Project description:Paediatric acute-onset neuropsychiatric syndrome (PANS) is characterised by infection-provoked abrupt and dramatic onset of obsessive compulsive disorder (OCD) or eating restriction, along with neurodevelopmental regression. Although the aetiology of PANS remains uncertain, due to a prevailing neuroimmune hypothesis, some children receive the immune modulator intravenous immunoglobulin (IVIg). Using single-cell RNA sequencing of peripheral immune cells, we examined gene expression in five children with PANS (mean age 10.2 years, range 5–17, 60% male) who were receiving IVIg. Maternal autoimmunity (n=3) and familial neurodevelopmental/neuropsychiatric disorders (n=5) were present. The index PANS event occurred age 1.8–13 years, characterised by abrupt eating restriction (n=5), developmental regression (n=4), and OCD (n=3). Single-cell RNA sequencing (144,470 cells) was performed in five patients pre- and post-IVIg, and four controls (mean age 13.5 years, range 11–16, 50% male). In PANS pre-IVIg compared to controls, there were downregulated immune pathways (defense response, innate immunity, secretory granules) in most cell types. However, NK cells exhibited upregulated immune pathways (response to corticosteroid), supporting a baseline ‘immune dysregulation’. In PANS pre-IVIg compared to controls, ribosomal pathways were upregulated in neutrophils and CD8 T cells, but downregulated in NK cells. Post-IVIg, the previously downregulated immune pathways were upregulated in most cell types, and the baseline ribosomal pathway abnormalities were reversed. Histone modification pathways (histone methyltransferase, chromatin) were downregulated in neutrophils and NK cells post-IVIg. We propose PANS is an epigenetic immune-brain disorder with cellular epigenetic, ribosomal, and immune dysregulation. Therefore, epigenetic and immune-modulating therapies, such as IVIg, may have a critical role in treating this disabling disorder.

2025-09-24 | GSE293230 | GEO

Joint Hypermobility in Paediatric Acute-Onset Neuropsychiatric Syndrome-A Preliminary Case-Control Study.

Project description: Not available

| S-EPMC8678126 | biostudies-literature

Proteomics and phosphoproteomics of medicinal cannabis plant extract (NTI164) treatment of paediatric acute-onset neuropsychiatric syndrome

Project description:Paediatric acute-onset neuropsychiatric Syndrome (PANS) is a syndrome of infection-provoked abrupt-onset obsessive-compulsive disorder (OCD) or eating restriction. This open-label trial investigated the safety and efficacy of NTI164, a cannabis-derived medicinal formulation rich in cannabinoids (minimal tetrahydrocannabinol), at 20 mg/kg/day for 12 weeks in 14 children with chronic-relapsing PANS (mean 12.1 years, females 29%). Proteomics, phosphoproteomics, and other omics approaches revealed baseline dysregulation of multiple signalling pathways in PANS versus controls that were significantly modified following NTI164 treatment.

2026-03-16 | PXD064184 | Pride

Clinical features of paediatric acute-onset neuropsychiatric syndrome: findings from a case- control study.

Project description: Not available

| S-EPMC6401534 | biostudies-literature

Epigenetic, ribosomal, and immune dysregulation in Paediatric Acute Neuropsychiatric Syndrome

Project description:This SuperSeries is composed of the SubSeries listed below.

2025-09-07 | GSE278680 | GEO

Medicinal cannabis plant extract (NTI164) modifies epigenetic, ribosomal, and immune pathways in Paediatric Acute-onset Neuropsychiatric Syndrome

Project description:Paediatric Acute-onset Neuropsychiatric Syndrome (PANS) is a syndrome of infection-provoked abrupt-onset obsessive-compulsive disorder (OCD) or eating restriction. This open-label trial investigated the safety and efficacy of NTI164, a cannabis-derived medicinal formulation rich in cannabinoids (minimal tetrahydrocannabinol), at 20mg/kg/day for 12 weeks in 14 children with chronic-relapsing PANS (mean 12.1 years, females 29%). Clinical assessments demonstrated significant improvements with NTI164 in disease severity, emotional regulation, anxiety, OCD, ADHD symptoms, and overall quality of kife (all p<0.05). Cytokine and multi-omic analyses, including transcriptomics, proteomics, phosphoproteomics, and DNA methylation revealed baseline dysregulation in epigenetic (chromatin remodelling, methylation, histone modifications, transcription factors), ribosomal, immune, and signalling pathways in PANS versus controls. These abnormalities were significantly modified following NTI164 treatment. These results suggest NTI164 has broad epigenetic and immune modulatory effects, modifying baseline immune cell abnormalities in PANS. While further controlled studies are warranted, NTI164 shows promise as a novel therapeutic for PANS.

2026-03-18 | GSE299764 | GEO

Medicinal Cannabis Plant Extract (NTI164) modifies epigenetic, ribosomal, and immune pathways in paediatric acute-onset neuropsychiatric syndrome

Project description:Paediatric Acute-onset Neuropsychiatric Syndrome (PANS) is a syndrome of infection-provoked abrupt-onset obsessive-compulsive disorder (OCD) or eating restriction. This open-label trial investigated the safety and efficacy of NTI164, a cannabis-derived medicinal formulation rich in cannabinoids (minimal tetrahydrocannabinol), at 20 mg/kg/day for 12 weeks in 14 children with chronic-relapsing PANS (mean 12.1 years, females 29%). Clinical assessments demonstrated significant improvements with NTI164 in disease severity, emotional regulation, anxiety, OCD, ADHD symptoms, and overall quality of life (all p<0.05). Cytokine and multi-omics analyses, including transcriptomics, proteomics, phosphoproteomics, and DNA methylation, revealed baseline dysregulation in epigenetic (chromatin remodelling, methylation, histone modifications, transcription factors), ribosomal, immune, and signalling pathways in PANS versus controls. These abnormalities were significantly modified following NTI164 treatment. These results suggest NTI164 has broad epigenetic and immune modulatory effects, modifying baseline immune cell abnormalities in PANS. While further controlled studies are warranted, NTI164 shows promise as a novel therapeutic for PANS.

2026-03-18 | GSE301611 | GEO

Paediatric Acute-onset Neuropsychiatric Syndrome (PANS) and intravenous immunoglobulin (IVIG): comprehensive open-label trial in ten children.

Project description: Not available

| S-EPMC9357317 | biostudies-literature