Evidence map›Paper›PMID 36824719›Full record

ArticleResearch square2023

Immunotherapy Responsiveness and Risk of Relapse in Down Syndrome Regression Disorder.

Jonathan Santoro, Noemi Spinazzi, Robyn Filipink, Panteha Hayati-Rezvan, Ryan Kammeyer, Lina Patel, Elise Sannar, Luke Dwyer, Abhik Banerjee, Mellad Khoshnood and 16 more

Abstract readPreprint
PubMed Full text at PMC Publisher
In one paragraph

Article in Research square, 2023. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Not yet cited in PubMed.

0numbers the graph read from it
0cells of the map it votes in
0citing 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

0 citing papers in PubMed.

No citing paper in PubMed yet.

4 · The record

Corrections and comments

5 · Who and what money

Authors and funding

26 authors.

Jonathan SantoroChildren's Hospital Los Angeles.ORCID 0000-0002-8350-8234
Noemi SpinazziKennedy-Krieger Institute and Johns Hopkins University.
Robyn FilipinkKennedy-Krieger Institute and Johns Hopkins University.
Panteha Hayati-RezvanKennedy-Krieger Institute and Johns Hopkins University.
Ryan KammeyerKennedy-Krieger Institute and Johns Hopkins University.
Lina PatelKennedy-Krieger Institute and Johns Hopkins University.
Elise SannarKennedy-Krieger Institute and Johns Hopkins University.
Luke DwyerKennedy-Krieger Institute and Johns Hopkins University.
Abhik BanerjeeKennedy-Krieger Institute and Johns Hopkins University.
Mellad KhoshnoodKennedy-Krieger Institute and Johns Hopkins University.
Sabaj JafarpourKennedy-Krieger Institute and Johns Hopkins University.
Natalie BoydKennedy-Krieger Institute and Johns Hopkins University.
Rebecca PartridgeKennedy-Krieger Institute and Johns Hopkins University.
Grace GombolayKennedy-Krieger Institute and Johns Hopkins University.
Alison ChristyKennedy-Krieger Institute and Johns Hopkins University.
Diego Real de AsuaKennedy-Krieger Institute and Johns Hopkins University.
Maria Del Carmen OrtegaKennedy-Krieger Institute and Johns Hopkins University.
Melanie ManningKennedy-Krieger Institute and Johns Hopkins University.
Heather Van MaterKennedy-Krieger Institute and Johns Hopkins University.
Gordon WorleyKennedy-Krieger Institute and Johns Hopkins University.
Cathy FranklinKennedy-Krieger Institute and Johns Hopkins University.
Maria StanleyKennedy-Krieger Institute and Johns Hopkins University.
Ruth BrownKennedy-Krieger Institute and Johns Hopkins University.
George CaponeKennedy-Krieger Institute and Johns Hopkins University.
Elieen Quinn
Michael Rafii

Funding

Southern California Clinical and Translational Science InstituteUL1TR001855 · NCATS · UNIVERSITY OF SOUTHERN CALIFORNIA · PI Thomas A Buchanan, Michele D. Kipke · 2016 to 2026
$81.3M
Southern California Clinical and Translational Science InstituteUL1TR000130 · NCATS · UNIVERSITY OF SOUTHERN CALIFORNIA · PI BUCHANAN, THOMAS A · 2012 to 2015
$36.0M
NCATS NIH HHS UL1 TR000130NCATS NIH HHS UL1 TR001855
6 · The paper itself

Abstract

Down syndrome regression disorder (DSRD) is a clinical symptom cluster consisting of neuropsychiatric regression without an identifiable cause. This study evaluated the clinical effectiveness of IVIg and evaluated clinical characteristics associated with relapse after therapy discontinuation. A prospective, multi-center, non-randomized, observational study was performed. Patients met criteria for DSRD and were treated with IVIg. All patients underwent a standardized wean off therapy after 9-12 months of treatment. Baseline, on therapy, and relapse scores of the Neuropsychiatric Inventory Total Score (NPITS), Clinical Global Impression-Severity (CGI-S), and the Bush-Francis Catatonia Rating Scale (BFCRS) were used to track clinical symptoms. Eighty-two individuals were enrolled in this study. Patients had lower BFCRS (MD: -6.68; 95% CI: -8.23, -5.14), CGI-S (MD: -1.27; 95% CI: -1.73, -0.81), and NPITS scores (MD: -6.50; 95% CI: -7.53, -5.47) while they were on therapy compared to baseline. Approximately 46% of the patients (n = 38) experienced neurologic relapse with wean of IVIg. Patients with neurologic relapse were more likely to have any abnormal neurodiagnostic study (χ2 = 11.82, p = 0.001), abnormal MRI (χ2 = 7.78, p = 0.005), and abnormal LP (χ2 = 5.45, p = 0.02), and a personal history of autoimmunity (OR: 6.11, p < 0.001) compared to patients without relapse. IVIg was highly effective in the treatment of DSRD. Individuals with a history of personal autoimmunity or neurodiagnostic abnormalities were more likely to relapse following weaning of immunotherapy, indicating the potential for, a chronic autoimmune etiology in some cases of DSRD.

Identifiers

PMID36824719
PMCPMC9949176

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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.