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

Author(s):  
Lucie Jurek ◽  
Matias Balthazar ◽  
Sheffali Gulati ◽  
Neda Novakovic ◽  
María Núñez ◽  
...  

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.

2018 ◽  
Vol 2018 ◽  
pp. 1-21 ◽  
Author(s):  
Boram Lee ◽  
Jihong Lee ◽  
Jin-Hong Cheon ◽  
Hyun-Kyung Sung ◽  
Seung-Hun Cho ◽  
...  

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.


Autism ◽  
2019 ◽  
Vol 24 (2) ◽  
pp. 274-284 ◽  
Author(s):  
Umberto Provenzani ◽  
Laura Fusar-Poli ◽  
Natascia Brondino ◽  
Stefano Damiani ◽  
Marco Vercesi ◽  
...  

The number of trials aimed at evaluating treatments for autism spectrum disorder has been increasing progressively. However, it is not clear which outcome measures should be used to assess their efficacy, especially for treatments which target core symptoms. The present review aimed to provide a comprehensive overview regarding the outcome measures used in clinical trials for people with autism spectrum disorder. We systematically searched the Web of KnowledgeSM database between 1980 and 2016 to identify published controlled trials investigating the efficacy of interventions in autism spectrum disorder. We included 406 trials in the final database, from which a total of 327 outcome measures were identified. Only seven scales were used in more than 5% of the studies, among which only three measured core symptoms (Autism Diagnostic Observation Schedule, Childhood Autism Rating Scale, and Social Responsiveness Scale). Of note, 69% of the tools were used in the literature only once. Our systematic review has shown that the evaluation of efficacy in intervention trials for autism spectrum disorder relies on heterogeneous and often non-specific tools for this condition. The fragmentation of tools may significantly hamper the comparisons between studies and thus the discovery of effective treatments for autism spectrum disorder. Greater consensus regarding the choice of these measures should be reached.


2021 ◽  
Vol 12 ◽  
Author(s):  
Tiantian Wang ◽  
Ling Shan ◽  
Chunyue Miao ◽  
Zhida Xu ◽  
Feiyong Jia

Background: The therapeutic effect of bumetanide on autism spectrum disorder (ASD) seems to be controversial. To obtain better evidence on the efficacy of bumetanide, a systematic review and meta-analysis were performed.Methods: Randomized, placebo-controlled trials (RCTs) of bumetanide treatment in children with ASD were identified through systematic review from database inception to January 17, 2021. Subsequently, a meta-analysis was carried out to examine the effect of bumetanide on the severity of symptoms of ASD as assessed by the Childhood Autism Rating Scale (CARS) and Social Responsive Scale (SRS); core symptoms according to criteria of the Diagnostic and Statistical Manual of Mental Disorders (DSM)-5 of the American Psychiatric Association [social affect (SA), restricted, repetitive patterns of behavior, interests, or activities (RRB) and sensory symptoms]; and the therapeutic effect as assessed by Clinical Global Impressions-Efficacy (CGI-E).Results: In total, six RCTs involving 496 participants with ASD were identified in our study. The results showed that bumetanide could significantly improve the severity of the ASD symptoms measured by CARS and SRS. There was also evidence that bumetanide had positive effect on the core symptoms of ASD such as the SA and RRB, but there was no statistically significant effect on sensory symptoms. A significant positive effect on CGI-E scores in ASD patients was also observed.Conclusion: Our meta-analysis provided some support that bumetanide could improve the symptoms of children with ASD. However, additional large-scale longitudinal studies that provide clearer information and better control for confounding factors are needed to confirm our findings.


Author(s):  
Holly K. Harris ◽  
Collin Lee ◽  
Georgios D. Sideridis ◽  
William J. Barbaresi ◽  
Elizabeth Harstad

Author(s):  
Cristiano Termine ◽  
Enzo Grossi ◽  
Valentina Anelli ◽  
Ledina Derhemi ◽  
Andrea E. Cavanna

Abstract Background The association of stereotypies and tics is not rare in children with severe autism spectrum disorder (ASD). The differential diagnosis between stereotypies and tics in this patient population can be difficult; however, it could be clinically relevant because of treatment implications. Methods A total of 108 video recordings of repetitive behaviors in young patients with stereotypies in the context of ASD were reviewed by a movement disorders expert and a trainee, in order to assess the prevalence of possible co-morbid tics. The Modified Rush Videotape Rating Scale (MRVS) was used to rate tic frequency and severity. Results Out of 27 patients with stereotypies (24 males; mean age 14 years), 18 (67%) reported possible tics. The most frequently observed tics were eye blinking, shoulder shrugging, neck bending, staring, and throat clearing. The mean MRVS score was 5, indicating mild tic severity. The only significant difference between patients with tics and patients without tics was the total number of stereotypies, which was higher in the subgroup of patients without tics (p = 0.01). Conclusions Expert review of video-recordings of repetitive behaviors in young patients with ASD and stereotypies suggests the possibility of a relatively high rate of co-morbid tics. These findings need to be integrated with a comprehensive clinical assessment focusing on the diagnostic re-evaluation of heterogeneous motor manifestations.


2021 ◽  
pp. 135910452110617
Author(s):  
Evanilza T. Adorno ◽  
Daiany C de J. Dos Santos ◽  
Beatriz M. DeJesus ◽  
Adrielle A. Passos ◽  
Lavínia Teixeira-Machado

This study investigated dance practice in psychosocial and functional aspects, and quality of life in children with Down syndrome and autism spectrum disorder. Children with DS and ASD, between 3 and 12 years old, attended a dance program during 16 sessions/lessons, lasting 60 min, twice a week, in suitable place. Functional Independence Measure (FIM), Childhood Autism Rating Scale SF-36 quality of life survey, and Knowledge, Attitude and Practice Inquiry (KAP Inquiry) were applied before and after dance classes. Eleven participants concluded the study. Functional independence changes were observed in relation to self-care, sphincter control, locomotion, and communication domains. Children’ “quality of life” reported by parents showed changes in functional capacity, vitality, mental health, physical and social aspects, and general state of health domains. These findings suggest that regular dance practice can underlie psychosocial adjustments in children with DS and ASD.


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