scholarly journals Effects of bumetanide on neurobehavioral function in children and adolescents with autism spectrum disorders

2017 ◽  
Vol 7 (3) ◽  
pp. e1056-e1056 ◽  
Author(s):  
E Lemonnier ◽  
N Villeneuve ◽  
S Sonie ◽  
S Serret ◽  
A Rosier ◽  
...  
Keyword(s):  
Children And Adolescents ◽  
Rating Scale ◽  
Pediatric Population ◽  
Age Groups ◽  
Autism Spectrum ◽  
Phase 2 Trial ◽  
Neurobehavioral Function ◽  
Efficacy Outcome ◽  
Loss Of Appetite ◽  
Core Symptoms

Abstract In animal models of autism spectrum disorder (ASD), the NKCC1 chloride-importer inhibitor bumetanide restores physiological (Cl−)i levels, enhances GABAergic inhibition and attenuates electrical and behavioral symptoms of ASD. In an earlier phase 2 trial; bumetanide reduced the severity of ASD in children and adolescents (3–11 years old). Here we report the results of a multicenter phase 2B study primarily to assess dose/response and safety effects of bumetanide. Efficacy outcome measures included the Childhood Autism Rating Scale (CARS), the Social Responsive Scale (SRS) and the Clinical Global Impressions (CGI) Improvement scale (CGI-I). Eighty-eight patients with ASD spanning across the entire pediatric population (2–18 years old) were subdivided in four age groups and randomized to receive bumetanide (0.5, 1.0 or 2.0 mg twice daily) or placebo for 3 months. The mean CARS value was significantly improved in the completers group (P: 0.015). Also, 23 treated children had more than a six-point improvement in the CARS compared with only one placebo-treated individual. Bumetanide significantly improved CGI (P: 0.0043) and the SRS score by more than 10 points (P: 0.02). The most frequent adverse events were hypokalemia, increased urine elimination, loss of appetite, dehydration and asthenia. Hypokalemia occurred mainly at the beginning of the treatment at 1.0 and 2.0 mg twice-daily doses and improved gradually with oral potassium supplements. The frequency and incidence of adverse event were directly correlated with the dose of bumetanide. Therefore, bumetanide improves the core symptoms of ASD and presents a favorable benefit/risk ratio particularly at 1.0 mg twice daily.

scholarly journals Response (minimum clinically relevant change) in ASD symptoms after an intervention according to CARS-2: consensus from an expert elicitation procedure

2021 ◽  
Author(s):  
Lucie Jurek ◽  
Matias Balthazar ◽  
Sheffali Gulati ◽  
Neda Novakovic ◽  
María Núñez ◽  
...  
Keyword(s):  
Autism Spectrum Disorder ◽  
Rating Scale ◽  
Expert Knowledge ◽  
Scientific Evidence ◽  
Autism Spectrum ◽  
Expert Elicitation ◽  
Spectrum Disorder ◽  
Initial Finding ◽  
Elicitation Process ◽  
Core Symptoms

AbstractThe lack of consensual measures to monitor core change in Autism Spectrum Disorder (ASD) or response to interventions leads to difficulty to prove intervention efficacy on ASD core symptoms. There are no universally accepted outcome measures developed for measuring changes in core symptoms. However, the CARS (Childhood Autism Rating Scale) is one of the outcomes recommended in the EMA Guideline on the clinical development of medicinal products for the treatment of ASD. Unfortunately, there is currently no consensus on the response definition for CARS among individuals with ASD. The aim of this elicitation process was to determine an appropriate definition of a response on the CARS2 scale for interventions in patients with Autism Spectrum Disorder (ASD). An elicitation process was conducted following the Sheffield Elicitation Framework (SHELF). Five experts in the field of ASD and two experts in expert knowledge elicitation participated in an 1-day elicitation workshop. Experts in ASD were previously trained in the SHELF elicitation process and received a dossier of scientific evidence concerning the topic. The response definition was set as the mean clinically relevant improvement averaged over all patients, levels of functioning, age groups ***and clinicians. Based on the scientific evidence and expert judgment, a normal probability distribution was agreed to represent the state of knowledge of this response with expected value 4.03 and standard deviation 0.664. Considering the remaining uncertainty of the estimation and the available literature, a CARS-2 improvement of 4.5 points has been defined as a threshold to conclude to a response after an intervention. A CARS-2 improvement of 4.5 points could be used to evaluate interventions' meaningfulness in indivudals. This initial finding represents an important new benchmark and may aid decision makers in evaluating the efficacy of interventions in ASD.

Firearm Injuries Affecting the Pediatric Population

PEDIATRICS ◽  
1992 ◽  
Vol 89 (4) ◽  
pp. 788-790
Author(s):  
Keyword(s):  
United States ◽  
Children And Adolescents ◽  
Pediatric Population ◽  
Age Groups ◽  
Unintentional Injury ◽  
The United States ◽  
Male Adolescents ◽  
Firearm Injuries ◽  
Group 2 ◽  
Developed Nations

In the United States approximately 30 000 people die from firearm injuries each year. Many more are wounded. In the mid 1980s, more than 3000 of the dead were children and adolescents aged 1 to 19 years.1 In 1989 nearly 4000 firearm deaths were among children 1 to 19 years of age, accounting for 12% of all deaths in that age group.2 All of these deaths or injuries affect other children because the victims who are killed or wounded are frequently relatives, neighbors, or friends. Comparison data for childhood age groups demonstrate that in 1987, 203 children aged 1 to 9 years, 484 children aged 10 to 14 years, and 2705 adolescents aged 15 to 19 years died as a result of firearm injuries.1 Firearm deaths include unintentional injuries, homicides, and suicides. Among the 1- to 9-year-olds, half of the deaths were homicides and half were unintentional. Among the 10- to 14-year-olds, one third of the deaths were homicides, one third were suicides, and one third were unintentional. Among the 15- to 19-year-olds, 48% were homicides, 42% were suicides, and 8% were unintentional.1 Firearm homicides are the leading cause of death for some US subpopulations, such as urban black male adolescents and young adults.3 Table 1 indicates how firearms contributed to the deaths of children and adolescents (homicides, suicides, and all causes) in 1987. Table 2 illustrates the unusual scale of firearm violence affecting young people in the United States compared with other developed nations.4 Firearm injuries are the fourth leading cause of unintentional injury deaths to children younger than 15 years of age in the US.5

scholarly journals Vitiligo in Children and Adolescents: a Lliterature Review / Vitiligo kod dece i omladine - pregled literature

2010 ◽  
Vol 2 (3) ◽  
pp. 95-104
Author(s):  
Sonja Prćić ◽  
Verica Đuran ◽  
Dragan Katanić
Keyword(s):  
Children And Adolescents ◽  
Autoimmune Thyroiditis ◽  
Thyroid Dysfunction ◽  
Social Acceptance ◽  
Pediatric Population ◽  
Age Groups ◽  
Autoimmune Disorders ◽  
Self Image ◽  
Annual Screening

Abstract Vitiligo is an acquired, often hereditary skin depigmentation disorder, characterized by discrete, well-circumscribed, chalk-white macules or patches. It affects all age groups, but in more than half of the patients it occurs before the age of twenty, when self-image is being formed and social acceptance is of great importance. Although similar to the disease in adults, vitiligo in children and adolescents does have differences in epidemiology, association with other endocrine and/or autoimmune disorders, and treatment. This is a review of vitiligo in the pediatric population, emphasizing key differences with vitiligo in adults. According to the literature reports, we suggest that children and adolescents with vitiligo, especially non-segmental type, should perform annual screening for thyroid dysfunction, particularly for parameters of autoimmune thyroiditis.

scholarly journals The Efficacy and Safety of Acupuncture for the Treatment of Children with Autism Spectrum Disorder: A Systematic Review and Meta-Analysis

2018 ◽  
Vol 2018 ◽  
pp. 1-21 ◽  
Author(s):  
Boram Lee ◽  
Jihong Lee ◽  
Jin-Hong Cheon ◽  
Hyun-Kyung Sung ◽  
Seung-Hun Cho ◽  
...  
Keyword(s):  
Autism Spectrum Disorder ◽  
Rating Scale ◽  
Meta Analysis ◽  
Children With Autism ◽  
Autism Spectrum ◽  
Spectrum Disorder ◽  
Efficacy And Safety ◽  
Communication Ability ◽  
Mean Differences ◽  
Core Symptoms

Objectives. We aimed to summarize and critically evaluate the available evidence regarding the efficacy and safety of acupuncture for children with autism spectrum disorder (ASD). Methods. We searched 13 databases for studies published up to December 2016. Randomized controlled trials (RCTs) evaluating the efficacy of acupuncture for children with ASD were included. Outcome measures were the overall scores on scales evaluating the core symptoms of ASD and the scores for each symptom, such as social communication ability and skills, stereotypies, language ability, and cognitive function. Effect sizes were presented as mean differences (MD). Results. Twenty-seven RCTs with 1736 participants were included. Acupuncture complementary to behavioral and educational intervention significantly decreased the overall scores on the Childhood Autism Rating Scale (CARS) (MD −8.10, 95% CI −12.80 to −3.40) and the Autism Behavior Checklist (MD −8.92, 95% CI −11.29 to −6.54); however, it was unclear which of the ASD symptoms improved. Acupuncture as a monotherapy also reduced the overall CARS score. The reported adverse events were acceptable. Conclusions. This review suggests that acupuncture may be effective and safe for pediatric ASD. However, it is not conclusive due to the heterogeneity of the acupuncture treatment methods used in the studies.

scholarly journals Risperidone or Aripiprazole Can Resolve Autism Core Signs and Symptoms in Young Children: Case Study

Children ◽  
2021 ◽  
Vol 8 (5) ◽  
pp. 318
Author(s):  
Hamza A. Alsayouf ◽  
Haitham Talo ◽  
Marisa L. Biddappa ◽  
Emily De Los Reyes
Keyword(s):  
Young Children ◽  
Behavioral Interventions ◽  
Rating Scale ◽  
Pediatric Population ◽  
Signs And Symptoms ◽  
Autism Spectrum ◽  
Standard Test ◽  
Pharmacological Intervention ◽  
Retrospective Case

Risperidone and aripiprazole are approved by the USA Food and Drug Administration for the treatment of irritability and aggression in children from the ages of 5 and 6 years, respectively. However, there are no approved medications for the treatment of autism spectrum disorder (ASD) core signs and symptoms. Nevertheless, early intervention is recognized as key to improving long-term outcomes. This retrospective case study included 10 children (mean age, 2 years 10 months) with ASD who presented with persistent irritability and aggression before 4 years of age that was unresponsive to behavioral interventions and sufficiently severe to consider pharmacological intervention with risperidone or aripiprazole combined with standard supportive therapies. Besides ameliorating comorbid behaviors, improvement was observed in ASD core signs and symptoms for all patients, with minimal-to-no symptoms observed in 60% of patients according to the Childhood Autism Rating Scale 2-Standard Test and Clinical Global Impression scales. Excessive weight gain in two patients was the only adverse effect observed that required intervention. This is the first study to suggest that ASD can potentially be treated in very young children (<4 years). Clinical trials are urgently required to validate these findings among this pediatric population.

scholarly journals Real-World Use of Do-it-Yourself Artificial Pancreas Systems in Children and Adolescents: Self-Reported Clinical Outcomes (Preprint)

2019 ◽  
Author(s):  
Katarina Braune ◽  
Shane O'Donnell ◽  
Bryan Cleal ◽  
Dana Lewis ◽  
Adrian Tappe ◽  
...  
Keyword(s):  
Children And Adolescents ◽  
Clinical Outcomes ◽  
Real World ◽  
Online Survey ◽  
Pediatric Population ◽  
Artificial Pancreas ◽  
Age Groups ◽  
Closed Loop Systems ◽  
Before And After ◽  
Do It Yourself

BACKGROUND Patient-driven initiatives have made uptake of Do-it-Yourself Artificial Pancreas Systems (DIYAPS) increasingly popular amongst people with diabetes of all ages. Observational studies show improvements in glycemic control and quality of life amongst adults. However, there remains a lack of research examining experiences of children and adolescents with DIYAPS in everyday life and their social context. OBJECTIVE This study assesses self-reported clinical outcomes of the pediatric population using DIYAPS in real-world use. METHODS An online survey was distributed to caregivers, assessing HbA1c and Time in Range (TIR) before and after, and problems during DIYAPS use. RESULTS 209 caregivers from 21 countries responded. Children were 47.4% female with a median age of 10y; a mean diabetes duration of 5.1y ±3.9 and 10.3m ±10.0 experience with DIYAPS. Clinical outcomes improved significantly, including HbA1c (from 6.91% ±0.88 to 6.27% ±0.67; P<.001) and TIR (from 64.2% ±15.94 to 80.68% ±9.26; P<.001). CONCLUSIONS Improved glycemic outcomes were found across all pediatric age groups including adolescents and very young children. These findings are in line with clinical trial results from commercially developed closed-loop systems. CLINICALTRIAL The study was approved by the Charité ethics committee (EA2/140/18).

Ratings of Speech Naturalness of Children Ages 8–16 Years

2005 ◽  
Vol 100 (2) ◽  
pp. 295-304 ◽  
Author(s):  
Suzanne Coughlin-Woods ◽  
Mark E. Lehman ◽  
Paul A. Cooke
Keyword(s):  
Children And Adolescents ◽  
Rating Scale ◽  
Age Groups ◽  
Cross Sectional Study ◽  
Treatment Programs ◽  
Cross Sectional ◽  
Likert Rating Scale ◽  
Impaired Children ◽  
Normal Speaking ◽  
Speech Naturalness

The focus of this cross-sectional study was the investigation of Speech Naturalness (speech that sounds normal or natural to the listener) of 60 normal speaking children and adolescents between the ages of 8 and 16 years. 26 naive adult listeners rated the naturalness of videotaped and computer-presented speech samples, using a 9-point-Likert rating scale (1: highly natural sounding speech and 9: highly unnatural sounding speech). The children and adolescents who participated as speakers were distributed among 5 age groups (8, 10, 12, 14, and 16 yr.) with 6 boys and 6 girls in each group. Each child demonstrated normal articulation, language, voice, and speech fluency skills. Age and sex comparisons indicated boys' and girls' speech was rated comparably; however, 8-yr.-olds' speech was rated as significantly less natural than those of 12-, 14-, and 16-yr.-olds. Preliminary ratings of Speech Naturalness for normal speaking children were presented. Suggestions for the clinical application of the findings as a target criterion in treatment programs with communicatively impaired children were suggested. Replication with a larger and more representative sample is in order.

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