What question did this study set out to answer?

To compare the effectiveness of lorazepam and IVIg against no treatment in patients with Down syndrome regression disorder.

March 10, 2026Open Access

A Comparative Effectiveness Study of Lorazepam or IVIg Versus no Treatment for Down Syndrome Regression Disorder

Key Points

To compare the effectiveness of lorazepam and IVIg against no treatment in patients with Down syndrome regression disorder.
Prospective, non-randomized observational study
Assessed therapeutic responses in 212 patients with DSRD
Measured outcomes at baseline, 12, and 24 weeks
Used linear mixed-effect models to analyze data
Outcomes included BFCRS, 25FW, CGI-S, NPIT, NPIST
Both lorazepam and IVIg improved outcomes compared to no treatment at 12 weeks (p < 0.001)
IVIg outperformed lorazepam in multiple outcomes at both 12 and 24 weeks
Significant differences were noted in BFCRS and NPIT scores favoring IVIg
Treatment response varied based on neurodiagnostic and lumbar puncture abnormalities
Suggested at least a 24-week treatment course for maximizing IVIg benefits

Abstract

Down syndrome regression disorder (DSRD) is manifested by acute or subacute onset of bradykinesia, catatonia, mutism, and neuropsychiatric symptoms. Both benzodiazepines and immunotherapy have been effective in treating DSRD, although no study has compared these treatments directly. This observational study compares the response of lorazepam and intravenous immunoglobulin (IVIg) to no therapy over 6 months. This prospective, non-randomized, observational natural history study assessed therapeutic responses in 212 patients meeting international criteria for DSRD. Outcomes were measured at baseline, 12, and 24 weeks using the Bush–Francis Catatonia Rating Scale (BFCRS), 25-foot walk (25FW), Clinical Global Impression-Severity (CGI-S), and Neuropsychiatric Inventory Total (NPIT) and Severity (NPIST) scores. Linear mixed-effect models were used to examine changes in outcomes. Patients received lorazepam (n = 85, 40%), IVIg (n = 68, 32%) or no therapy (n = 59, 28%). By 12 weeks, both active therapies outperformed no therapy on all outcomes (all p < 0.001), with IVIg showing additional advantages over lorazepam on BFCRS mean difference (95% CI): − 2.24 (− 3.72, − 0.75), NPIT − 9.22 (− 11.22, − 7.23), and NPIST − 3.16 (− 4.07, − 2.26). By 24 weeks, IVIg was superior to lorazepam on all outcomes 25FW: − 1.80 (− 2.58, − 1.02); BFCRS: − 7.09 (− 8.58, − 5.60); CGI-S: − 0.63 (− 1.01, − 0.25); NPIT: − 12.23 (− 14.22, − 10.23); NPIST: − 3.80 (− 4.71, − 2.89). Treatment response varied by lumbar puncture abnormality (BFCRS), any neurodiagnostic study abnormality (CGI-S, NPIT), and serum cytokines and MRI abnormality (NPIT), with IVIg demonstrating greater benefits among patients with such abnormalities. At the endpoint of 24 weeks, both IVIg and lorazepam were superior to no treatment for DSRD but IVIg was superior to lorazepam for all five outcomes. Between 12 and 24 weeks of treatment, IVIg showed greater improvement in all outcomes than did lorazepam, suggesting that at least a 24-week treatment course is necessary for maximum IVIg benefit.

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