Immunotherapy responsiveness and risk of relapse in Down syndrome regression disorder.

Humans Down Syndrome / therapy Immunoglobulins, Intravenous Prospective Studies Immunotherapy Recurrence

Journal

Translational psychiatry
ISSN: 2158-3188
Titre abrégé: Transl Psychiatry
Pays: United States
ID NLM: 101562664

Informations de publication

Date de publication:
08 08 2023
Historique:
received: 27 01 2023
accepted: 02 08 2023
revised: 01 08 2023
medline: 10 8 2023
pubmed: 9 8 2023
entrez: 8 8 2023
Statut: epublish

Résumé

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 (χ

Identifiants

DOI: 10.1038/s41398-023-02579-z PMID: 37553347 PMC: PMC10409776
pubmed: 37553347
doi: 10.1038/s41398-023-02579-z
pii: 10.1038/s41398-023-02579-z
pmc: PMC10409776
doi:

Substances chimiques

Immunoglobulins, Intravenous 0

Types de publication

Observational Study Journal Article Research Support, N.I.H., Extramural Research Support, Non-U.S. Gov't

Langues

eng

Sous-ensembles de citation

IM

Pagination

276

Subventions

Organisme : NICHD NIH HHS
ID : P50 HD103538
Pays : United States
Organisme : NCATS NIH HHS
ID : UL1 TR000130
Pays : United States
Organisme : NCATS NIH HHS
ID : UL1 TR001855
Pays : United States

Commentaires et corrections

Type : UpdateOf

Informations de copyright

© 2023. Springer Nature Limited.

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Auteurs

Jonathan D Santoro (JD)

Division of Neurology, Children's Hospital Los Angeles, Los Angeles, CA, USA. santoroj@usc.edu.
Department of Neurology, Keck School of Medicine of the University of Southern California, Los Angeles, CA, USA. santoroj@usc.edu.
ORCID: 0000-0002-8350-8234

Noemi A Spinazzi (NA)

Department of Pediatrics, Benioff Children's Hospital, University of California San Francisco, San Francisco, CA, USA.

Robyn A Filipink (RA)

Division of Child Neurology, UPMC Children's Hospital of Pittsburgh, Pittsburgh, PA, USA.

Panteha Hayati-Rezvan (P)

Division of Research on Children, Youth and Families, Keck School of Medicine of USC, Los Angeles, CA, USA.

Ryan Kammeyer (R)

Department of Neurology, Children's Hospital of Colorado, Aurora, CO, USA.

Lina Patel (L)

Department of Psychiatry, University of Colorado School of Medicine, Aurora, CO, USA.

Elise A Sannar (EA)

Department of Psychiatry, University of Colorado School of Medicine, Aurora, CO, USA.

Luke Dwyer (L)

Department of Psychiatry, University of Utah, Salt Lake City, UT, USA.

Abhik K Banerjee (AK)

Division of Neurology, Children's Hospital Los Angeles, Los Angeles, CA, USA.

Mellad Khoshnood (M)

Division of Neurology, Children's Hospital Los Angeles, Los Angeles, CA, USA.

Saba Jafarpour (S)

Division of Neurology, Children's Hospital Los Angeles, Los Angeles, CA, USA.

Natalie K Boyd (NK)

Division of Neurology, Children's Hospital Los Angeles, Los Angeles, CA, USA.

Rebecca Partridge (R)

Virginia Mason Health System, Issaquah, WA, USA.

Grace Y Gombolay (GY)

Department of Pediatrics, Division of Neurology Emory University and Children's Healthcare of Atlanta, Atlanta, GA, USA.

Alison L Christy (AL)

Providence Health System, Portland, OR, USA.

Diego Real de Asua (D)

Adult Down Syndrome Outpatient Clinic, Department of Internal Medicine, Fundación de Investigación Biomédica, Hospital Universitario de La Princesa, Madrid, Spain.

Maria Del Carmen Ortega (M)

Department of Psychiatry, Clinica Universidad de Navarra, Madrid, Spain.
ORCID: 0000-0002-3964-0123

Melanie A Manning (MA)

Department of Pediatrics, Stanford University School of Medicine, Palo Alto, CA, USA.

Heather Van Mater (H)

Division of Rheumatology, Department of Pediatrics, Duke University School of Medicine, Durham, NC, USA.

Gordan Worley (G)

Division of Pediatric Neurology and Developmental Medicine, Department of Pediatrics, Duke University School of Medicine, Durham, NC, USA.

Cathy Franklin (C)

Queensland Center for Intellectual and Developmental Disability, Mater Research Institute, The University of Queensland, South Brisbane, QLD, Australia.

Maria A Stanley (MA)

Department of Pediatrics, University of Wisconsin School of Medicine and Public Health, Madison, WI, USA.

Ruth Brown (R)

Department of Psychology, Virginia Commonwealth University, Richmond, VA, USA.

George T Capone (GT)

Department of Pediatrics, Johns Hopkins School of Medicine, Baltimore, MD, USA.

Eileen A Quinn (EA)

Department of Pediatrics, University of Toledo College of Medicine and Life Science, Toledo, OH, USA.

Michael S Rafii (MS)

Department of Neurology, Keck School of Medicine of the University of Southern California, Los Angeles, CA, USA.
Alzheimer's Therapeutic Research Institute (ATRI), Keck School of Medicine at the University of Southern California, San Diego, CA, USA.
ORCID: 0000-0003-2640-2094

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Classifications MeSH

questionsmedicales.fr › Eucaryotes › Animaux › Chordés › Vertébrés › Mammifères › Eutheria › Primates › Haplorhini › Catarrhini › Hominidae › Humains › Immunotherapy responsiveness and risk of...
questionsmedicales.fr › Malformations et maladies congénitales, héréditaires et néonatales › Maladies génétiques congénitales › Maladies chromosomiques › Syndrome de Down › Immunotherapy responsiveness and risk of...
questionsmedicales.fr › Acides aminés, peptides et protéines › Protéines › Globulines › Sérum-globulines › Immunoglobulines › Anticorps › Immunoglobulines par voie veineuse › Immunotherapy responsiveness and risk of...
questionsmedicales.fr › Environnement et santé publique › Santé publique › Méthodes épidémiologiques › Caractéristiques des études épidémiologiques › Études épidémiologiques › Études de cohortes › Études prospectives › Immunotherapy responsiveness and risk of...
questionsmedicales.fr › Thérapeutique › Biothérapie › Immunothérapie › Immunotherapy responsiveness and risk of...
questionsmedicales.fr › États, signes et symptômes pathologiques › Processus pathologiques › Caractéristiques de la maladie › Récidive › Immunotherapy responsiveness and risk of...