TY - JOUR
T1 - The use of oxytocin to improve feeding and social skills in infants with prader-willi syndrome
AU - Tauber, Maithe
AU - Boulanouar, Kader
AU - Diene, Gwenaelle
AU - Çabal-Berthoumieu, Sophie
AU - Ehlinger, Virginie
AU - Fichaux-Bourin, Pascale
AU - Molinas, Catherine
AU - Faye, Sandy
AU - Valette, Marion
AU - Pourrinet, Jeanne
AU - Cessans, Catie
AU - Viaux-Sauvelon, Sylvie
AU - Bascoul, Celine
AU - Guedeney, Antoine
AU - Delhanty, Patric
AU - Geenen, Vincent
AU - Martens, Henri
AU - Muscatelli, Francoise
AU - Cohen, David
AU - Consoli, Angele
AU - Payoux, Pierre
AU - Arnaud, Catherine
AU - Salles, Jean Pierre
N1 - Publisher Copyright:
© 2017 by the American Academy of Pediatrics.
PY - 2017/2
Y1 - 2017/2
N2 - BACKGROUND AND OBJECTIVES: Patients with Prader-Willi syndrome (PWS) display poor feeding and social skills as infants and fewer hypothalamic oxytocin (OXT)-producing neurons were documented in adults. Animal data demonstrated that early treatment with OXT restores sucking after birth. Our aim is to reproduce these data in infants with PWS. METHODS: We conducted a phase 2 escalating dose study of a short course (7 days) of intranasal OXT administration. We enrolled 18 infants with PWS under 6 months old (6 infants in each step) who received 4 IU of OXT either every other day, daily, or twice daily. We investigated the tolerance and the effects on feeding and social skills and changes in circulating ghrelin and brain connectivity by functional MRI. RESULTS: No adverse events were reported. No dose effect was observed. Sucking assessed by the Neonatal Oral-Motor Scale was abnormal in all infants at baseline and normalized in 88% after treatment. The scores of Neonatal Oral-Motor Scale and videofluoroscopy of swallowing significantly decreased from 16 to 9 (P < .001) and from 18 to 12.5 (P < .001), respectively. Significant improvements in Clinical Global Impression scale scores, social withdrawal behavior, and mother-infant interactions were observed. We documented a significant increase in acylated ghrelin and connectivity of the right superior orbitofrontal network that correlated with changes in sucking and behavior. CONCLUSIONS: OXT is well tolerated in infants with PWS and improves feeding and social skills. These results open perspectives for early treatment in neurodevelopment diseases with feeding problems.
AB - BACKGROUND AND OBJECTIVES: Patients with Prader-Willi syndrome (PWS) display poor feeding and social skills as infants and fewer hypothalamic oxytocin (OXT)-producing neurons were documented in adults. Animal data demonstrated that early treatment with OXT restores sucking after birth. Our aim is to reproduce these data in infants with PWS. METHODS: We conducted a phase 2 escalating dose study of a short course (7 days) of intranasal OXT administration. We enrolled 18 infants with PWS under 6 months old (6 infants in each step) who received 4 IU of OXT either every other day, daily, or twice daily. We investigated the tolerance and the effects on feeding and social skills and changes in circulating ghrelin and brain connectivity by functional MRI. RESULTS: No adverse events were reported. No dose effect was observed. Sucking assessed by the Neonatal Oral-Motor Scale was abnormal in all infants at baseline and normalized in 88% after treatment. The scores of Neonatal Oral-Motor Scale and videofluoroscopy of swallowing significantly decreased from 16 to 9 (P < .001) and from 18 to 12.5 (P < .001), respectively. Significant improvements in Clinical Global Impression scale scores, social withdrawal behavior, and mother-infant interactions were observed. We documented a significant increase in acylated ghrelin and connectivity of the right superior orbitofrontal network that correlated with changes in sucking and behavior. CONCLUSIONS: OXT is well tolerated in infants with PWS and improves feeding and social skills. These results open perspectives for early treatment in neurodevelopment diseases with feeding problems.
UR - http://www.scopus.com/inward/record.url?scp=85014101624&partnerID=8YFLogxK
U2 - 10.1542/peds.2016-2976
DO - 10.1542/peds.2016-2976
M3 - Article
C2 - 28100688
AN - SCOPUS:85014101624
SN - 0031-4005
VL - 139
JO - Pediatrics
JF - Pediatrics
IS - 2
M1 - e20162976
ER -