Safety and efficacy of a novel mini-pool intravenous immunoglobulin therapy in children with primary immunodeficiency.

Abstract

Intravenous polyvalent immunoglobulins (IVIG) for prophylaxis in patients with primary immunodeficiency disorders (PIDs) exposes them to life-threatening infections and debilitating diseases. To improve access to IVIG in lower middle-income countries, the WHO recommends a stepwise approach for the local production of purified and virus-inactivated plasma immunoglobulins by national blood transfusion services using new technologies and medical devices. One new technology relies on single-use sterile medical devices for the purification of plasma immunoglobulin G (IgG), as well as lipid-enveloped virus inactivation from mini-pools of recovered plasma separated from whole blood (mini-pool IVIG [MP-IVIG]). This study aimed to compare the safety and efficacy of MP-IVIG to standard IVIG (STD-IVIG). In this prospective crossover clinical study, we investigated the safety and efficacy of MP-IVIG for STD-IVIG preparations as a replacement therapy in a cohort of 21 paediatric patients with PID. Both MP-IVIG and STD-IVIG were effective in reducing the frequency of severe bacterial infections and hospital admissions in patients with PID. Mild side effects have been observed in seven patients (6.2%) with PID who received MP-IVIG and five patients (5.3%) who received STD-IVIG. No moderate or severe side effects or haemolytic transfusion reactions were reported. The mortality rates were also comparable and were not related to the study products. MP-IVIG presented no safety issues and was as effective as STD-IVIG in IgG replacement in patients with PID. Due to the small numbers, the results have to be addressed with caution.