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Abstract Details

Superiority of Immunotherapy to Benzodiazepines and No Treatment in Down Syndrome Regression Disorder
Child Neurology and Developmental Neurology
P8 - Poster Session 8 (8:00 AM-9:00 AM)
6-007
This study sought to assess the efficacy of lorazepam, IVIg and no treatment in individuals with Down Syndrome Regression Disorder (DSRD).
DSRD is a neuropsychiatric condition manifested by acute or subacute onset of bradykinesia, catatonia, mutism, and neuropsychiatric symptoms in an otherwise healthy individual with DS. Prior studies have demonstrated efficacy of both benzodiazepines and immunotherapy in this condition although none have compared these treatments directly as first line therapy.

This study was a prospective, non-randomized, observational assessment of the therapeutic efficacy of individuals receiving IVIg, lorazepam, and no treatment for DSRD. All patients met international criteria for DSRD. Therapeutic responses were assessed at baseline, 12 weeks, and 24 weeks. The primary outcome measures were the Bush-Francis Catatonia Rating Scale (BFCRS), timed 25-foot walk (25FW),  Neuropsychiatric Inventory Questionnaire (NPI-Q), and Clinical Global Improvement Scale (CGI-S).

134 individuals qualified for this study with 47 (35%) receiving no therapy, 43 (32%) receiving lorazepam and 44 (33%) receiving IVIg. Compared to no treatment, IVIg led to improvements in all measured therapeutic responses within the first 12 weeks, with additional gains from 12 to 24 weeks, except for the CGI-S score. Lorazepam resulted in symptom improvements in 25FW, BFCRS, and CGI-S within the first 12 weeks, and in NPIT and NPIST scores in the subsequent 12 weeks, although CGI-S showed worsening symptoms. IVIg provided greater benefits than lorazepam, with notable improvements in BFCRS, NPIT, and NPIST scores during the first 12 weeks, and further improvements in 25FW, BFCRS, CGI-S, and NPIT scores during the subsequent 12 weeks.

In a large, cohort of individuals with DSRD, IVIg was superior to lorazepam on multiple primary clinical markers of disease activity, with both therapies were superior to receiving no treatment. Further, prospective randomized trials on immunotherapy in DSRD are advised.
Authors/Disclosures
Shermila Pia, MD
PRESENTER 		Dr. Pia has nothing to disclose.
Jonathan Santoro, MD (Department of Neurology, Children's Hospital Los Angeles) Dr. Santoro has received personal compensation in the range of $5,000-$9,999 for serving as a Consultant for UCB. Dr. Santoro has received personal compensation in the range of $10,000-$49,999 for serving as a Consultant for Cycle Pharma. Dr. Santoro has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Dianthus. Dr. Santoro has received personal compensation in the range of $500-$4,999 for serving as a Consultant for National Down Syndrome Society.
Saba Jafarpour, MD (Children’s Hospital of Los Angeles) Dr. Jafarpour has nothing to disclose.
Panteha Hayati Rezvan, PhD Dr. Hayati Rezvan has nothing to disclose.
Mellad Khoshnood, MD (Children's Hospital Los Angeles) Dr. Khoshnood has nothing to disclose.
Natalie Boyd (Children's Hospital Los Angeles) Natalie Boyd has nothing to disclose.
Benjamin Vogel Benjamin Vogel has nothing to disclose.
Lina Nguyen (Children's Hospital Los Angeles) Lina Nguyen has nothing to disclose.
Lilia Kazerooni, BS Miss Kazerooni has nothing to disclose.
Noemi Spinazzi, MD Dr. Spinazzi has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Ionis Pharmaceuticals. Dr. Spinazzi has received personal compensation in the range of $500-$4,999 for serving as an Expert Witness for Saldivar & Associates. The institution of Dr. Spinazzi has received research support from NIH.
Nidhiben A. Anadani, MD (University Of Oklahoma Health Science Center) Dr. Anadani has nothing to disclose.
Kristen Fisher, DO (Baylor College of Medicine) Dr. Fisher has nothing to disclose.
Robyn A. Filipink, MD (Children'S Hospital of Pittsburgh of UPMC) The institution of an immediate family member of Dr. Filipink has received research support from NIH . Dr. Filipink has received personal compensation in the range of $0-$499 for serving as a Author with Medlink.
Melanie Manning (Stanford School of Medicine) Melanie Manning has nothing to disclose.
Catherine Franklin (The University of Queensland) Catherine Franklin has nothing to disclose.
Eileen Quinn, MD Dr. Quinn has nothing to disclose.
Michael S. Rafii, MD, PhD (USC Alzheimer'S Therapeutic Research Institute) Dr. Rafii has received personal compensation in the range of $500-$4,999 for serving as a Consultant for AC Immune. Dr. Rafii has received personal compensation in the range of $5,000-$9,999 for serving as a Consultant for Keystone Bio. Dr. Rafii has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Alzheon.