Fenfluramine below the age of 2 years in Dravet syndrome: What about safety and efficacy?

Dravet syndrome (DS) is a rare developmental and epileptic encephalopathy. Infants with DS are especially vulnerable to the detrimental effects of prolonged and frequent seizures on development. Fenfluramine (FFA) is approved for the treatment of DS in patients aged 2 years and older. This study aims to evaluate the safety and efficacy of FFA in patients with DS younger than 2 years. We analyzed safety, tolerability, seizure, and neuropsychological outcome in a real-world setting. Developmental profile was investigated using Griffiths Mental Development Scales (GMDS). Five patients received FFA at a mean age of 14.9 months (9.6-18.6). Median follow-up was 13 months (interquartile range [IQR] = 12.9-24.4). All patients showed good tolerance to FFA. No significant variation of body mass index or echocardiographic issue was observed. Monthly median convulsive seizure frequency (MCSF) was 1.71 (IQR = 1.56-3.27) at the 6-month baseline period and .92 (IQR = .43-1.28) at last follow-up, with a median 54.43 (IQR = 40.91-60.83) percentage reduction in MCSF. Two of five patients had a performance improvement on GMDS subscales. Overall, the use of FFA below the age of 2 years in our small sample of patients was safe and represents a promising opportunity for seizure control and for protection of the neurodevelopmental outcome.

Juvenile Chronic Inflammatory Demyelinating Polyneuropathy Epidemiology in Sardinia, Insular Italy.

BACKGROUND: Chronic inflammatory demyelinating polyradiculoneuropathy (CIDP) is a rare and disabling immunomediated radiculoneuropathy. Its worldwide epidemiology is heterogeneous and, in adults, CIDP prevalence varies from 0.6 to 9 cases per 100,000 population. Juvenile CIDP (jCIDP) is even rarer, with age-specific prevalence rates varying from 0.23 to 1.26 owing to different diagnostic criteria (American Academy of Neurology [AAN] and European Federation of Neurological Societies/Peripheral Nerve Society [EFNS/PNS]), different age grouping or, genuine differences. OBJECTIVES: We assessed jCIDP incidence and prevalence in Sardinia, an area at very-high risk for autoimmune diseases, using comparable methods. DESIGN: The study area was the northern Sardinia, insular Italy, with 491,571 inhabitants and a pediatric population (0-18 years) of 79,086 individuals. RESULTS: On prevalence day (December 31, 2019) the total crude, age-specific prevalence rate were 6.32 per 100,000 according with AAN criteria, 7.58 per 100,000 population with European Neuromuscular Center (ENMC) criteria, and 8.85 per 100,000 population with both 2006 and 2010 EFNS/PNS criteria. Crude mean incidence rate were 0.42 per 100,000 per year with AAN criteria, 0.50 per 100,000 per year with ENMC criteria, and 0.59 per 100,000 per year using 2006 and 2010 EFNS/PNS criteria. Of the eight patients, six had typical CIDP, one had multifocal-acquired demyelinating sensory and motor neuropathy (MADSAM), and one chronic immune sensory polyradiculopathy (CISP). Patient's disability was generally mild. Clinical course was progressive, monophasic, or relapsing. CONCLUSION: jCIDP prevalence and incidence rates in Sardinia were criteria-dependent, the lowest obtained when using AAN criteria, the highest using the EFNS/PNS. Nonetheless, even with the exclusion of the "possible" category, by using comparable methodology, prevalence rates in Sardinia are considerably higher than the range reported in all previous jCIDP studies.

COVID-19 Pandemic School Disruptions and Acute Mental Health in Children and Adolescents.

Importance: There are suggestions that school pressure may be stressful and a factor in child and adolescent mental health disturbances, but data about this association are scarce and inconclusive. Objective: To assess whether varying degrees of school interruption were associated with changes in emergency department (ED) psychiatric visits of children and adolescents before and after the COVID-19 outbreak. Design, Setting, and Participants: A cross-sectional observational study was conducted at 9 urban university hospitals in Italy. All ED visits from January 1, 2018, to December 31, 2021, for psychiatric reasons of patients younger than 18 years were examined for demographic characteristics and type of psychopathologic factors. Data analysis was conducted from July 1 to August 31, 2023. Exposure: The disruption in the usual succession of school and holiday periods brought on by the COVID-19 pandemic at different times and with various degrees of intensity. Main Outcomes and Measures: Total number of pediatric ED visits, psychiatric ED visits, and psychiatric ED visits categorized by specific reasons (eg, psychomotor agitation, suicide ideation [SI] or suicide attempt [SA], and eating disorders) on a weekly basis. Results: A total of 13 014 psychiatric ED visits (1.3% of all pediatric ED visits) were recorded (63.2% females; mean [SD] age, 13.8 [3.8] years). The number of ED psychiatric visits increased over time (incidence rate ratio [IRR], 1.19; 95% CI, 1.16-1.22 for each year). Significant increases in ED visits were observed for eating disorders (294.8%), SI (297.8%), and SA (249.1%). School opening, but not social lockdown restriction, was associated with an increase in the number of ED psychiatric visits (IRR, 1.29; 95% CI, 1.23-1.34), which was evident for females and for SI with SA. Socioeconomic status was associated with an increase in psychiatric visits for males (IRR, 1.12; 95% CI, 1.04-1.20) but not females (IRR, 1.04; 95% CI, 0.98-1.10). Conclusions and Relevance: In this study, school opening was associated with an increased incidence of acute psychiatric emergencies among children and adolescents, suggesting that school can be a substantial source of stress with acute mental health implications.