Table 4.
Outcome | Suramin | Placebo | ||||||||||||
---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
Instrument | Factor or behavior | Time after treatment (days) | Difference from baseline (mean ± SD) | 95% CI | d a | N | P b | P c | Difference from baseline (mean ± SD) | 95% CI | d a | N | P b | P c |
Primary outcomes | ||||||||||||||
ADOS‐2 | Comparison | 45 | −1.6 ± 0.55 | −2.3 to −0.9 | 2.9 | 5 | 0.0028 | 0.038 | −0.4 ± 0.55 | −1.1 to +0.28 | 0.7 | 5 | 0.18 | 0.16 |
Raw | 45 | −4.6 ± 1.9 | −7.0 to −2.2 | 2.4 | 5 | 0.0062 | 0.039 | −0.4 ± 1.8 | −2.7 to +1.9 | 0.22 | 5 | 0.65 | 0.58 | |
Social | 45 | −3.2 ± 1.9 | −5.6 to −0.8 | 1.7 | 5 | 0.020 | 0.043 | 0.0 ± 1.7 | −2.2 to +2.2 | 0 | 5 | 0.99 | 0.71 | |
Restr/Rep | 45 | −1.4 ± 0.89 | −2.5 to −0.29 | 1.6 | 5 | 0.025 | 0.059 | −0.4 ± 2.1 | −3.0 to +2.2 | 0.19 | 5 | 0.69 | 0.58 | |
EOWPVT | Vocabulary | 45 | −4.2 ± 8.3 | −14.5 to +6.1 | −0.51 | 5 | 0.32 | 0.50 | +2.0 ± 4.6 | −3.8 to +7.8 | 0.43 | 5 | 0.39 | 0.50 |
Secondary outcomes | ||||||||||||||
ABC | Stereotypy | 7 | −3.6 ± 2.1 | −6.2 to −1.0 | 1.7 | 5 | 0.018 | 0.043 | +0.4 ± 1.9 | −2.0 to +2.8 | −0.21 | 5 | 0.67 | 0.68 |
Stereotypy | 45 | −4.0 ± 2.3 | −6.9 to −1.1 | 1.7 | 5 | 0.019 | 0.042 | +1.0 ± 4.3 | −4.3 to +6.3 | −0.23 | 5 | 0.63 | 0.69 | |
ATEC | Total | 7 | −10 ± 7.7 | −20 to −0.46 | 1.3 | 5 | 0.044 | 0.043 | +7.2 ± 14 | −10 to +25 | −0.51 | 5 | 0.32 | 0.35 |
Language | 7 | −2.2 ± 1.5 | −4.0 to −0.36 | 1.4 | 5 | 0.021 | 0.059 | 0.0 ± 4.1 | −5.0 to +5.0 | 0 | 5 | 0.99 | 0.89 | |
Sociability | 7 | −3.6 ± 2.6 | −6.8 to −0.36 | 1.4 | 5 | 0.025 | 0.063 | −0.8 ± 2.8 | −4.3 to +2.6 | 0.29 | 5 | 0.55 | 0.58 | |
Language | 45 | −2.0 ± 1.4 | −2.7 to −0.49 | 1.4 | 5 | 0.034 | 0.059 | −0.2 ± 2.9 | −3.8 to +3.4 | 0.07 | 5 | 0.88 | 0.79 | |
CGI | Overall ASD | 45 | −1.8 ± 1.04 | −3.4 to −0.15 | 1.7 | 5 | 0.05 | n/a | 0.0 ± 0.34 | −0.55 to +0.55 | 0 | 5 | 0.99 | n/a |
E. Language | 45 | −2.0 ± 1.04 | −3.6 to −0.35 | 1.9 | 5 | 0.01 | n/a | 0.0 ± 0.34 | −0.55 to +0.55 | 0 | 5 | 0.99 | n/a | |
Social Inter. | 45 | −2.0 ± 1.04 | −3.6 to −0.35 | 1.9 | 5 | 0.01 | n/a | 0.0 ± 0.34 | −0.55 to +0.55 | 0 | 5 | 0.99 | n/a | |
RBQ | Total | 45 | −3.2 ± 5.8 | −10.4 to +4.0 | 0.55 | 5 | 0.28 | 0.22 | −0.8 ± 3.3 | −4.9 to 3.3 | 0.24 | 5 | 0.62 | 0.47 |
ADOS‐2, autism diagnostic observation schedule, 2nd edition; EOWPVT, Expressive One‐Word Picture Vocabulary Test; ABC, aberrant behavior checklist; ATEC, autism treatment evaluation checklist; CGI, clinical global impression survey; RBQ, repetitive behavior questionnaire; Restr/Rep, restricted or repetitive behaviors; Overall ASD Sx, overall ASD symptoms; E. Language, expressive language; Social Inter., social interaction. Analysis . ADOS, EOWPVT, ABC, ATEC, and RBQ scores were analyzed by paired analysis before and after treatment using each subject as their own control. CGI was analyzed by two‐way ANOVA (symptom × time before and after treatment) with post hoc correction. Nonparametric P values were not calculated (n/a). Interpretation . ADOS, ABC, ATEC, CGI, and RBQ are severity scores; negative differences from baseline reflect decreased severity, that is, improvement. EOWPVT is a performance score; negative differences reflect a decrease.
A positive Cohen's d reflects improvement, and a negative d reflects a decrease by convention. Cohen's d is likely an overestimate of the actual treatment effect based on the large mean differences and small standard deviations found before and after treatment in this small study.
P value from parametric paired t‐test analysis.
P value from nonparametric paired Wilcoxon signed‐rank sum analysis.