Distal Innervation in a Child Affected from a 3 Syndrome of Caudal Regression

Caudal regression syndrome (CRS) is a congenital abnormality characterized by an 17 incomplete development of the spinal cord (SC) and other abnormalities. We studied a 9-months 18 old CRS child presenting: interruption of SC at L2-L3 level, sacral agenesis, lack of innervation of 19 the inferior limbs (flaccid paraplegia) and neurogenic bladder and bowel. Given the effects of 20 growth hormone (GH) on the proliferation, differentiation and migration of neural stem cells 21 (NSCs), we treated him with GH and rehabilitation, trying to induce the recovery of main sequelae. 22 GMFM-88 test score was 12.31%. After a blood analysis, GH treatment (0.3 mg/day, 5 days/week, 3 23 months and then 15 days without GH) and rehabilitation commenced. This protocol was followed 24 during 5 years, being the last GH dose 1 mg/day. Blood analysis and physical exams were performed 25 every 3 months initially and every 6 months later. Six months after commencing the treatment 26 GMFM-88 score increased to 39.48%. Responses to sensitive stimuli appeared in most of the 27 territories explored; 18 months later sensitive innervation was complete and the patient moved any 28 muscle over the knees and controlled his sphincters. Three years later he walked with the help of 29 canes, there was plantar flexion and GMFM-88 score was 78.48%. In summary, GH plus 30 rehabilitation may be useful for innervating distal territories, below the level of the incomplete 31 spinal cord in CRS. Most likely, GH acts on ependymal SC NSCs, as the hormone does in the 32 neurogenic niches in the brain. 33