Considerations from the 2017 IMFAR Preconference on Measuring Meaningful Outcomes from School-Age to Adulthood / Vanessa H. BAL in Autism Research, 11-11 (November 2018)  

Public ISBD [article]  inAutism Research > 11-11 (November 2018) . - p.1446-1454 Titre : Considerations from the 2017 IMFAR Preconference on Measuring Meaningful Outcomes from School-Age to Adulthood Type de document : Texte imprimé et/ou numérique Auteurs : Vanessa H. BAL, Auteur ; R. L. HENDREN, Auteur ; Tony CHARMAN, Auteur ; Leonard ABBEDUTO, Auteur ; Connie KASARI, Auteur ; L. G. KLINGER, Auteur ; W. ENCE, Auteur ; T. GLAVIN, Auteur ; G. LYONS, Auteur ; E. ROSENBERG, Auteur Article en page(s) : p.1446-1454 Langues : Anglais (eng) Mots-clés : lifespan  outcome  stakeholders  strengths Index. décimale : PER Périodiques Résumé : The autism spectrum disorder (ASD) research community is increasingly considering the importance of measuring outcomes that are meaningful to individuals with ASD and their families. The 2017 IMFAR preconference aimed to gain the perspectives of how to define and measure "meaningful outcomes" from 280 participants, including people with ASD and their families, service providers, and researchers. Six themes were identified: (a) the definition of "outcome" varies by context and perspective; (b) the need to broaden the scope of what researchers measure; (c) the need for new assessment tools; (d) the need to expand data analytic methods; (e) where to focus (with emphasis on considering different developmental stages and aspects of diversity); and (f) a need for community partnerships to bridge research and daily practice. The challenge that the research community now faces is how to move the evidence base for clinical practice forward while keeping alive the divergence of views and considerations that are relevant for thinking about complex outcomes for the highly heterogeneous group of individuals with ASD. This commentary provides recommendations, with an emphasis on lifespan viewpoints that encompass individual strengths and preferences. Autism Research 2018, 11: 1446-1454. (c) 2018 International Society for Autism Research, Wiley Periodicals, Inc. LAY SUMMARY: The 2017 IMFAR preconference aimed to gain the perspectives of how to define and measure "meaningful outcomes" from a variety of stakeholders. This commentary outlines the six themes identified from keynote and panel presentations and audience-participated discussions. Recommendations are made to emphasize perspectives that look across the lifespan and encompass individual strengths and preferences. En ligne : http://dx.doi.org/10.1002/aur.2034 Permalink : https://www.cra-rhone-alpes.org/cid/opac_css/index.php?lvl=notice_display&id=370 [article] Considerations from the 2017 IMFAR Preconference on Measuring Meaningful Outcomes from School-Age to Adulthood [Texte imprimé et/ou numérique] / Vanessa H. BAL, Auteur ; R. L. HENDREN, Auteur ; Tony CHARMAN, Auteur ; Leonard ABBEDUTO, Auteur ; Connie KASARI, Auteur ; L. G. KLINGER, Auteur ; W. ENCE, Auteur ; T. GLAVIN, Auteur ; G. LYONS, Auteur ; E. ROSENBERG, Auteur . - p.1446-1454. Langues : Anglais (eng) in Autism Research > 11-11 (November 2018) . - p.1446-1454 Mots-clés : lifespan  outcome  stakeholders  strengths Index. décimale : PER Périodiques Résumé : The autism spectrum disorder (ASD) research community is increasingly considering the importance of measuring outcomes that are meaningful to individuals with ASD and their families. The 2017 IMFAR preconference aimed to gain the perspectives of how to define and measure "meaningful outcomes" from 280 participants, including people with ASD and their families, service providers, and researchers. Six themes were identified: (a) the definition of "outcome" varies by context and perspective; (b) the need to broaden the scope of what researchers measure; (c) the need for new assessment tools; (d) the need to expand data analytic methods; (e) where to focus (with emphasis on considering different developmental stages and aspects of diversity); and (f) a need for community partnerships to bridge research and daily practice. The challenge that the research community now faces is how to move the evidence base for clinical practice forward while keeping alive the divergence of views and considerations that are relevant for thinking about complex outcomes for the highly heterogeneous group of individuals with ASD. This commentary provides recommendations, with an emphasis on lifespan viewpoints that encompass individual strengths and preferences. Autism Research 2018, 11: 1446-1454. (c) 2018 International Society for Autism Research, Wiley Periodicals, Inc. LAY SUMMARY: The 2017 IMFAR preconference aimed to gain the perspectives of how to define and measure "meaningful outcomes" from a variety of stakeholders. This commentary outlines the six themes identified from keynote and panel presentations and audience-participated discussions. Recommendations are made to emphasize perspectives that look across the lifespan and encompass individual strengths and preferences. En ligne : http://dx.doi.org/10.1002/aur.2034 Permalink : https://www.cra-rhone-alpes.org/cid/opac_css/index.php?lvl=notice_display&id=370

Efficacy and safety of memantine in children with autism spectrum disorder: Results from three phase 2 multicenter studies / A. Y. HARDAN in Autism, 23-8 (November 2019)  

Public ISBD [article]  inAutism > 23-8 (November 2019) . - p.2096-2111 Titre : Efficacy and safety of memantine in children with autism spectrum disorder: Results from three phase 2 multicenter studies Type de document : Texte imprimé et/ou numérique Auteurs : A. Y. HARDAN, Auteur ; R. L. HENDREN, Auteur ; Michael G. AMAN, Auteur ; A. ROBB, Auteur ; R. D. MELMED, Auteur ; K. A. ANDERSEN, Auteur ; R. LUCHINI, Auteur ; R. RAHMAN, Auteur ; S. ALI, Auteur ; X. D. JIA, Auteur ; M. MALLICK, Auteur ; J. E. LATEINER, Auteur ; Rohan H. C. PALMER, Auteur ; S. M. GRAHAM, Auteur Article en page(s) : p.2096-2111 Langues : Anglais (eng) Mots-clés : Asperger's disorder  Social Responsiveness Scale  autism spectrum disorders  clinical trial  medication  memantine  pervasive developmental disorder-not otherwise specified  randomized withdrawal  school-age children Index. décimale : PER Périodiques Résumé : Three phase 2 trials were conducted to assess the efficacy and long-term safety of weight-based memantine extended release (ER) treatment in children with autism spectrum disorder. MEM-MD-91, a 50-week open-label trial, identified memantine extended-release treatment responders for enrollment into MEM-MD-68, a 12-week randomized, double-blind, placebo-controlled withdrawal trial. MEM-MD-69 was an open-label extension trial in which participants from MEM-MD-68, MEM-MD-91, and open-label trial MEM-MD-67 were treated 48 weeks with memantine extended release. In MEM-MD-91, 517 (59.6%) participants were confirmed Social Responsiveness Scale responders at week 12; mean Social Responsiveness Scale total raw scores improved two to three times a minimal clinically important difference of 10 points. In MEM-MD-68, there was no difference between memantine and placebo on the primary efficacy parameter, the proportion of patients with a loss of therapeutic response (defined as 10-point increase from baseline in Social Responsiveness Scale total raw score). MEM-MD-69 exploratory analyses revealed mean standard deviation improvement in Social Responsiveness Scale total raw score of 32.4 (26.4) from baseline of the first lead-in study. No new safety concerns were evident. While the a priori-defined efficacy results of the double-blind trial were not achieved, the considerable improvements in mean Social Responsiveness Scale scores from baseline in the open-label trials were presumed to be clinically important. En ligne : http://dx.doi.org/10.1177/1362361318824103 Permalink : https://www.cra-rhone-alpes.org/cid/opac_css/index.php?lvl=notice_display&id=407 [article] Efficacy and safety of memantine in children with autism spectrum disorder: Results from three phase 2 multicenter studies [Texte imprimé et/ou numérique] / A. Y. HARDAN, Auteur ; R. L. HENDREN, Auteur ; Michael G. AMAN, Auteur ; A. ROBB, Auteur ; R. D. MELMED, Auteur ; K. A. ANDERSEN, Auteur ; R. LUCHINI, Auteur ; R. RAHMAN, Auteur ; S. ALI, Auteur ; X. D. JIA, Auteur ; M. MALLICK, Auteur ; J. E. LATEINER, Auteur ; Rohan H. C. PALMER, Auteur ; S. M. GRAHAM, Auteur . - p.2096-2111. Langues : Anglais (eng) in Autism > 23-8 (November 2019) . - p.2096-2111 Mots-clés : Asperger's disorder  Social Responsiveness Scale  autism spectrum disorders  clinical trial  medication  memantine  pervasive developmental disorder-not otherwise specified  randomized withdrawal  school-age children Index. décimale : PER Périodiques Résumé : Three phase 2 trials were conducted to assess the efficacy and long-term safety of weight-based memantine extended release (ER) treatment in children with autism spectrum disorder. MEM-MD-91, a 50-week open-label trial, identified memantine extended-release treatment responders for enrollment into MEM-MD-68, a 12-week randomized, double-blind, placebo-controlled withdrawal trial. MEM-MD-69 was an open-label extension trial in which participants from MEM-MD-68, MEM-MD-91, and open-label trial MEM-MD-67 were treated 48 weeks with memantine extended release. In MEM-MD-91, 517 (59.6%) participants were confirmed Social Responsiveness Scale responders at week 12; mean Social Responsiveness Scale total raw scores improved two to three times a minimal clinically important difference of 10 points. In MEM-MD-68, there was no difference between memantine and placebo on the primary efficacy parameter, the proportion of patients with a loss of therapeutic response (defined as 10-point increase from baseline in Social Responsiveness Scale total raw score). MEM-MD-69 exploratory analyses revealed mean standard deviation improvement in Social Responsiveness Scale total raw score of 32.4 (26.4) from baseline of the first lead-in study. No new safety concerns were evident. While the a priori-defined efficacy results of the double-blind trial were not achieved, the considerable improvements in mean Social Responsiveness Scale scores from baseline in the open-label trials were presumed to be clinically important. En ligne : http://dx.doi.org/10.1177/1362361318824103 Permalink : https://www.cra-rhone-alpes.org/cid/opac_css/index.php?lvl=notice_display&id=407

Identification of urinary metabolites that correlate with clinical improvements in children with autism treated with sulforaphane from broccoli / Stephen BENT in Molecular Autism, 9 (2018)  

Public ISBD [article]  inMolecular Autism > 9 (2018) . - 35p. Titre : Identification of urinary metabolites that correlate with clinical improvements in children with autism treated with sulforaphane from broccoli Type de document : Texte imprimé et/ou numérique Auteurs : Stephen BENT, Auteur ; B. LAWTON, Auteur ; T. WARREN, Auteur ; F. WIDJAJA, Auteur ; K. DANG, Auteur ; J. W. FAHEY, Auteur ; Brian CORNBLATT, Auteur ; J. M. KINCHEN, Auteur ; K. DELUCCHI, Auteur ; R. L. HENDREN, Auteur Article en page(s) : 35p. Langues : Anglais (eng) Mots-clés : Adolescent  Antioxidants/administration & dosage/analysis/therapeutic use  Autistic Disorder/drug therapy/urine  Biomarkers/urine  Brassica/chemistry  Child  Female  Humans  Isothiocyanates/administration & dosage/analysis/therapeutic use  Male  Metabolome  Social Behavior  Young Adult  Antioxidant  Autism  Biomarker  Metabolomics Index. décimale : PER Périodiques Résumé : Background: Children with autism spectrum disorder (ASD) have urinary metabolites suggesting impairments in several pathways, including oxidative stress, inflammation, mitochondrial dysfunction, and gut microbiome alterations. Sulforaphane, a supplement with indirect antioxidant effects that are derived from broccoli sprouts and seeds, was recently shown to lead to improvements in behavior and social responsiveness in children with ASD. We conducted the current open-label study to determine if we could identify changes in urinary metabolites that were associated with clinical improvements with the goal of identifying a potential mechanism of action. Methods: Children and young adults enrolled in a school for children with ASD and related neurodevelopmental disorders were recruited to participate in a 12-week, open-label study of sulforaphane. Fasting urinary metabolites and measures of behavior (Aberrant Behavior Checklist-ABC) and social responsiveness (Social Responsiveness Scale-SRS) were measured at baseline and at the end of the study. Pearson's correlation coefficient was calculated for the pre- to post-intervention change in each of the two clinical scales (ABS and SRS) versus the change in each metabolite. Results: Fifteen children completed the 12-week study. Mean scores on both symptom measures showed improvements (decreases) over the study period, but only the change in the SRS was significant. The ABC improved - 7.1 points (95% CI - 17.4 to 3.2), and the SRS improved - 9.7 points (95% CI - 18.7 to - 0.8). We identified 77 urinary metabolites that were correlated with changes in symptoms, and they clustered into pathways of oxidative stress, amino acid/gut microbiome, neurotransmitters, hormones, and sphingomyelin metabolism. Conclusions: Urinary metabolomics analysis is a useful tool to identify pathways that may be involved in the mechanism of action of treatments targeting abnormal physiology in ASD. Trial registration: This study was prospectively registered at clinicaltrials.gov (NCT02654743) on January 11, 2016. En ligne : https://dx.doi.org/10.1186/s13229-018-0218-4 Permalink : https://www.cra-rhone-alpes.org/cid/opac_css/index.php?lvl=notice_display&id=371 [article] Identification of urinary metabolites that correlate with clinical improvements in children with autism treated with sulforaphane from broccoli [Texte imprimé et/ou numérique] / Stephen BENT, Auteur ; B. LAWTON, Auteur ; T. WARREN, Auteur ; F. WIDJAJA, Auteur ; K. DANG, Auteur ; J. W. FAHEY, Auteur ; Brian CORNBLATT, Auteur ; J. M. KINCHEN, Auteur ; K. DELUCCHI, Auteur ; R. L. HENDREN, Auteur . - 35p. Langues : Anglais (eng) in Molecular Autism > 9 (2018) . - 35p. Mots-clés : Adolescent  Antioxidants/administration & dosage/analysis/therapeutic use  Autistic Disorder/drug therapy/urine  Biomarkers/urine  Brassica/chemistry  Child  Female  Humans  Isothiocyanates/administration & dosage/analysis/therapeutic use  Male  Metabolome  Social Behavior  Young Adult  Antioxidant  Autism  Biomarker  Metabolomics Index. décimale : PER Périodiques Résumé : Background: Children with autism spectrum disorder (ASD) have urinary metabolites suggesting impairments in several pathways, including oxidative stress, inflammation, mitochondrial dysfunction, and gut microbiome alterations. Sulforaphane, a supplement with indirect antioxidant effects that are derived from broccoli sprouts and seeds, was recently shown to lead to improvements in behavior and social responsiveness in children with ASD. We conducted the current open-label study to determine if we could identify changes in urinary metabolites that were associated with clinical improvements with the goal of identifying a potential mechanism of action. Methods: Children and young adults enrolled in a school for children with ASD and related neurodevelopmental disorders were recruited to participate in a 12-week, open-label study of sulforaphane. Fasting urinary metabolites and measures of behavior (Aberrant Behavior Checklist-ABC) and social responsiveness (Social Responsiveness Scale-SRS) were measured at baseline and at the end of the study. Pearson's correlation coefficient was calculated for the pre- to post-intervention change in each of the two clinical scales (ABS and SRS) versus the change in each metabolite. Results: Fifteen children completed the 12-week study. Mean scores on both symptom measures showed improvements (decreases) over the study period, but only the change in the SRS was significant. The ABC improved - 7.1 points (95% CI - 17.4 to 3.2), and the SRS improved - 9.7 points (95% CI - 18.7 to - 0.8). We identified 77 urinary metabolites that were correlated with changes in symptoms, and they clustered into pathways of oxidative stress, amino acid/gut microbiome, neurotransmitters, hormones, and sphingomyelin metabolism. Conclusions: Urinary metabolomics analysis is a useful tool to identify pathways that may be involved in the mechanism of action of treatments targeting abnormal physiology in ASD. Trial registration: This study was prospectively registered at clinicaltrials.gov (NCT02654743) on January 11, 2016. En ligne : https://dx.doi.org/10.1186/s13229-018-0218-4 Permalink : https://www.cra-rhone-alpes.org/cid/opac_css/index.php?lvl=notice_display&id=371

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