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. Author manuscript; available in PMC: 2023 Apr 24.
Published in final edited form as: Am J Psychiatry. 2022 Dec 7;180(1):23–40. doi: 10.1176/appi.ajp.21100992

Table 2.

Most cited biomarkers: interventional studies

BM Reference Modality Study Design Intervention Sample size
[drug/placebo]
Age
(years)
Biomarker
outcome
ASD Behavior
measures
Behavioral
improvement
Biomarker x
Behavior
Correlation*
PMID
Glutathione (GSH) Bertoglio et al., 2010 Plasma RDBPC, crossover Methyl B12 30/0 3-8 Not different AbBC, CARS, CBCL, PIA-CV No Not tested 20804367
Al-Ayadhi et al., 2013 Plasma RDBPC Camel milk 49/11 2-12 Post > Pre CARS, WSQ Yes Not tested 24069051
Faber et al., 2015 Plasma Open-label Cleanroom 10/0 3-12 Not stated ATEC, CARS, GARS-2, PDDBI No Not tested 25887094
Wink et al., 2016 Plasma RDBPC N-acetylcysteine 16/15 4-12 Post > Pre SRS, VABS No Not tested 27103982
IL-6 Tsilioni et al., 2015 Serum Open-label Diet 40/0 4-10 Pre > Post VABS Yes Yes 26418275
Melamed et al., 2018 Blood Open-label Immunoglobulin, intravenous 17/0 4-14 Not different ADOS, SRS Yes Not tested 29427532
BDNF Makkonen et al., 2011 CSF Open-label Fluoxetine 13/0 5-16 Pre > Post ATEC Yes Not tested 29056860
Pardo et al., 2013 CSF, plasma Open-label Minocycline 11/0 3-5 Pre > Post CGI-anchored, VABS No Not tested 23566357
Hellings et al., 2015 Serum Open-label Loxapine 16/0 13-65 Post > Pre RBS-R Yes Not tested 25782098
Serotonin Bent et al., 2018 Urine Open-label Sulforaphane 21/0 5-22 Not stated AbBC, SRS Yes Yes 29854372
GABA Connery et al., 2018 Plasma Open-label Immunoglobulin, intravenous 31/0 9.7 (4.4) Not different AbBC, SRS Yes Not tested 30097568
Oxytocin Andari et al., 2010 Plasma RDBPC, cross-over Oxytocin, intranasal 33/0 17-39 Post > Pre Social behavior Yes Not tested 20160081
Parker et al., 2017 Plasma RDBPC Oxytocin, intranasal 32/0 6-12 Not stated SRS Yes Yes 28696286
Alpha power Kang et al., 2019 EEG (resting state) Open-label rTMS treatment 16/16 7.8 (2.1) Post > Pre AuBC Yes Not tested 31228356
Murias et al., 2018 EEG (social vs non-social) Open-label Autologous umbilical cord blood 25/0 2-6 Post > Pre VABS Yes Yes 30070044
Fixation time (proportional looking times) Bradshaw et al., 2019 Eyes, mouth, face; social vs nonsocial Pilot, randomized PRISM 12/11 1.5-4 Not different ADOS, VABS No No 30891960
Umbricht et al., 2017 Social vs non-social RDBPC, cross-over V1a receptor antagonist, intravenous 19/0 18-45 Post > Pre (social) ASR, RMET, SCIT, AbBC reduced Yes Not tested 27711048
Andari et al., 2010 Mouth, eyes, face RDBPC, cross-over Oxytocin, intranasal 20/0 17-39 Post > Pre Social behavior Yes Yes 20160081
Fractional Anisotropy Carpenter et al., 2019 DTI Open-label Autologous umbilical cord blood 19/0 2-6 Post > Pre VABS Yes Yes 30620122
Functional activation (after biological motion) Ventola et al., 2015 Whole-brain Other CT PRT 15/0 4.5-7.7 Post > Pre (in one of 2 groups) ADOS, SRS-2 Yes Yes 25370452
Yang et al., 2016 Whole-brain Open-label PRT 20/0 5.90 (1.07) Not stated SRS Yes Yes 27845779
 Yang et al., 2017 Whole-brain Pilot VR-SCT 17/0 18-31 Not stated ACS-SP, SAT Yes Yes 28384509
*

correlation was not tested, the authors looked for the association of severity of symptoms in relation to the biomarker under study

proportional looking times; AbBC = Aberrant Behavior Checklist; ADOS = Autism Diagnostic Observation Scale; ARS = Affective Speech Recognition; ASD = Autism Spectrum Disorder; ATEC = Autism Treatment Evaluation Checklist; ACS= WMS-IV Social Perception Subtest; AuBC = Autism Behavior Checklist; BASC = Behavior Assessment System for Children; BDNF = Brain-derived neurotrophic factor; BM = Biomarker; CARS = Childhood Autism Rating Scale; CBCL = Child Behavior Checklist; CGI = Clinical Global Impression; CSF = cerebrospinal fluid; DTI = Diffusion Tensor Imaging; EEG = Electroencephalogram; FU = Follow-up after treatment; GABA = Gamma aminobutyric acid; GARS-2 = Gilliam Autism Rating Scale-2; GSH = Glutathione; IL-6 = Interleukin-6; Other CT = Other Clinical Trial; PDDBI = Pervasive Developmental Disorder Behavior Inventory; PIA-CV = Parent Interview for Autism-Clinical Version; PMID = PubMed reference number; PRISM = Pivotal Response Intervention for Social Motivation; PRT= Pivotal Response Treatment; RBS-R = Repetitive Behavior Scale-Revised; RDBPC = Randomized double-blind placebo-controlled; RMET = Reading the Mind in the Eyes Test; rTMS = Repetitive transcranial magnetic stimulation; SAT= Social Attribution Task; SCIT = Scripted Communication and Interaction Test; SRS = Social Responsiveness Scale; VABS = Vineland Adaptive Behavior Scales; VR-SCT= Virtual reality Social Cognition treatment; WSQ = Wing Subgroups Questionnaire.