Journal
JOURNAL OF PEDIATRICS
Volume 158, Issue 2, Pages 337-339Publisher
MOSBY-ELSEVIER
DOI: 10.1016/j.jpeds.2010.07.006
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Funding
- NCI NIH HHS [P30 CA006973] Funding Source: Medline
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Patients with rapid-onset obesity, hypothalamic dysfunction, hypoventilation, autonomic dysregulation, and neural crest tumor syndrome have poor long-term outcomes. We report a patient who was treated successfully with high-dose cyclophosphamide immunoablation. This experience offers a novel therapeutic approach and an indirect insight into the underlying pathogenesis of this syndrome. (J Pediatr 2011;158:337-9)
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