Abstract Details

Title Safety and Tolerability of Intravenous Immunoglobulin Infusion in Down Syndrome Regression Disorder

Topic Child Neurology and Developmental Neurology

Presentation(s) P6 - Poster Session 6 (8:00 AM-9:00 AM)

Poster/Presentation
Number 8-007

Objective This study sought to evaluate the safety and tolerability of IVIg in individuals with DSRD compared to a real-world cohort of individuals with pediatric onset neuroimmunologic disorders.

Background Three large multi-center studies have identified the clinical utility of intravenous immunoglobulin (IVIg) in the treatment of Down syndrome regression disorder (DSRD). Yet the tolerability of infusions in individuals with DS and the safety of IVIg remains unknown in this population.

Design/Methods A single-center, retrospective chart review evaluating clinically documented infusion reactions was performed for individuals meeting international consensus criteria for DSRD and having IVIg infusions between 2019-2023. Infusion reactions were evaluated for severity and need for alterations in infusion plan. This cohort was compared against an age and sex matched cohort of children with neuroimmuologic conditions who had also received IVIg infusions.

Results In total, 127 individuals with DSRD and 186 individuals with other neuroimmunologic disorders were enrolled. There was no difference in the overall rate of adverse reactions (AEs)between the DSRD and general neuroimmunology cohorts (p=0.31, 95%CI: 0.80-2.00), but cardiac-related AEs specifically were more common among the DSRD group (p= 0.02, 95%CI: 1.23-17.54). When AEs did occur, there was no difference in frequency of pharmacologic intervention (p= 0.12, 95%CI:0.34-1.13) or discontinuation of therapy (p=0.74, 95%CI: 0.06-7.44). There was a higher incidence of lab abnormalities on IVIG among the general neuroimmunology cohort (p=0.03, 95%CI: 0.24-0.94) compared to the DSRD cohort. Transaminitis was the most common laboratory abnormality in the DSRD group.

Conclusions In a large cohort of individuals with DSRD, there were no significant differences in the safety and tolerability of IVIg compared to a cohort of children and young adults with neuroimmunologic conditions.

Authors/Disclosures
Jonathan Santoro, MD (Department of Neurology, Children's Hospital Los Angeles) PRESENTER	Dr. Santoro has received personal compensation in the range of $5,000-$9,999 for serving as a Consultant for UCB. Dr. Santoro has received personal compensation in the range of $10,000-$49,999 for serving as a Consultant for Cycle Pharma. Dr. Santoro has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Dianthus. Dr. Santoro has received personal compensation in the range of $500-$4,999 for serving as a Consultant for National Down Syndrome Society.
Saba Jafarpour, MD (Children’s Hospital of Los Angeles)	Dr. Jafarpour has nothing to disclose.
Mellad Khoshnood, MD (Children's Hospital Los Angeles)	Dr. Khoshnood has nothing to disclose.
Natalie Boyd (Children's Hospital Los Angeles)	Natalie Boyd has nothing to disclose.
Benjamin Vogel	Benjamin Vogel has nothing to disclose.
Lina Nguyen (Children's Hospital Los Angeles)	Lina Nguyen has nothing to disclose.
Michael S. Rafii, MD, PhD (USC Alzheimer'S Therapeutic Research Institute)	Dr. Rafii has received personal compensation in the range of $500-$4,999 for serving as a Consultant for AC Immune. Dr. Rafii has received personal compensation in the range of $5,000-$9,999 for serving as a Consultant for Keystone Bio. Dr. Rafii has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Alzheon.

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