We are all likely familiar with the feeding difficulties of infant patients with Prader-Willi syndrome (PWS) but did you ever think that a deficiency of oxytocin could be at play? Maybe if you are an endocrinologist you would be familiar with the fact that adults with Prader-Willi lack adequate amounts of neurons that produce oxytocin, but could that also be the case in infancy? Tauber et al. (10.1542/peds.2016-2976) performed a phase 2 trial of increasing doses of a short 7 day course of intranasal oxytocin to 18 babies with PWS less than 6 months of age receiving the medication every other day, daily, or twice daily and then looked at the ability of these babies to tolerate the dosages and to see if there was improvement in feeding and social skills due to an increase in circulating ghrelin or functional MRI changes. The results are quite impressive despite the small sample size of 6 infants in each group. While dosages did not make a difference, just giving oxytocin did, with improved swallowing, less social withdrawal behavior, and better mother-baby interactions, as well as increased ghrelin and fMRI changes that correlated with improved sucking and behavior. This study surprised us because it will open the door to further studies that may indicate a key role for oxytocin in the early treatment of PWS infants to improve their feeding and social behaviors. To help us better understand what is happening in this study, we asked Dr. Nancy MacIver, endocrinologist from Duke to write a commentary regarding the importance of these findings and their implications for other feeding and social skill disorders. It probably will not be long until we see trials of children with autism spectrum disorders turning to oxytocin as well—but step one is to at least repeat the findings in this study in a much larger clinical sample than 18 PWS patients. We look forward to that happening.
