Evidence map›Paper›PMID 41795763›Full record

ArticleNeurology and therapy2026

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

Jonathan D Santoro, Saba Jafarpour, Panteha Hayati Rezvan, Mellad M Khoshnood, Benjamin N Vogel, Lina Nguyen, Lilia Kazerooni, Noemi A Spinazzi, Nidhiben Anadani, Kristen S Fisher and 5 more

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In one paragraph

Article in Neurology and therapy, 2026. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 1 paper.

0numbers the graph read from it
0cells of the map it votes in
1citing papers in PubMed
–field-weighted citation impact
1 · What the graph read from it

What it found

Each row is one number read from the abstract, on the scale the paper reported it, with its interval. Left of the dashed line favours the treatment, right favours the comparator. Under each row is the sentence it came from. New to these charts? A ten-minute tutorial.

The abstract states no effect estimate the extractor could read, or names no intervention and outcome on the map, so this paper lights no cell and moves no belief. It is still indexed, cited and linked below.

2 · The registry

The trial behind it

Trials whose registry record cites this paper, or whose number appears in the abstract. A trial that started after this paper was published is citing it as background, not reporting it.

Neither the registry nor the abstract names a trial number. If this is a trial report, that itself is worth knowing.

3 · Its place in the literature

Who cites it

1 citing paper in PubMed.

  1. Review
4 · The record

Corrections and comments

PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.

5 · Who and what money

Authors and funding

15 authors.

Jonathan D SantoroDivision of Neuroimmunology, Department of Pediatrics, Children's Hospital Los Angeles, 4650 Sunset Blvd, Mailstop 82, Los Angeles, CA, 90027, USA. santoroj@usc.edu.ORCID http://orcid.org/0000-0002-8350-8234
Saba JafarpourDivision of Neuroimmunology, Department of Pediatrics, Children's Hospital Los Angeles, 4650 Sunset Blvd, Mailstop 82, Los Angeles, CA, 90027, USA.
Panteha Hayati RezvanThe Saban Research Institute, Biostatistics, Informatics, and Data Management Core, Children's Hospital Los Angeles, Los Angeles, CA, USA.
Mellad M KhoshnoodCottage Hospital, Santa Barbara, CA, USA.
Benjamin N VogelDivision of Neuroimmunology, Department of Pediatrics, Children's Hospital Los Angeles, 4650 Sunset Blvd, Mailstop 82, Los Angeles, CA, 90027, USA.
Lina NguyenDivision of Neuroimmunology, Department of Pediatrics, Children's Hospital Los Angeles, 4650 Sunset Blvd, Mailstop 82, Los Angeles, CA, 90027, USA.
Lilia KazerooniDivision of Neuroimmunology, Department of Pediatrics, Children's Hospital Los Angeles, 4650 Sunset Blvd, Mailstop 82, Los Angeles, CA, 90027, USA.
Noemi A SpinazziDepartment of Pediatrics, University of California San Francisco, Oakland, CA, USA.
Nidhiben AnadaniDepartment of Neurology, University of Oklahoma, Oklahoma, OK, USA.
Kristen S FisherDepartment of Neurology, Texas Children's Hospital, Houston, TX, USA.
Robyn A FilipinkDivision of Child Neurology, Department of Pediatrics, UPMC's Children's Hospital of Pittsburgh, Pittsburgh, PA, USA.
Melanie A ManningDepartment of Genetics, Stanford University, Palo Alto, CA, USA.
Cathy FranklinMater Research Institute, University of Queensland, Brisbane, Australia.
Eileen A QuinnDepartment of Pediatrics, University of Toledo College of Medicine, Toledo, OH, USA.
Michael S RafiiDepartment of Neurology, Keck School of Medicine of the University of Southern California, Los Angeles, CA, USA.

Funding

No grant is acknowledged in the PubMed record.

6 · The paper itself

Abstract

introductionDown 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.

methodsThis 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.

resultsPatients 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.

conclusionsAt 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.

Indexed as

CatatoniaDown syndromeImmunotherapyIVIgLorazepamRegression

Identifiers

PMID41795763
PMCPMC13172088

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Read under generation 80e0d062 · epoch 390. Bibliography from PubMed, PubMed Central and OpenAlex; grants from NIH RePORTER; trial links from ClinicalTrials.gov; estimates, votes and beliefs from the OpenQuestion graph.