

The primary objective of this prospective, open-label study was to evaluate the safety and tolerability of home treatment with a 16%, ready-to-use, human normal immunoglobulin solution for subcutaneous administration (SCIg 16%, Subcuvia, Baxter Medical AB, Kista, Sweden) in children with primary immunodeficiency (PI) previously receiving intravenous immunoglobulins (IVIg) treatment. Secondary objectives were to evaluate the efficacy of SCIg 16% through documented bacterial infections, IgG trough levels, quality of life (Child Health Questionnaire [CHQ]), healthcare resource utilization and patient preference. Twelve patients with PI were included in the study. There were no significant changes from baseline in vital signs or laboratory parameters. Most adverse events (311 of 328) were mild injection site reactions that, in most cases, resolved after 1-2 months. SCIg 16% maintained consistently high IgG trough levels, and the rate of bacterial infections was not different from that seen with previous IVIg treatment. SCIg treatment was also associated with significantly fewer lost work and school days and lower direct healthcare-related expenses and time consumed due to hospital or physician visits. Home-based SCIg treatment was well tolerated, at least as effective as IVIg for preventing infections, and preferred by children with PI previously receiving IVIg treatment.