Emerging therapeutic approaches for Tourette syndrome and other tic disorders - a systematic review of current clinical trials.

Tourette syndrome and other tic disorders are prevalent neurodevelopmental disorders typically treated with behavioral techniques or pharmacological interventions, primarily antipsychotics. However, many patients do not achieve sufficient response to conventional treatments, underscoring the need for further research in this area. To provide a comprehensive overview of ongoing research activities, we systematically searched the clinical registries of the World Health Organization (WHO) and of the United States National Institutes of Health (NIH) for currently planned or ongoing registered clinical studies.

Limited usefulness of neurocognitive functioning indices as predictive markers for treatment response to methylphenidate or neurofeedback@home in children and adolescents with ADHD.

Introduction: Earlier studies exploring the value of executive functioning (EF) indices for assessing treatment effectiveness and predicting treatment response in attention-deficit/hyperactivity disorder (ADHD) mainly focused on pharmacological treatment options and revealed rather heterogeneous results. Envisioning the long-term goal of personalized treatment selection and intervention planning, this study comparing methylphenidate treatment (MPH) and a home-based neurofeedback intervention (NF@Home) aimed to expand previous findings by assessing objective as well as subjectively reported EF indices and by analyzing their value as treatment and predictive markers.

Methods: Children and adolescents ( = 146 in the per protocol sample) aged 7-13 years with a formal diagnosis of an inattentive or combined presentation of ADHD were examined.

Perspectives on ADHD in children and adolescents as a social construct amidst rising prevalence of diagnosis and medication use.

The diagnosis of attention-deficit hyperactivity disorder (ADHD) is based on the presence of pervasive, persistent symptoms of inattention and/or hyperactivity/impulsivity typically emerging early in life and resulting in significant functional impairment. In contrast to a worldwide epidemiological prevalence of approximately 5% in children and 2-3% in adults, there are significant variations in the prevalence of administrative ADHD diagnoses and medication use. We assert that in order to explore the underlying dynamics of this phenomenon, a thorough understanding of the construct ADHD is necessary.

Memantine as treatment for compulsivity in child and adolescent psychiatry: Descriptive findings from an incompleted randomized, double-blind, placebo-controlled trial.

Background: Autism spectrum disorder (ASD) and obsessive-compulsive disorder (OCD) are mental disorders with a considerable overlap in terms of their defining symptoms. The glutamatergic agent memantine appears to be a promising candidate for the treatment of ASD and OCD in children and adolescents. The aim of this study was to investigate the clinical efficacy and tolerability/safety of memantine in this population.

Actigraphy-Derived Sleep Profiles of Children with and without Attention-Deficit/Hyperactivity Disorder (ADHD) over Two Weeks-Comparison, Precursor Symptoms, and the Chronotype.

Although sleep problems are common in children with ADHD, their extent, preceding risk factors, and the association between neurocognitive performance and neurobiological processes in sleep and ADHD, are still largely unknown. We examined sleep variables in school-aged children with ADHD, addressing their intra-individual variability (IIV) and considering potential precursor symptoms as well as the chronotype. Additionally, in a subgroup of our sample, we investigated associations with neurobehavioral functioning ( = 44).

Medication Adherence in Adolescents with Psychiatric Disorders.

This study investigates whether adolescents' adherence to psychotropic medication is associated with demographic and socioeconomic factors, and to what extent parents' assessments of their offspring's attitudes toward treatment correspond with the adolescents' self-report. This study is part of the multicenter SEMA study (Subjective Experience and Medication Adherence in Adolescents with Psychiatric Disorders). Adolescents' subjective attitudes toward medication and their adherence were assessed using the patient and parent versions of the QATT (Questionnaire on Attitudes Toward Treatment) and the MARS (Medication Adherence Rating Scale).

Evidence-based pharmacological treatment options for ADHD in children and adolescents.

Attention-deficit hyperactivity disorder (ADHD) is a neurodevelopmental disorder characterized by inattention, hyperactivity, and impulsivity, causing functional impairment. Its prevalence lies at approximately 5% in children and adolescents and at approximately 2.5% in adults.

Personalized at-home neurofeedback compared to long-acting methylphenidate in children with ADHD: NEWROFEED, a European randomized noninferiority trial.

Background: Neurofeedback is considered a promising intervention for the treatment of attention-deficit hyperactivity disorder (ADHD). NEWROFEED is a prospective, multicentre, randomized (3:2), reference drug-controlled trial in children with ADHD aged between 7 and 13 years. The main objective of NEWROFEED was to demonstrate the noninferiority of personalized at-home neurofeedback (NF) training versus methylphenidate in the treatment of children with ADHD.

Patterns of extreme temperature-related catastrophic events in Europe including the Russian Federation: a cross-sectional analysis of the Emergency Events Database.

Objective: To investigate reported extreme temperature-related catastrophic events and associated mortality on the European continent including the Russian Federation.

Design: Cross-sectional respecting Strengthening the Reporting of Observational Studies in Epidemiology (STROBE) criteria.

Settings: Data source: Emergency Events Database (EM-DAT).

"Include me if you can"-reasons for low enrollment of pediatric patients in a psychopharmacological trial.

Background: Low recruitment in clinical trials is a common and costly problem which undermines medical research. This study aimed to investigate the challenges faced in recruiting children and adolescents with obsessive-compulsive disorder and autism spectrum disorder for a randomized, double-blind, placebo-controlled clinical trial and to analyze reasons for non-participation. The trial was part of the EU FP7 project TACTICS (Translational Adolescent and Childhood Therapeutic Interventions in Compulsive Syndromes).

Quantitative retrospective natural history modeling for orphan drug development.

The natural history of most rare diseases is incompletely understood and usually relies on studies with low level of evidence. Consistent with the goals for future research of rare disease research set by the International Rare Diseases Research Consortium in 2017, the purpose of this paper is to review the recently developed method of quantitative retrospective natural history modeling (QUARNAM) and to illustrate its usefulness through didactically selected analyses examples in an overall population of 849 patients worldwide with seven (ultra-) rare neurogenetic disorders. A quantitative understanding of the natural history of the disease is fundamental for the development of specific interventions and counseling afflicted families.

FDA orphan drug designations for lysosomal storage disorders - a cross-sectional analysis.

Purpose: To provide a quantitative clinical-regulatory insight into the status of FDA orphan drug designations for compounds intended to treat lysosomal storage disorders (LSDs).

Methods: Assessment of the drug pipeline through analysis of the FDA database for orphan drug designations with descriptive and comparative statistics.

Results: Between 1983 and 2019, 124 orphan drug designations were granted by the FDA for compounds intended to treat 28 lysosomal storage diseases.

Disease awareness or subtle product placement? Orphan diseases featured in the television series "House, M.D." - a cross-sectional analysis.

Background: Approximately 7% of the general population is affected by an orphan disease, which, in the United States, is defined as affecting fewer than 1 in 1500 people. Disease awareness is often low and time-to-diagnosis delayed. Different legislations worldwide have created incentives for pharmaceutical companies to develop drugs for orphan diseases.

Disasters in Germany and France: An Analysis of the Emergency Events Database From a Pediatric Perspective.

Objective: The objective of this study was to conduct comprehensive analyses of disaster patterns for Germany and France from a pediatric perspective.

Methods: An analysis of the Emergency Events Database (EM-DAT), epidemiological database with standard methods of descriptive and comparative statistics respecting the strengthening the reporting of observational studies in epidemiology (STROBE) criteria, was performed.

Results: Between 2006 and 2016, there were 41 and 42 disasters in Germany and France claiming 259 and 4973 lives, respectively.

"Drugs Don't Work in Patients Who Don't Take Them".

The more recent term "adherence" is now taking the place of the earlier notion of "compliance," as it emphasizes the physician-patient partnership. To date, however, it has not been clearly defined. There are many limitations to measuring adherence, and presently no standard methods have been established.

Screening for ADHD-Related Symptoms in Preschoolers Should Be Considered-Results From a Representative Sample of 5-Year-Olds From a German Metropolitan Region.

Early assessment and intervention are crucial to alleviate symptoms and prevent long-term negative outcomes in children suffering from Attention-deficit/hyperactivity disorder (ADHD). In Germany, at present, no standardized screening for ADHD is routinely administered. This study aims to evaluate a potential screening measure in a study population that is representative for a primary school entrance exam population in a German metropolitan region.

Effects of long-term methylphenidate use on growth and blood pressure: results of the German Health Interview and Examination Survey for Children and Adolescents (KiGGS).

Background: Concerns have been raised over the safety of methylphenidate (MPH), with regard to adverse effects on growth and blood pressure. Our study investigates whether, and to what extent, methylphenidate use in boys with ADHD is associated with having low body mass index (BMI), having low height, and increased systolic and diastolic blood pressure.

Methods: Data used for this study stem from the German KiGGS dataset.

"When I Stop My Medication, Everything Goes Wrong": Content Analysis of Interviews with Adolescent Patients Treated with Psychotropic Medication.

Objectives: Medication nonadherence constitutes a major problem in adolescent psychiatry. Previous studies have identified various factors associated with nonadherent behavior. The aim of this study is to explore adolescents' health beliefs and subjective perceptions relating to psychotropic medication, and to statistically link these to reported medication adherence.

Glutamatergic Agents in the Treatment of Compulsivity and Impulsivity in Child and Adolescent Psychiatry: a Systematic Review of the Literature.

Objective: Research has implicated glutamatergic projections between the various frontal subregions in the pathogenesis of compulsivity and impulsivity. Reducing striatal glutamate release, or antagonising the action of glutamate at its receptors, may therefore represent viable treatment strategies. Several glutamatergic agents with regulatory approval for other indications are available and may be of potential benefit in the treatment of compulsivity/impulsivity in psychiatric disorders in paediatric patients.

Critical appraisal of genotype assessment in molybdenum cofactor deficiency.

Introduction: Molybdenum cofactor deficiency (MoCD) is an ultra-orphan, life-threatening disease. Substrate substitution therapy has successfully been performed in single cases of MoCD type A and clinical trials are underway for drug registration. We present an innovative approach for classification of genotype severity to test the hypothesis that milder sequence variants in MoCD result in a less severe disease phenotype quantitated by patient survival.

Non-adherence to Psychotropic Medication Among Adolescents - A Systematic Review of the Literature.

Introduction: Whether patients take their medication as prescribed is of increasing importance in adolescent psychiatry since both the number of efficacious pharmaceutical treatments and the rate of prescriptions of psychotropic compounds are on the rise. Previous research showed high rates of medication nonadherence among both adolescents with medical disorders and adult patients with psychiatric disorders.

Methods: The present review was performed according to PRISMA guidelines and evaluates existing scientific literature concerning adherence to psychotropic medication among adolescents.

Defining the hidden evidence in autism research. Forty per cent of rigorously designed clinical trials remain unpublished - a cross-sectional analysis.

Autism spectrum disorders (ASD) have a prevalence of up to 2.7% and show significant rates of comorbidities. Pharmacological treatment can be difficult.