Lack of effect of vitamin D3 supplementation in autism: a 20-week, placebo-controlled RCT

Objectives Data suggest a potential role for vitamin D in autism spectrum disorder (ASD). We wanted to assess the effect of vitamin D-3 supplementation compared with placebo in children with ASD. Design This was a double-blind, randomised, placebo-controlled trial. Setting A paediatric outpatient centre at high latitude over the winter season in Dublin, Ireland (53 degrees N). Patients 42 children with ASD. Interventions 2000 IU vitamin D 3 supplementation or placebo daily for 20 weeks. Main outcome measures Assessments were completed at baseline and after 20 weeks of supplementation. The primary outcome was the stereotypic behaviour subscale from the Aberrant Behaviour Checklist (ABC). Secondary exploratory outcomes included additional subscales from the ABC, the Social Responsiveness Scale and rating on the Developmental Disabilities-Children's Global Assessment Scale (DD-CGAS) as well as biochemical parameters of total vitamin D status (25-hydroxyvitamin D (25(OH) D)), immunity and systemic inflammation. Results 38 children completed the trial. Baseline 25(OH) D was 254.2 +/- 19.7 nmol/L. Following vitamin D-3 supplementation, there was a significant increase in 25(OH) D to 83.8 nmol/L (p=0.0016) but no effect on the primary endpoint. However, there was an improvement in self-care on DD-CGAS (p=0.02). In contrast, there was also a trend toward decreased inappropriate speech in the placebo group (p=0.08). Conclusion Vitamin D supplementation had no effect on the primary outcome with limited and inconsistent effects in children with ASD. Considering the other promising data as well as the relative safety and cheapness of vitamin D supplementation, further trials are warranted.