TY - JOUR
T1 - Improved behavior and neuropsychological function in children with ROHHAD after high-dose cyclophosphamide
AU - Jacobson, Lisa A.
AU - Rane, Shruti
AU - McReynolds, Lisa J.
AU - Steppan, Diana A.
AU - Chen, Allen R.
AU - Paz-Priel, Ido
N1 - Publisher Copyright:
© 2016 by the American Academy of Pediatrics.
PY - 2016/7
Y1 - 2016/7
N2 - Rapid-onset obesity with hypothalamic dysfunction, hypoventilation and autonomic dysregulation (ROHHAD) is a rare, generally progressive, and potentially fatal syndrome of unclear etiology. The syndrome is characterized by normal development followed by a sudden, rapid hyperphagic weight gain beginning during the preschool period, hypothalamic dysfunction, and central hypoventilation, and is often accompanied by personality changes and developmental regression, leading to substantial morbidity and mortality. We describe 2 children who had symptomatic and neuropsychological improvement after high-dose cyclophosphamide treatment. Our experience supports an autoimmune pathogenesis and provides the first neuropsychological profile of patients with rapid-onset obesity with hypothalamic dysfunction, hypoventilation, and autonomic dysregulation.
AB - Rapid-onset obesity with hypothalamic dysfunction, hypoventilation and autonomic dysregulation (ROHHAD) is a rare, generally progressive, and potentially fatal syndrome of unclear etiology. The syndrome is characterized by normal development followed by a sudden, rapid hyperphagic weight gain beginning during the preschool period, hypothalamic dysfunction, and central hypoventilation, and is often accompanied by personality changes and developmental regression, leading to substantial morbidity and mortality. We describe 2 children who had symptomatic and neuropsychological improvement after high-dose cyclophosphamide treatment. Our experience supports an autoimmune pathogenesis and provides the first neuropsychological profile of patients with rapid-onset obesity with hypothalamic dysfunction, hypoventilation, and autonomic dysregulation.
UR - http://www.scopus.com/inward/record.url?scp=84976902115&partnerID=8YFLogxK
UR - http://www.scopus.com/inward/citedby.url?scp=84976902115&partnerID=8YFLogxK
U2 - 10.1542/peds.2015-1080
DO - 10.1542/peds.2015-1080
M3 - Article
C2 - 27313069
AN - SCOPUS:84976902115
SN - 0031-4005
VL - 138
JO - Pediatrics
JF - Pediatrics
IS - 1
M1 - e20151080
ER -