An assessment of efficacy and safety of replacement therapy with subcutaneous immunoglobulin 16.5% administered by rapid push method in patients with inborn errors of immunity: the results of a prospective multicenter study

Subcutaneous immunoglobulins (SCIgs) have been widely used in patients with inborn errors of immunity (IEIs) and have been shown to be effective and well tolerated. Here, we assessed efficacy and safety of replacement therapy with SCIgs in a large heterogeneous cohort of patients with IEIs. The study was approved by the Independent Ethics Committee and the Scientific Council of the Dmitry Rogachev National Medical Research Center of Pediatric Hematology, Oncology and Immunology of Ministry of Healthcare of Russia. All the patients and/or their legal representatives signed informed consent for participation in the study. Patients with IEIs, previously treated with intravenous immunoglobulins (IVIgs) were switched to SCIgs 16.5%. The drug was administered every 7–10 days using rapid push method for 12 months. In the end, we analyzed the following efficacy and safety criteria: the rate of infections and associated events, serum immunoglobulin G concentrations, adverse reactions and patient compliance. We had recruited 233 patients (the median age was 11 years, the percentage of male patients was 66.0%), and 200 out of these completed the study. A total of 8787 SCIg infusions were administered during the study. The infection rate (per 1 patient per month) on SCIg treatment was 0.05, which was significantly lower compared to the IVIg therapy (p 0.001). The median trough IgG level was 6.9 g/l on IVIgs, 9.0 g/l during the first 6 months of SCIgs and 9.2 g/l over 7–12 months of SCIg treatment. Systemic reactions were registered in 12.45% of IVIG infusions and in 1.93% of SCIg infusions (р 0,001). Our study demonstrates the safety and efficacy of replacement therapy with SCIgs administered via rapid push method.