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Persico et al. Rare Dis Orphan Drugs J 2023;2:xx https://dx.doi.org/10.20517/rdodj.2023.08 Page 7 of 21

Table 2. Clinical and psychodiagnostic assessment performed at each time point
Clinical Assessment Psychodiagnostic assessment
T0 √ Physical and neurological examination √ Questionnaires: SRS, RBS-R, ABC, CPRS, CBCL, SSP, QOLA, WHOQOL,
Baseline √ Family, developmental, educational, medical and SCQ
psychiatric history √ Clinical scales: VAS, CGAS and CGI-S (clinician ratings); VAS (parental
√ Auxological parameters (height, weight, head ratings)
circumference)
√ Routine hematology and blood chemistry √ Test: ADOS2; ADI-R; GMDS-III or cognitive test; VABS-II; CARS2
T1 √ Physical and neurological examination √ Questionnaires: SRS, RBS-R, ABC, CPRS, CBCL, SSP, QOLA, WHOQOL,
4 months √ Anamnestic data (Period I) SCQ
√ Measures of therapy adherence √ Clinical scales: VAS, CGAS, and CGI-I (clinician ratings); VAS and CGI-I
√ Drug safety monitoring
(parental ratings)
√ Test: VABS-II; CARS2
√ Physical and neurological examination √ Questionnaires: SRS, RBS-R, ABC, CPRS, CBCL, SSP, QOLA, WHOQOL,
T2 √ Anamnestic data (Period II) SCQ
8 months √ Measures of therapy adherence
√ Clinical scales: VAS, CGAS, and CGI-I (clinician ratings); VAS and CGI-I
√ Drug safety monitoring
(parental ratings)
√ Test: VABS-II; CARS2
VAS: Visual Analog Scale; CGAS: Children's Global Assessment Scale; CGI-I: Clinical Global Impression-Improvement; SRS: Social
Responsiveness Scale, RBS-R: Repetitive Behaviors Scale - Revised; ABC: Aberrant Behavior Checklist; CPRS: Conners' Parent Rating Scale-
Revised, CBCL: Child Behavior Checklist, SSP: Short Sensory Profile, QOLA: The Quality of Life in Autism Questionnaire; WHOQOL: The World
Health Organization's Quality of Life Questionnaire; SCQ: Social Communication Questionnaire; GMDS-III: Griffiths Developmental Rating Scales;
VABS-II: Vineland Adaptive Behavior Scale; CARS2: Childhood Autism Rating Scale 2.

Primary outcome measures include:

A. Vineland Adaptive Behaviour Scales-II (VABS-II) , a standardized semi-structured parental interview
[40]
to measure adaptive functioning in real life. Standard scores have a mean of 100 and a standard deviation of
15.

[41]
B. Childhood Autism Rating Scale 2 (CARS2) , for trained clinicians to rate the presence and severity of
signs and symptoms of ASD by direct observation of the child. Scores range from 15 to 60 (< 30, non-
autistic; 30-36.5, mild to moderate autism; 37-60, severe autism).

C. Clinical Global Impression - Severity/Improvement scale (CGI-S/I) , a 7-point scale for the clinician to
[42]
quantify illness severity (CGI-S) at baseline, patient improvement/worsening (CGI-I) at follow-up, and
treatment side effects.

D. Visual Analog Scales (VAS) [43,44] , spanning 16 items listed in Table 3, and ranging from 0 = “no impact” to
10 = “severe impact”. Items include all core ASD symptoms based on DSM-5 criteria, fine and gross motor
function, as well as an additional set of behaviors reflecting different aspects of social cognition (imitation,
play, joint attention, enjoyment in shared play). The latter variables were chosen for their relevance in
neurodevelopmental disorders and based on our prior observations in patients receiving an open treatment
with MST . VAS was completed both by clinicians and parents at each time point.
[35]

Secondary Outcome Measures include:
A. Children's Global Assessment Scale (CGAS) , which provides a global measure of the level of
[45]
functioning in children and adolescents. The measure provides a single rating on a 0-100 scale, with higher
scores indicating better functioning.