Autism & Developmental

Urinary dopamine metabolites as indicators of the responsiveness to fenfluramine treatment in children with autistic behavior.

Barthelemy et al. (1989) · Journal of autism and developmental disorders 1989
★ The Verdict

Low urine HVA predicted which autistic kids improved on fenfluramine, pointing to dopamine as a key player.

✓ Read this if BCBAs who work with autistic children and want a quick primer on early biomarker work.
✗ Skip if Clinicians only interested in currently available medications.

01Research in Context

01

What this study did

Doctors gave autistic children a daily pill called fenfluramine. They checked urine for a dopamine waste product called HVA before and after.

Thirteen kids joined the small trial. The team watched behavior and looked for any link between HVA change and clinical gain.

02

What they found

Six of the 13 children got noticeably better. Only these six showed a jump in urinary HVA, hinting that dopamine paths matter.

Low baseline HVA flagged likely responders, giving a simple lab clue.

03

How this fits with other research

Hansen et al. (1989) ran a similar 1989 trial and saw zero benefit, even though the drug lowered serotonin. The split may rest on different kids or rating tools.

Aman et al. (1993) later tested the same pill in youth with ID plus ADHD and found gains in attention, showing the drug can help outside autism.

Dunlap et al. (1991) used age and short tests to forecast who would thrive on methylphenidate, mirroring this paper’s urine-based prediction idea.

04

Why it matters

You now know that a cheap urine test once helped spot autistic kids who might gain from fenfluramine. While the drug is off the market, the logic lives on: baseline biology can guide medication choices. Keep the concept in mind as new dopamine drugs emerge, and always pair pills with solid behavior plans.

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When reading future drug-biomarker papers, ask if baseline levels predict response.

02At a glance

Intervention
not applicable
Design
randomized controlled trial
Sample size
13
Population
autism spectrum disorder
Finding
positive
Magnitude
medium

03Original abstract

Modifications in serotonin and dopamine metabolism were evaluated in 13 children with autistic behavior and related to their responsiveness to fenfluramine treatment. A double-blind medication-placebo crossover design was used. Each patient received 1.5 mg/kg fenfluramine daily for 3 months followed and preceded by placebo for 1 month. Clinical improvement was observed in 6 children (responders). It included reduction of behavioral symptoms such as motor activity, anxiety, mood disturbances, and distractibility. Modifications of serotonin (5-HT), dopamine (DA), and DA metabolites [homovanillic acid (HVA) and dihydroxyphenylacetic acid (DOPAC)] were assessed at urinary levels. Responders and nonresponders showed a significant decrease of urinary 5-HT levels on fenfluramine. The main differences between the two groups of subjects were found with HVA, the major metabolite of DA. Fenfluramine significantly increased HVA levels in responders whereas no significant modification was found in nonresponders. Moreover the initial level of HVA (lower in responders) significantly differentiated the two groups. These results suggest that the clinical response to fenfluramine could be related to the dopaminergic action of this drug and that urinary DA metabolite levels could be considered as indicators of the responsiveness to fenfluramine treatment in children with autistic behavior.

Journal of autism and developmental disorders, 1989 · doi:10.1007/BF02211844