Thyroid hormone and folinic acid in young children with Down syndrome: the phase 3 ACTHYF trial

摘要: To determine whether folinic acid (FA) and thyroxine, in combination or alone, benefit psychomotor development young patients with Down syndrome (DS). The Assessment of Systematic Treatment With Folinic Acid Thyroid Hormone on Psychomotor Development Syndrome Young Children (ACTHYF) was a single-center, randomized, double-blind, placebo-controlled phase 3 trial DS infants aged 6–18 months. Patients were randomly assigned to one four treatments: placebo, acid (FA), L-thyroxine, FA+L-thyroxine, administered for 12 Randomization done by age sex. primary endpoint adjusted change from baseline Griffiths Mental Scale global quotient (GDQ) after Of 175 143 completed the study. modified intention-to-treat (mITT) population included all randomized who did not prematurely discontinue due elevated thyroid stimulating hormone (TSH). Baseline characteristics mITT well balanced between groups, reliable developmental assessment outcomes. Adjusted mean GDQ showed similar decreases groups (placebo: −5.10 [95% confidence interval (CI) −7.84 −2.37]; FA: −4.69 CI −7.73 −1.64]; L-thyroxine: −3.89 −6.94 −0.83]; FA+L-thyroxine: −3.86 −6.67 −1.06]), no significant difference any active treatment group versus placebo. This does support hypotheses that thyroxine and/or improve children are synergistic. is registered ClinicalTrials.gov number, NCT01576705.