Autologous umbilical cord blood infusion for the treatment of autism in young children: A within-subjects open label study on safety (assessed via caregiver report) and efficacy / Charmain Samantha TAN ; Natasha RIARD ; Yeleswarapu Sita PADMINI ; Lourdes Mary DANIEL ; Arun PRASATH ; Ah. Moy TAN ; Thiam Chye TAN ; Rehena SULTANA ; Joyce Ching Mei LAM in Autism Research, 17-8 (August 2024)  

Public ISBD [article]  inAutism Research > 17-8 (August 2024) . - p.1721-1734 Titre : Autologous umbilical cord blood infusion for the treatment of autism in young children: A within-subjects open label study on safety (assessed via caregiver report) and efficacy Type de document : texte imprimé Auteurs : Charmain Samantha TAN, Auteur ; Natasha RIARD, Auteur ; Yeleswarapu Sita PADMINI, Auteur ; Lourdes Mary DANIEL, Auteur ; Arun PRASATH, Auteur ; Ah. Moy TAN, Auteur ; Thiam Chye TAN, Auteur ; Rehena SULTANA, Auteur ; Joyce Ching Mei LAM, Auteur Article en page(s) : p.1721-1734 Langues : Anglais (eng) Index. décimale : PER Périodiques Résumé : Abstract This study aimed to document the safety and efficacy of a single infusion of autologous umbilical cord blood (UCB) in 20 autistic children aged 24-72 months. A pre-post treatment within-subjects open label design was used. At T = 0, 6, 12, and 18 months, participants underwent detailed and structured safety evaluations (via caregiver report), Vineland Adaptive Behavior Scale (Vineland-3), Stanford Binet Intelligence Scale (SB-5), Expressive One-Word Picture Vocabulary Test, Brief Observation of Social Communication Change (BOSCC), Pervasive Developmental Disorder-Behavior Inventory, Repetitive Behavior Scale-Revised, Sensory Experience Questionnaire (SEQ-2.1), Child Behavior Checklist, Clinical Global Impression-Severity and Improvement (CGI-I) Scales, and eye-gaze tracking. UCB infusion was conducted at T = 6 months, hence, 0-6 months was the control period, and 6-18 months the follow-up period. Of 20 children recruited, 19 completed the study and 1 was withdrawn due to UCB not meeting quality control criteria for infusion. There were 15 males and 4 females with an overall mean (SD) age of 4.15 (0.62)?years. Mean (SD) cell dose administered was 38.16 (9.82) million cells/kg. None suffered serious adverse events although there were mild behavioral side effects and one unit grew coagulase negative staphylococcus from a post-thaw sample. There were no significant differences in Vineland-3, SB-5, BOSCC, and SEQ-2.1 scores at T = 12 and T = 18 months. Twelve participants had T = 18 CGI-I scores of 2-3 (minimally to much improved), seven participants had scores of 4 (no change). Autologous UCB infusion in autistic children is generally safe but not without risks, including that of infection. In this within-subjects study, some children showed global symptom improvements while others showed no change. Stem cell therapies for autism should only be conducted under strict clinical trial conditions with clear risk discussions. En ligne : https://doi.org/10.1002/aur.3187 Permalink : https://www.cra-rhone-alpes.org/cid/opac_css/index.php?lvl=notice_display&id=533 [article] Autologous umbilical cord blood infusion for the treatment of autism in young children: A within-subjects open label study on safety (assessed via caregiver report) and efficacy [texte imprimé] / Charmain Samantha TAN, Auteur ; Natasha RIARD, Auteur ; Yeleswarapu Sita PADMINI, Auteur ; Lourdes Mary DANIEL, Auteur ; Arun PRASATH, Auteur ; Ah. Moy TAN, Auteur ; Thiam Chye TAN, Auteur ; Rehena SULTANA, Auteur ; Joyce Ching Mei LAM, Auteur . - p.1721-1734. Langues : Anglais (eng) in Autism Research > 17-8 (August 2024) . - p.1721-1734 Index. décimale : PER Périodiques Résumé : Abstract This study aimed to document the safety and efficacy of a single infusion of autologous umbilical cord blood (UCB) in 20 autistic children aged 24-72 months. A pre-post treatment within-subjects open label design was used. At T = 0, 6, 12, and 18 months, participants underwent detailed and structured safety evaluations (via caregiver report), Vineland Adaptive Behavior Scale (Vineland-3), Stanford Binet Intelligence Scale (SB-5), Expressive One-Word Picture Vocabulary Test, Brief Observation of Social Communication Change (BOSCC), Pervasive Developmental Disorder-Behavior Inventory, Repetitive Behavior Scale-Revised, Sensory Experience Questionnaire (SEQ-2.1), Child Behavior Checklist, Clinical Global Impression-Severity and Improvement (CGI-I) Scales, and eye-gaze tracking. UCB infusion was conducted at T = 6 months, hence, 0-6 months was the control period, and 6-18 months the follow-up period. Of 20 children recruited, 19 completed the study and 1 was withdrawn due to UCB not meeting quality control criteria for infusion. There were 15 males and 4 females with an overall mean (SD) age of 4.15 (0.62)?years. Mean (SD) cell dose administered was 38.16 (9.82) million cells/kg. None suffered serious adverse events although there were mild behavioral side effects and one unit grew coagulase negative staphylococcus from a post-thaw sample. There were no significant differences in Vineland-3, SB-5, BOSCC, and SEQ-2.1 scores at T = 12 and T = 18 months. Twelve participants had T = 18 CGI-I scores of 2-3 (minimally to much improved), seven participants had scores of 4 (no change). Autologous UCB infusion in autistic children is generally safe but not without risks, including that of infection. In this within-subjects study, some children showed global symptom improvements while others showed no change. Stem cell therapies for autism should only be conducted under strict clinical trial conditions with clear risk discussions. En ligne : https://doi.org/10.1002/aur.3187 Permalink : https://www.cra-rhone-alpes.org/cid/opac_css/index.php?lvl=notice_display&id=533

A Direct Comparison of Three Screening Methods for Autism Spectrum Disorder in a High-Likelihood Sibling Population / Chui Mae WONG in Journal of Autism and Developmental Disorders, 55-4 (April 2024)  

Public ISBD [article]  inJournal of Autism and Developmental Disorders > 55-4 (April 2024) . - p.1274-1285 Titre : A Direct Comparison of Three Screening Methods for Autism Spectrum Disorder in a High-Likelihood Sibling Population Type de document : texte imprimé Auteurs : Chui Mae WONG, Auteur ; Nurhafizah MOHD ZAMBRI, Auteur ; Hui Hua FAN, Auteur ; Lily H. S. LAU, Auteur ; Lourdes Mary DANIEL, Auteur ; Hwan Cui KOH, Auteur Article en page(s) : p.1274-1285 Langues : Anglais (eng) Index. décimale : PER Périodiques Résumé : Targeted screening of children at increased likelihood of autism is recommended. However, autism screening tools are usually validated for use mainly in low-likelihood populations. This study compared the accuracy of the Modified Checklist for Autism in Toddlers, Revised with Follow-up (M-CHAT-R/F), the ASDetect app, and the Social Attention and Communication Surveillance, Revised (SACS-R). Siblings of autistic children underwent autism screening at 12, 18 and 30 months old. At each visit, caregivers completed the M-CHAT-R/F and ASDetect while trained nurses tested the siblings using the SACS-R. At 36 to 48 months, the siblings underwent an Autism Diagnostic Observation Schedule-Second Edition (ADOS-2) assessment. 189 siblings were screened, 141 completed the study, and 32 were confirmed to have autism. Although not validated for use at 12 months, the M-CHAT-R/F had the best sensitivity among the three tools for this age group, suggesting that early signs are already apparent to caregivers. The M-CHAT-R/F had overall better sensitivity (0.72-0.83) across all age groups, but with overall lower specificity (0.55-0.77). The SACS-R and ASDetect had better positive predictive values at 18 and 30 months (0.60-0.68), while the M-CHAT-R/F was 0.43-0.48. Negative predictive values were generally high across all three tools across all age groups (0.78-0.93). Targeted screening of children at high likelihood of autism yielded a detection rate of 22.7% and should therefore be implemented routinely to facilitate early detection and intervention. The performance of autism screening tools should be examined in higher-likelihood populations for targeted screening of these children. En ligne : https://doi.org/10.1007/s10803-024-06294-6 Permalink : https://www.cra-rhone-alpes.org/cid/opac_css/index.php?lvl=notice_display&id=550 [article] A Direct Comparison of Three Screening Methods for Autism Spectrum Disorder in a High-Likelihood Sibling Population [texte imprimé] / Chui Mae WONG, Auteur ; Nurhafizah MOHD ZAMBRI, Auteur ; Hui Hua FAN, Auteur ; Lily H. S. LAU, Auteur ; Lourdes Mary DANIEL, Auteur ; Hwan Cui KOH, Auteur . - p.1274-1285. Langues : Anglais (eng) in Journal of Autism and Developmental Disorders > 55-4 (April 2024) . - p.1274-1285 Index. décimale : PER Périodiques Résumé : Targeted screening of children at increased likelihood of autism is recommended. However, autism screening tools are usually validated for use mainly in low-likelihood populations. This study compared the accuracy of the Modified Checklist for Autism in Toddlers, Revised with Follow-up (M-CHAT-R/F), the ASDetect app, and the Social Attention and Communication Surveillance, Revised (SACS-R). Siblings of autistic children underwent autism screening at 12, 18 and 30 months old. At each visit, caregivers completed the M-CHAT-R/F and ASDetect while trained nurses tested the siblings using the SACS-R. At 36 to 48 months, the siblings underwent an Autism Diagnostic Observation Schedule-Second Edition (ADOS-2) assessment. 189 siblings were screened, 141 completed the study, and 32 were confirmed to have autism. Although not validated for use at 12 months, the M-CHAT-R/F had the best sensitivity among the three tools for this age group, suggesting that early signs are already apparent to caregivers. The M-CHAT-R/F had overall better sensitivity (0.72-0.83) across all age groups, but with overall lower specificity (0.55-0.77). The SACS-R and ASDetect had better positive predictive values at 18 and 30 months (0.60-0.68), while the M-CHAT-R/F was 0.43-0.48. Negative predictive values were generally high across all three tools across all age groups (0.78-0.93). Targeted screening of children at high likelihood of autism yielded a detection rate of 22.7% and should therefore be implemented routinely to facilitate early detection and intervention. The performance of autism screening tools should be examined in higher-likelihood populations for targeted screening of these children. En ligne : https://doi.org/10.1007/s10803-024-06294-6 Permalink : https://www.cra-rhone-alpes.org/cid/opac_css/index.php?lvl=notice_display&id=550

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