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

Abstract

Background and ObjectivesIntravenous 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).Materials and MethodsIn 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.ResultsBoth 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.ConclusionMP‐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.