ESPE Yearbook of Paediatric Endocrinology

To read the full abstract: Ann Neurol 2018;84:611–615

This paper examined the concentrations of arginine vasopressin (AVP) and oxytocin (OXY) in the cerebrospinal fluid (CSF) of autistic children (n=36) compared to controls children without psychiatric disease who needed lumbar puncture for some medical reason (n=36). The authors hypothesized that if these neuropeptides are involved in autism development, differences in levels would be more apparent in CSF than in plasma.

The results showed lower concentrations of CSF AVP in autistic children compared to controls, but no significant difference in OXT. Furthermore, CSF AVP concentrations predicted greater symptom severity in males, but not in females. This work partially confirmed the initial hypothesis: AVP concentrations in CSF are more sensitive to detect a pathologic process within the brain than concentrations in peripheral blood. The lack of association with OXT is surprising as OXT deficit has been widely implicated in the social deficit of autism. In neuroendocrine experimental research, assessment of neuropeptide concentration in CSF in domestic animals is considered as a valuable marker of its concentration in the brain. For instance, this approach has been used to study the effect of Kisspeptin on GnRH secretion in sheep. CSF is also considered as a way to deliver neuropeptides at distance of their synthesis. These results are in accord with recent results showing CSF AVP level is a marker of sociality in nonhuman primates¹.

From a clinical perspective, CSF AVP concentrations performed poorly to correctly assign individuals into one or other group, hence, low AVP CSF cannot be used in the diagnosis of autism. This study has other limitations. As recognised by the authors, in the control group CSF was collected because of some clinical indication and they may not be representative of normal children. Also, for autistic children, lumbar puncture was performed while fasted and under anesthesia, clearly very different than in the control group. However, it is unlikely that these differences explain the lower CSF AVP levels in the autism group.

Oxytocin administration has been proposed to rescue social deficit in both mice and humans. By a similar concept, these results suggest AVP administration as a potential a new way to treat social deficit in autism.

Reference: 1. Parker KJ, Garner JP, Oztan O, Tarara ER, Li J, Sclafani V, Del Rosso LA, Chun K, Berquist SW, Chez MG, Partap S, Hardan AY, Sherr EH, Capitanio JP Arginine vasopressin in cerebrospinal fluid is a marker of sociality in nonhuman primates. Sci Transl Med. 2018;10(439):