Abstract
BACKGROUND: Immunoglobulin products are widely used for the treatment of immunodeficiency and autoimmune disorders. Although clinical trials have demonstrated their efficacy and tolerability, data describing their postmarketing safety profile in pediatric populations remain limited, particularly regarding rare and serious adverse events. This study aimed to characterize adverse event reports associated with immunoglobulin use in children during the postmarketing period. METHODS: Pediatric adverse event reports (<18 years) submitted to the U.S. Food and Drug Administration Adverse Event Reporting System between January 1, 2001 and December 31, 2023 were analyzed. Descriptive statistics were used to summarize reported adverse events, and selected events and deaths were reviewed individually. RESULTS: In total, 2997 pediatric adverse event reports were identified, of which 1855 (61.9%) were classified as serious. The most frequently reported adverse events were headache (n = 394), pyrexia (n = 294), urticaria (n = 231), vomiting (n = 229), nausea (n = 182), and infusion-related reaction (n = 151). Clinically significant adverse events were infrequent, including hemolytic disorders (n = 82), aseptic meningitis (n = 80), anaphylaxis (n = 34), thromboembolic events (n = 34), renal impairment (n = 22), and transfusion-related acute lung injury (n = 7). Adverse event patterns were similar between intravenous and subcutaneous formulations, except for higher proportions of systemic infusion reactions with intravenous administration and injection-site reactions with subcutaneous administration. Among 110 reported deaths, the leading causes were unspecified (n = 37), nervous system disorders (n = 20), infections (n = 16), and general disorders (n = 11). CONCLUSION: The postmarketing safety profile of immunoglobulin in pediatric patients was consistent with clinical data observed in prelicensure studies and described in the package insert, with no new safety concerns identified. This assessment is subject to important limitations, including reliance on passive surveillance data and the lack of exposure denominators, which should be considered when interpreting the results.