Related Experiment Video
Updated: Jun 4, 2025

In Vitro Modeling of Down Syndrome Neurogenesis Using Human-Induced Pluripotent Stem Cells
Published on: March 7, 2025
Safety and Tolerability of Home Infusions in Down Syndrome Regression Disorder
Jonathan D Santoro1, Lina Nguyen2, Nicole A Nishimori2
1Division of Neuroimmunology, Department of Pediatrics, Children's Hospital Los Angeles, Los Angeles, California; Department of Neurology, Keck School of Medicine of the University of Southern California, Los Angeles, California.
Purpose:
Down syndrome regression disorder (DSRD) is a rare neuropsychiatric condition affecting otherwise healthy individuals with Down syndrome. Multiple studies on DSRD have revealed that immunotherapy with intravenous immunoglobulin (IVIg) is both safe and effective, although site of infusion has never been studied. This study sought to evaluate the safety and tolerability of IVIg in individuals with DSRD receiving home-based infusions.
Methods:
A single-center, retrospective chart review evaluating infusion reactions was performed for individuals meeting criteria for DSRD and having received IVIg infusions between 2019 and 2024. Adverse events (AEs) were evaluated for severity and need for alterations in infusion plan. A cohort of individuals receiving home-based infusions was compared with a cohort of individuals receiving infusions at an academic medical center.
Findings:
A total of 315 individuals (162 institutional infusions [51%] and 153 home infusions [49%]) met the inclusion criteria. There were no statistical differences between the demographic and clinical features of the cohorts. Individuals receiving home infusions had the same rate of AE during an infusion (P = 0.14), although they did have a lower number of total AEs (P < 0.001). Individuals receiving home infusions experienced a lower number of behavioral issues with infusions (P = 0.03) and had significantly lower discontinuations of infusions secondary to behavioral issues (P = 0.04).
Implications:
Rates of AEs and serious AEs in those with DSRD were the same regardless of site of infusion. These data should be considered in policy regarding the appropriateness of home-based infusions as a safe alternative, when suitable for patients and caregivers, for individuals with DSRD.
Related Concept Videos
Drug Dosage Regimen: Overview
Typically, the starting dose and dosing interval are guided by the manufacturer's recommendations based on clinical trials conducted during and after drug...
Factors Affecting Drug Response: Overview
Dosage Regimen: Fixed Dose
Fixed-dose regimens can be used for various routes of administration, including intravenous (IV) injections and oral medications. For IV administration, a predetermined amount of the drug is...
One-Compartment Model: IV Infusion
The one-compartment model for IV infusion uses mathematical equations to describe the rate of change in drug quantity in the body. At steady-state or infusion equilibrium, the drug input...
Two-Compartment Open Model: IV Infusion
The model illustrates the decrease in plasma drug concentration from the central compartment with a specific equation. It shows that under steady-state conditions, the drug's input rate...
Rational Dosage Regimen: Maintenance Dose and Loading Dose
In most cases, drugs are administered repetitively or infused continuously to maintain a steady-state concentration in the body. At a steady...

