Correction: The Autism Impact Measure (AIM): Meaningful Change Thresholds and Core Symptom Changes Over One Year from an Online Survey in the U.S / Mariabeth SILKEY in Journal of Autism and Developmental Disorders, 53-3 (March 2023)  

Public ISBD [article]  inJournal of Autism and Developmental Disorders > 53-3 (March 2023) . - p.1298-1298 Titre : Correction: The Autism Impact Measure (AIM): Meaningful Change Thresholds and Core Symptom Changes Over One Year from an Online Survey in the U.S Type de document : texte imprimé Auteurs : Mariabeth SILKEY, Auteur ; Gonzalo DURÁN-PACHECO, Auteur ; Michelle JOHNSON, Auteur ; Chuang LIU, Auteur ; Susanne CLINCH, Auteur ; J. Kiely LAW, Auteur ; Georg LOSS, Auteur Article en page(s) : p.1298-1298 Langues : Anglais (eng) Index. décimale : PER Périodiques En ligne : https://doi.org/10.1007/s10803-022-05700-1 Permalink : https://www.cra-rhone-alpes.org/cid/opac_css/index.php?lvl=notice_display&id=500 [article] Correction: The Autism Impact Measure (AIM): Meaningful Change Thresholds and Core Symptom Changes Over One Year from an Online Survey in the U.S [texte imprimé] / Mariabeth SILKEY, Auteur ; Gonzalo DURÁN-PACHECO, Auteur ; Michelle JOHNSON, Auteur ; Chuang LIU, Auteur ; Susanne CLINCH, Auteur ; J. Kiely LAW, Auteur ; Georg LOSS, Auteur . - p.1298-1298. Langues : Anglais (eng) in Journal of Autism and Developmental Disorders > 53-3 (March 2023) . - p.1298-1298 Index. décimale : PER Périodiques En ligne : https://doi.org/10.1007/s10803-022-05700-1 Permalink : https://www.cra-rhone-alpes.org/cid/opac_css/index.php?lvl=notice_display&id=500

Development of a Goal Attainment Scale (GAS) outcome measure for clinical interventional studies in paediatric autism / Hannah STAUNTON in Autism, 29-12 (December 2025)  

Public ISBD [article]  inAutism > 29-12 (December 2025) . - p.2975-2990 Titre : Development of a Goal Attainment Scale (GAS) outcome measure for clinical interventional studies in paediatric autism Type de document : texte imprimé Auteurs : Hannah STAUNTON, Auteur ; Tammy MCIVER, Auteur ; Julian TILLMANN, Auteur ; Susanne CLINCH, Auteur ; Vivienne HANRAHAN, Auteur ; Bethany EWENS, Auteur ; Caroline AVERIUS, Auteur ; Alexandra I. BARSDORF, Auteur ; Aurélie BARANGER, Auteur ; Elizabeth BERRY KRAVIS, Auteur ; Tony CHARMAN, Auteur ; Haraldt NEERLAND, Auteur ; Alison T. SINGER, Auteur ; Pamela VENTOLA, Auteur ; Zackary JK WILLIAMS, Auteur ; Louise BARRETT, Auteur Article en page(s) : p.2975-2990 Langues : Anglais (eng) Mots-clés : adolescents  autism  autism spectrum disorders  clinical study  GAS  Goal Attainment Scaling  outcome measure  personalised  qualitative research  school-age children Index. décimale : PER Périodiques Résumé : Existing clinical outcome assessments (COAs) have limitations in capturing treatment effects in autism for those who choose to enrol in clinical research. Some COAs include items that are not relevant or equally important to all autistic people; others are not sufficiently comprehensive. Goal Attainment Scaling (GAS), which measures individual experiences against personalised goals, has been proposed as a more individualised and change-sensitive approach to assess outcomes in clinical studies. We developed a novel GAS measure for use in paediatric autism studies based on qualitative interviews with autistic adolescents, parents and clinicians. Applying a methodologically robust approach, concept elicitation interviews (N = 40) were first conducted to understand and generate a bank of personalised goals, followed by cognitive debriefing interviews (N = 39) to provide feedback on a draft GAS manual. Twenty-five personalised goals were generated for core autistic characteristics (communication, socialisation, restrictive and repetitive behaviours), as well as associated characteristics and impacts. In debriefing interviews, adolescents and parents supported the use of personalised goals and clinicians recommended using such goals in clinical studies and practice. In conclusion, GAS measures may overcome some of the challenges with existing outcome measures and complement future outcome measurement strategies for clinical studies in paediatric autism.Lay Abstract Autistic children and adolescents and their parents support the use of personalised goals as a way of measuring the effects of new treatments for those who choose to enrol in clinical research. Health professionals also recommend using personalised goals in both their research and in treatment plans. Establishing personalised goals, a process known as Goal Attainment Scaling (GAS), means identifying goals according to the needs of the individual, as opposed to using standardised outcomes for all participants. Autism is a condition with many different characteristics and degrees of impact, and the tools commonly used to measure treatment effects, known as clinical outcome assessments (COAs), have limitations. Some COAs, for example, include outcomes that are not relevant to all autistic people; others do not include enough outcomes to represent the experience of all autistic people. GAS, which measures individual experiences against personalised goals, may be complementary to existing COAs to provide an individualised or tailor-made tool to evaluate outcomes as a result of an intervention. We developed a GAS measure for researchers/health professionals to use in studies with autistic children and adolescents. Our research was based on a total of 40 interviews with adolescents, parents and clinicians, asking open-ended questions about how children and adolescents experience autism in order to understand and create a bank of potential personalised goals. We then conducted 39 further interviews to ask them for their feedback on the first draft of the measure. In the end, we compiled a list of 25 personalised goals related to core and associated characteristics of autistic children and adolescents, including goals related to communication, socialisation, and restricted and repetitive behaviours. We hope that these findings will help to improve the measurement of meaningful outcomes for autistic children and adolescents in future studies. En ligne : https://dx.doi.org/10.1177/13623613251349904 Permalink : https://www.cra-rhone-alpes.org/cid/opac_css/index.php?lvl=notice_display&id=571 [article] Development of a Goal Attainment Scale (GAS) outcome measure for clinical interventional studies in paediatric autism [texte imprimé] / Hannah STAUNTON, Auteur ; Tammy MCIVER, Auteur ; Julian TILLMANN, Auteur ; Susanne CLINCH, Auteur ; Vivienne HANRAHAN, Auteur ; Bethany EWENS, Auteur ; Caroline AVERIUS, Auteur ; Alexandra I. BARSDORF, Auteur ; Aurélie BARANGER, Auteur ; Elizabeth BERRY KRAVIS, Auteur ; Tony CHARMAN, Auteur ; Haraldt NEERLAND, Auteur ; Alison T. SINGER, Auteur ; Pamela VENTOLA, Auteur ; Zackary JK WILLIAMS, Auteur ; Louise BARRETT, Auteur . - p.2975-2990. Langues : Anglais (eng) in Autism > 29-12 (December 2025) . - p.2975-2990 Mots-clés : adolescents  autism  autism spectrum disorders  clinical study  GAS  Goal Attainment Scaling  outcome measure  personalised  qualitative research  school-age children Index. décimale : PER Périodiques Résumé : Existing clinical outcome assessments (COAs) have limitations in capturing treatment effects in autism for those who choose to enrol in clinical research. Some COAs include items that are not relevant or equally important to all autistic people; others are not sufficiently comprehensive. Goal Attainment Scaling (GAS), which measures individual experiences against personalised goals, has been proposed as a more individualised and change-sensitive approach to assess outcomes in clinical studies. We developed a novel GAS measure for use in paediatric autism studies based on qualitative interviews with autistic adolescents, parents and clinicians. Applying a methodologically robust approach, concept elicitation interviews (N = 40) were first conducted to understand and generate a bank of personalised goals, followed by cognitive debriefing interviews (N = 39) to provide feedback on a draft GAS manual. Twenty-five personalised goals were generated for core autistic characteristics (communication, socialisation, restrictive and repetitive behaviours), as well as associated characteristics and impacts. In debriefing interviews, adolescents and parents supported the use of personalised goals and clinicians recommended using such goals in clinical studies and practice. In conclusion, GAS measures may overcome some of the challenges with existing outcome measures and complement future outcome measurement strategies for clinical studies in paediatric autism.Lay Abstract Autistic children and adolescents and their parents support the use of personalised goals as a way of measuring the effects of new treatments for those who choose to enrol in clinical research. Health professionals also recommend using personalised goals in both their research and in treatment plans. Establishing personalised goals, a process known as Goal Attainment Scaling (GAS), means identifying goals according to the needs of the individual, as opposed to using standardised outcomes for all participants. Autism is a condition with many different characteristics and degrees of impact, and the tools commonly used to measure treatment effects, known as clinical outcome assessments (COAs), have limitations. Some COAs, for example, include outcomes that are not relevant to all autistic people; others do not include enough outcomes to represent the experience of all autistic people. GAS, which measures individual experiences against personalised goals, may be complementary to existing COAs to provide an individualised or tailor-made tool to evaluate outcomes as a result of an intervention. We developed a GAS measure for researchers/health professionals to use in studies with autistic children and adolescents. Our research was based on a total of 40 interviews with adolescents, parents and clinicians, asking open-ended questions about how children and adolescents experience autism in order to understand and create a bank of potential personalised goals. We then conducted 39 further interviews to ask them for their feedback on the first draft of the measure. In the end, we compiled a list of 25 personalised goals related to core and associated characteristics of autistic children and adolescents, including goals related to communication, socialisation, and restricted and repetitive behaviours. We hope that these findings will help to improve the measurement of meaningful outcomes for autistic children and adolescents in future studies. En ligne : https://dx.doi.org/10.1177/13623613251349904 Permalink : https://www.cra-rhone-alpes.org/cid/opac_css/index.php?lvl=notice_display&id=571

Effect of Children?s Autism Spectrum Disorder Severity on Family Strain and Sleep Quality: A Cross-Sectional Online Survey in the U.S / Gonzalo DURÁN-PACHECO in Journal of Autism and Developmental Disorders, 53-5 (May 2023)  

Public ISBD [article]  inJournal of Autism and Developmental Disorders > 53-5 (May 2023) . - p.1795-1808 Titre : Effect of Children?s Autism Spectrum Disorder Severity on Family Strain and Sleep Quality: A Cross-Sectional Online Survey in the U.S Type de document : texte imprimé Auteurs : Gonzalo DURÁN-PACHECO, Auteur ; Mariabeth SILKEY, Auteur ; Michelle JOHNSON, Auteur ; Chuang LIU, Auteur ; Susanne CLINCH, Auteur ; J. Kiely LAW, Auteur ; Georg LOSS, Auteur Article en page(s) : p.1795-1808 Langues : Anglais (eng) Index. décimale : PER Périodiques Résumé : To better understand the impact of children s autism spectrum disorder (ASD) severity on families, we evaluated pathways through which ASD severity affected child sleep quality, caregiver strain, and caregiver sleep quality. In a cross-sectional analysis through the U.S.-wide Simons Foundation Powering Autism Research for Knowledge (SPARK) cohort. Participants were caregivers of dependents with ASD aged 3-17 years (N=3150). We found that increased severity strongly affects caregiver strain and child sleep quality. Child sleep quality was a minor mediator of increasing caregiver strain. Caregiver sleep quality depended on ASD severity only through child sleep quality and caregiver strain. Interventions aimed at improving child sleep quality or reducing caregiver strain could positively impact families of children with ASD. En ligne : https://doi.org/10.1007/s10803-022-05457-7 Permalink : https://www.cra-rhone-alpes.org/cid/opac_css/index.php?lvl=notice_display&id=500 [article] Effect of Children?s Autism Spectrum Disorder Severity on Family Strain and Sleep Quality: A Cross-Sectional Online Survey in the U.S [texte imprimé] / Gonzalo DURÁN-PACHECO, Auteur ; Mariabeth SILKEY, Auteur ; Michelle JOHNSON, Auteur ; Chuang LIU, Auteur ; Susanne CLINCH, Auteur ; J. Kiely LAW, Auteur ; Georg LOSS, Auteur . - p.1795-1808. Langues : Anglais (eng) in Journal of Autism and Developmental Disorders > 53-5 (May 2023) . - p.1795-1808 Index. décimale : PER Périodiques Résumé : To better understand the impact of children s autism spectrum disorder (ASD) severity on families, we evaluated pathways through which ASD severity affected child sleep quality, caregiver strain, and caregiver sleep quality. In a cross-sectional analysis through the U.S.-wide Simons Foundation Powering Autism Research for Knowledge (SPARK) cohort. Participants were caregivers of dependents with ASD aged 3-17 years (N=3150). We found that increased severity strongly affects caregiver strain and child sleep quality. Child sleep quality was a minor mediator of increasing caregiver strain. Caregiver sleep quality depended on ASD severity only through child sleep quality and caregiver strain. Interventions aimed at improving child sleep quality or reducing caregiver strain could positively impact families of children with ASD. En ligne : https://doi.org/10.1007/s10803-022-05457-7 Permalink : https://www.cra-rhone-alpes.org/cid/opac_css/index.php?lvl=notice_display&id=500

Large multicenter randomized trials in autism: key insights gained from the balovaptan clinical development program / Suma JACOB in Molecular Autism, 13 (2022)  

Public ISBD [article]  inMolecular Autism > 13 (2022) . - 25 p. Titre : Large multicenter randomized trials in autism: key insights gained from the balovaptan clinical development program Type de document : texte imprimé Auteurs : Suma JACOB, Auteur ; Evdokia ANAGNOSTOU, Auteur ; Eric HOLLANDER, Auteur ; Roger JOU, Auteur ; Nora MCNAMARA, Auteur ; Linmarie SIKICH, Auteur ; Russell TOBE, Auteur ; Declan G.M. MURPHY, Auteur ; James T. MCCRACKEN, Auteur ; Elizabeth ASHFORD, Auteur ; Christopher H. CHATHAM, Auteur ; Susanne CLINCH, Auteur ; Janice SMITH, Auteur ; Kevin B. SANDERS, Auteur ; Lorraine MURTAGH, Auteur ; Jana NOELDEKE, Auteur ; Jeremy VEENSTRA-VANDERWEELE, Auteur Article en page(s) : 25 p. Langues : Anglais (eng) Mots-clés : Autism Spectrum Disorder/complications/drug therapy  Autistic Disorder/complications/drug therapy  Benzodiazepines  Humans  Pyridines  Quality of Life  Randomized Controlled Trials as Topic  Triazoles  Autism spectrum disorder  Balovaptan  Placebo response  V1aduct  Vanilla  aV1ation  attended advisory boards for Fraser, Minnesota Independence College & Community,  and F. Hoffmann-La Roche Ltd. EA has received grant support from F. Hoffmann-La  Roche Ltd and SynapDx  royalties from APPI, Springer, and Wiley  has acted as a  consultant for Quadrant, F. Hoffmann-La Roche Ltd, and SynapDx  has received  honorarium for a webinar from AIDE  has a patent on anxiety meter  and has  received a study drug and in kind supports from AMO Pharma. EH has received  research grants from the Department of Defense, Food and Drug Administration, GW  Pharma, and F. Hoffmann-La Roche Ltd  and editorial stipends from Elsevier  and  served on scientific advisory boards for GW Pharma and F. Hoffmann-La Roche Ltd.  RJ’s institution received payments for clinical trial agreements from Roche  Translational & Clinical Research Center, Inc. and Genentech, Inc. NM has  provided research support to F. Hoffmann-La Roche Ltd  and consulted for Shire  grant support has been received from Forest Research Institute, Genentech,  Lundbeck, Pfizer, F. Hoffmann-La Roche Ltd, Shire, Sunovion, and Zynerba. LS has  received funding from the National Institute of Child Health and Human  Development  was paid by F. Hoffmann-La Roche Ltd as a clinical research site  investigator for involvement in conducting the VANILLA and aV1ation studies  has  attended an advisory board for F. Hoffmann-La Roche Ltd  and has a patent pending  for new formulation of intranasal oxytocin by Duke University  part of her salary  is paid by the Duke Clinical Research Institute where she is providing thought  leadership for trials sponsored by Tris Pharmaceuticals. RT has received grant  support from Janssen and F. Hoffmann-La Roche Ltd  and has attended advisory  boards for F. Hoffmann-La Roche Ltd. DM receives funding support from the  EU/EFPIA/SFARI/Autistica/AUTISM SPEAKS Innovative Medicines Initiative 2 Joint  Undertaking (AIMS-2-TRIALS Grant No. 777394)  has received grant support from F  Hoffmann-La Roche and Shire  royalties from Springer and Wiley  and has attended  advisory boards for F Hoffmann-La Roche and Servier. JM has received research  support from F. Hoffmann-La Roche Ltd  has attended advisory boards for GW  Pharmaceuticals and F. Hoffmann-La Roche Ltd  and was a consultant for Octopharma  and TRIS Pharmaceuticals. JV-VW has received research support from NIH, the  Simons Foundation, Health Canada, F. Hoffmann-La Roche Ltd, Janssen, Acadia, and  Zynerba  honoraria for lectures at the  American Academy of Child and Adolescent Psychiatry, Karolinska Institute, Mount  Sinai, National Institute of Neurological Disease and Stroke, Florida Atlantic  University, UCLA, Stanford University, Child Mind Institute, and Pennsylvania  State University  has attended advisory boards for Roche  serves on the medical  and/or scientific advisory boards for Autism Speaks, the Simons Foundation Autism  Research Initiative, and the Brain Behavior Research Foundation  and serves as  the co-chair of the Autism and Intellectual Disability Committee for the American  Academy of Child and Adolescent Psychiatry. EAs is an employee of F. Hoffmann-La  Roche Ltd, and has stock options in F. Hoffmann-La Roche Ltd. CC and SC are  employees of F. Hoffmann-La Roche Ltd, and have stocks in F. Hoffmann-La Roche  Ltd. JS, JN, KS, and LM are employees of F. Hoffmann-La Roche Ltd and have stocks  and stock options in F. Hoffmann-La Roche Ltd. Index. décimale : PER Périodiques Résumé : BACKGROUND: Autism spectrum disorder (ASD) is a common and heterogeneous neurodevelopmental condition that is characterized by the core symptoms of social communication difficulties and restricted and repetitive behaviors. At present, there is an unmet medical need for therapies to ameliorate these core symptoms in order to improve quality of life of autistic individuals. However, several challenges are currently faced by the ASD community relating to the development of pharmacotherapies, namely in the conduct of clinical trials. Balovaptan is a V1a receptor antagonist that has been investigated to improve social communication difficulties in individuals with ASD. In this viewpoint, we draw upon our recent first-hand experiences of the balovaptan clinical development program to describe current challenges of ASD trials. DISCUSSION POINTS: The balovaptan trials were conducted in a wide age range of individuals with ASD with the added complexities associated with international trials. When summarizing all three randomized trials of balovaptan, a placebo response was observed across several outcome measures. Placebo response was predicted by greater baseline symptom severity, online recruitment of participants, and less experienced or non-academic trial sites. We also highlight challenges relating to selection of outcome measures in ASD, the impact of baseline characteristics, and the role of expectation bias in influencing trial results. CONCLUSION: Taken together, the balovaptan clinical development program has advanced our understanding of the key challenges facing ASD treatment research. The insights gained can be used to inform and improve the design of future clinical trials with the collective aim of developing efficacious therapies to support individuals with ASD. En ligne : http://dx.doi.org/10.1186/s13229-022-00505-6 Permalink : https://www.cra-rhone-alpes.org/cid/opac_css/index.php?lvl=notice_display&id=491 [article] Large multicenter randomized trials in autism: key insights gained from the balovaptan clinical development program [texte imprimé] / Suma JACOB, Auteur ; Evdokia ANAGNOSTOU, Auteur ; Eric HOLLANDER, Auteur ; Roger JOU, Auteur ; Nora MCNAMARA, Auteur ; Linmarie SIKICH, Auteur ; Russell TOBE, Auteur ; Declan G.M. MURPHY, Auteur ; James T. MCCRACKEN, Auteur ; Elizabeth ASHFORD, Auteur ; Christopher H. CHATHAM, Auteur ; Susanne CLINCH, Auteur ; Janice SMITH, Auteur ; Kevin B. SANDERS, Auteur ; Lorraine MURTAGH, Auteur ; Jana NOELDEKE, Auteur ; Jeremy VEENSTRA-VANDERWEELE, Auteur . - 25 p. Langues : Anglais (eng) in Molecular Autism > 13 (2022) . - 25 p. Mots-clés : Autism Spectrum Disorder/complications/drug therapy  Autistic Disorder/complications/drug therapy  Benzodiazepines  Humans  Pyridines  Quality of Life  Randomized Controlled Trials as Topic  Triazoles  Autism spectrum disorder  Balovaptan  Placebo response  V1aduct  Vanilla  aV1ation  attended advisory boards for Fraser, Minnesota Independence College & Community,  and F. Hoffmann-La Roche Ltd. EA has received grant support from F. Hoffmann-La  Roche Ltd and SynapDx  royalties from APPI, Springer, and Wiley  has acted as a  consultant for Quadrant, F. Hoffmann-La Roche Ltd, and SynapDx  has received  honorarium for a webinar from AIDE  has a patent on anxiety meter  and has  received a study drug and in kind supports from AMO Pharma. EH has received  research grants from the Department of Defense, Food and Drug Administration, GW  Pharma, and F. Hoffmann-La Roche Ltd  and editorial stipends from Elsevier  and  served on scientific advisory boards for GW Pharma and F. Hoffmann-La Roche Ltd.  RJ’s institution received payments for clinical trial agreements from Roche  Translational & Clinical Research Center, Inc. and Genentech, Inc. NM has  provided research support to F. Hoffmann-La Roche Ltd  and consulted for Shire  grant support has been received from Forest Research Institute, Genentech,  Lundbeck, Pfizer, F. Hoffmann-La Roche Ltd, Shire, Sunovion, and Zynerba. LS has  received funding from the National Institute of Child Health and Human  Development  was paid by F. Hoffmann-La Roche Ltd as a clinical research site  investigator for involvement in conducting the VANILLA and aV1ation studies  has  attended an advisory board for F. Hoffmann-La Roche Ltd  and has a patent pending  for new formulation of intranasal oxytocin by Duke University  part of her salary  is paid by the Duke Clinical Research Institute where she is providing thought  leadership for trials sponsored by Tris Pharmaceuticals. RT has received grant  support from Janssen and F. Hoffmann-La Roche Ltd  and has attended advisory  boards for F. Hoffmann-La Roche Ltd. DM receives funding support from the  EU/EFPIA/SFARI/Autistica/AUTISM SPEAKS Innovative Medicines Initiative 2 Joint  Undertaking (AIMS-2-TRIALS Grant No. 777394)  has received grant support from F  Hoffmann-La Roche and Shire  royalties from Springer and Wiley  and has attended  advisory boards for F Hoffmann-La Roche and Servier. JM has received research  support from F. Hoffmann-La Roche Ltd  has attended advisory boards for GW  Pharmaceuticals and F. Hoffmann-La Roche Ltd  and was a consultant for Octopharma  and TRIS Pharmaceuticals. JV-VW has received research support from NIH, the  Simons Foundation, Health Canada, F. Hoffmann-La Roche Ltd, Janssen, Acadia, and  Zynerba  honoraria for lectures at the  American Academy of Child and Adolescent Psychiatry, Karolinska Institute, Mount  Sinai, National Institute of Neurological Disease and Stroke, Florida Atlantic  University, UCLA, Stanford University, Child Mind Institute, and Pennsylvania  State University  has attended advisory boards for Roche  serves on the medical  and/or scientific advisory boards for Autism Speaks, the Simons Foundation Autism  Research Initiative, and the Brain Behavior Research Foundation  and serves as  the co-chair of the Autism and Intellectual Disability Committee for the American  Academy of Child and Adolescent Psychiatry. EAs is an employee of F. Hoffmann-La  Roche Ltd, and has stock options in F. Hoffmann-La Roche Ltd. CC and SC are  employees of F. Hoffmann-La Roche Ltd, and have stocks in F. Hoffmann-La Roche  Ltd. JS, JN, KS, and LM are employees of F. Hoffmann-La Roche Ltd and have stocks  and stock options in F. Hoffmann-La Roche Ltd. Index. décimale : PER Périodiques Résumé : BACKGROUND: Autism spectrum disorder (ASD) is a common and heterogeneous neurodevelopmental condition that is characterized by the core symptoms of social communication difficulties and restricted and repetitive behaviors. At present, there is an unmet medical need for therapies to ameliorate these core symptoms in order to improve quality of life of autistic individuals. However, several challenges are currently faced by the ASD community relating to the development of pharmacotherapies, namely in the conduct of clinical trials. Balovaptan is a V1a receptor antagonist that has been investigated to improve social communication difficulties in individuals with ASD. In this viewpoint, we draw upon our recent first-hand experiences of the balovaptan clinical development program to describe current challenges of ASD trials. DISCUSSION POINTS: The balovaptan trials were conducted in a wide age range of individuals with ASD with the added complexities associated with international trials. When summarizing all three randomized trials of balovaptan, a placebo response was observed across several outcome measures. Placebo response was predicted by greater baseline symptom severity, online recruitment of participants, and less experienced or non-academic trial sites. We also highlight challenges relating to selection of outcome measures in ASD, the impact of baseline characteristics, and the role of expectation bias in influencing trial results. CONCLUSION: Taken together, the balovaptan clinical development program has advanced our understanding of the key challenges facing ASD treatment research. The insights gained can be used to inform and improve the design of future clinical trials with the collective aim of developing efficacious therapies to support individuals with ASD. En ligne : http://dx.doi.org/10.1186/s13229-022-00505-6 Permalink : https://www.cra-rhone-alpes.org/cid/opac_css/index.php?lvl=notice_display&id=491

The Autism Impact Measure (AIM): Meaningful Change Thresholds and Core Symptom Changes Over One Year from an Online Survey in the U.S / Mariabeth SILKEY in Journal of Autism and Developmental Disorders, 53-9 (September 2023)  

Public ISBD [article]  inJournal of Autism and Developmental Disorders > 53-9 (September 2023) . - p.3422-3434 Titre : The Autism Impact Measure (AIM): Meaningful Change Thresholds and Core Symptom Changes Over One Year from an Online Survey in the U.S Type de document : texte imprimé Auteurs : Mariabeth SILKEY, Auteur ; Gonzalo DURÁN-PACHECO, Auteur ; Michelle JOHNSON, Auteur ; Chuang LIU, Auteur ; Susanne CLINCH, Auteur ; J. Kiely LAW, Auteur ; Georg LOSS, Auteur Article en page(s) : p.3422-3434 Langues : Anglais (eng) Index. décimale : PER Périodiques Résumé : Validated outcome measures with the capacity to reflect meaningful change are key to assessing potential interventions for autism spectrum disorder (ASD). We derive clinically meaningful change thresholds (MCTs) of the Autism Impact Measure (AIM) and identify factors associated with meaningful change. Baseline and 12-months follow-up survey of caregivers of 2,761 children with ASD aged 3-17 years from the U.S. Simons Foundation Powering Autism Research for Knowledge (SPARK) cohort were analyzed. Using caregiver-reported anchors for change, the 12-month change in estimated AIM MCT (95% confidence interval) for symptom improvement was -4.5 (-7.61, -1.37) points and 9.9 (5.12, 14.59) points for symptom deterioration. These anchor-based MCTs will facilitate future assessments of caregiver-reported change in AIM scores. En ligne : https://doi.org/10.1007/s10803-022-05635-7 Permalink : https://www.cra-rhone-alpes.org/cid/opac_css/index.php?lvl=notice_display&id=511 [article] The Autism Impact Measure (AIM): Meaningful Change Thresholds and Core Symptom Changes Over One Year from an Online Survey in the U.S [texte imprimé] / Mariabeth SILKEY, Auteur ; Gonzalo DURÁN-PACHECO, Auteur ; Michelle JOHNSON, Auteur ; Chuang LIU, Auteur ; Susanne CLINCH, Auteur ; J. Kiely LAW, Auteur ; Georg LOSS, Auteur . - p.3422-3434. Langues : Anglais (eng) in Journal of Autism and Developmental Disorders > 53-9 (September 2023) . - p.3422-3434 Index. décimale : PER Périodiques Résumé : Validated outcome measures with the capacity to reflect meaningful change are key to assessing potential interventions for autism spectrum disorder (ASD). We derive clinically meaningful change thresholds (MCTs) of the Autism Impact Measure (AIM) and identify factors associated with meaningful change. Baseline and 12-months follow-up survey of caregivers of 2,761 children with ASD aged 3-17 years from the U.S. Simons Foundation Powering Autism Research for Knowledge (SPARK) cohort were analyzed. Using caregiver-reported anchors for change, the 12-month change in estimated AIM MCT (95% confidence interval) for symptom improvement was -4.5 (-7.61, -1.37) points and 9.9 (5.12, 14.59) points for symptom deterioration. These anchor-based MCTs will facilitate future assessments of caregiver-reported change in AIM scores. En ligne : https://doi.org/10.1007/s10803-022-05635-7 Permalink : https://www.cra-rhone-alpes.org/cid/opac_css/index.php?lvl=notice_display&id=511

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