title: Tetrahydrobiopterin in the treatment of children with autistic disorder: a double-blind placebo-controlled crossover study.
abstract: Twelve children, all boys, aged 4 to 7 years, with a diagnosis of autistic disorder and low concentrations of spinal 6R-l-erythro-5,6,7,8-tetrahydrobiopterin (tetrahydrobiopterin) were selected to participate in a double-blind, randomized, placebo-controlled, crossover study. The children received a daily dose of 3 mg tetrahydrobiopterin per kilogram during 6 months alternating with placebo. Treatment-induced effects were assessed with the Childhood Autism Rating Scale every third month. The results showed small nonsignificant changes in the total scores of Childhood Autism Rating Scale after 3- and 6-month treatment. Post hoc analysis looking at the 3 core symptoms of autism, that is, social interaction, communication, and stereotyped behaviors, revealed a significant improvement of the social interaction score after 6 months of active treatment. In addition, a high positive correlation was found between response of the social interaction score and IQ. The results indicate a possible effect of tetrahydrobiopterin treatment.
authors.affiliation: Department of Neuroscience, Hospital Pharmacy, Uppsala University, PET-Centre, Uppsala Imanet AB, Sweden.
authors.forename: Torsten
authors.initials: T
authors.lastname: Danfors
authors.forename: Anne-Liis
authors.initials: AL
authors.lastname: von Knorring
authors.forename: Per
authors.initials: P
authors.lastname: Hartvig
authors.forename: Bengt
authors.initials: B
authors.lastname: Langstrom
authors.forename: Robert
authors.initials: R
authors.lastname: Moulder
authors.forename: Bo
authors.initials: B
authors.lastname: Stromberg
authors.forename: Richard
authors.initials: R
authors.lastname: Torstenson
authors.forename: Ulrika
authors.initials: U
authors.lastname: Wester
authors.forename: Yasuyoshi
authors.initials: Y
authors.lastname: Watanabe
authors.forename: Orvar
authors.initials: O
authors.lastname: Eeg-Olofsson
mesh_terms: D001321:Autistic Disorder / Q000188:drug therapy* / Q000523:psychology; D001708:Biopterins / Q000009:adverse effects / Q000031:analogs & derivatives* / Q000134:cerebrospinal fluid / Q000627:therapeutic use; D002648:Child; D002675:Child, Preschool; D003142:Communication; D018592:Cross-Over Studies; D004311:Double-Blind Method; D005260:Female; D006801:Humans; D007361:Intelligence Tests; D007398:Interpersonal Relations; D008297:Male; D011569:Psychiatric Status Rating Scales; D013239:Stereotyped Behavior; D016896:Treatment Outcome
chemical_list: D001708:Biopterins; C003402:sapropterin