Intranasal Oxytocin Improves Behavior in Boys With Prader-Willi Syndrome | Research

The takeaway

Three months of intranasal oxytocin improved social behavior and eating behavior in boys with Prader-Willi syndrome aged 3-11 years, with no safety concerns in this randomized crossover trial.

Social behavior: +4.5 vs -4.0 (P=.025)
Boys with PWS improved on oxytocin while deteriorating on placebo over 3 months

What the researchers found

Intranasal oxytocin (16-40 IU/day for 3 months) improved social behavior and stabilized eating behavior in boys with PWS and in children with the deletion genotype, with no overall group effects.

Why it matters

PWS is characterized by severe behavioral challenges and uncontrollable hunger with no effective pharmacological treatment. Finding that oxytocin improves behavior in specific PWS subgroups opens a potential treatment avenue for this difficult condition.

The numbers in context

26 children; 16-40 IU/day; 3 months; boys: +4.5 vs -4.0 (P=.025); hyperphagia 0.0 vs -3.5 (P=.046); deletion subtype improved; no SAEs

How the study worked

Randomized, double-blind, placebo-controlled, crossover study in the Dutch PWS Reference Center. 26 children aged 3-11 years. Intranasal oxytocin 16-40 IU/day for 3 months. Outcomes: Oxytocin Questionnaire and Dykens hyperphagia questionnaire.

Who was studied

Children with Prader-Willi syndrome aged 3-11 years (Dutch PWS Reference Center)

What this study cannot tell us

Small sample size (n=26). Subgroup analyses (boys, deletion genotype) were not pre-specified primary outcomes. Crossover design assumes no carryover effects. Not all children showed benefit.

How to read the evidence

Small randomized, double-blind, placebo-controlled crossover trial. Moderate evidence quality limited by sample size and post-hoc subgroup analyses.

When this study was published

Published in 2021, contributing to the growing evidence for oxytocin therapy in PWS and related neurodevelopmental conditions.

The bigger picture

PWS results from dysfunction in the hypothalamic region where oxytocin is produced. Restoring oxytocin signaling through intranasal administration addresses a fundamental aspect of the syndrome's pathology, with potential to improve both social functioning and the devastating hyperphagia that defines the condition.

Questions still open

Why does oxytocin benefit boys but not girls with PWS?
What is the optimal long-term dosing of oxytocin for PWS?
Should oxytocin treatment be initiated earlier in infancy for PWS?

Common questions

Can oxytocin help children with Prader-Willi syndrome?

This trial found that intranasal oxytocin improved social behavior and stabilized eating behavior in boys with PWS. Children with the deletion type of PWS also showed improvement. The treatment was safe and well-tolerated, but not all children benefited, so individual responses should be carefully monitored.

Why might oxytocin help with Prader-Willi syndrome?

PWS involves dysfunction in the hypothalamus, where oxytocin is produced. Patients with PWS may have impaired oxytocin signaling, contributing to social difficulties and eating problems. Intranasal oxytocin may partially restore this missing signaling.

Read the original research

Oxytocin in young children with Prader-Willi syndrome: Results of a randomized, double-blind, placebo-controlled, crossover trial investigating 3 months of oxytocin.

Clinical endocrinology, 94(5), 774-785

View on PubMed ↗Publisher’s paper ↗

Citation

Damen, Layla; Grootjen, Lionne N; Juriaans, Alicia F; Donze, Stephany H; Huisman, T Martin; Visser, Jenny A; Delhanty, Patric J D; Hokken-Koelega, Anita C S. (2021). Oxytocin in young children with Prader-Willi syndrome: Results of a randomized, double-blind, placebo-controlled, crossover trial investigating 3 months of oxytocin.. Clinical endocrinology, 94(5), 774-785. https://doi.org/10.1111/cen.14387